**Psychosocial Considerations for Children and Adolescents Living with Rare Diseases**

Editors

**Lori Wiener Maureen E. Lyon**

MDPI • Basel • Beijing • Wuhan • Barcelona • Belgrade • Manchester • Tokyo • Cluj • Tianjin

*Editors* Lori Wiener National Cancer Institute USA

Maureen E. Lyon Children's National Hospital USA

*Editorial Office* MDPI St. Alban-Anlage 66 4052 Basel, Switzerland

This is a reprint of articles from the Special Issue published online in the open access journal *Children* (ISSN 2227-9067) (available at: https://www.mdpi.com/journal/children/special issues/ psychosocial considerations).

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## **Contents**

## **About the Editors**

#### **Lori Wiener**

Lori Wiener is co-director of the Behavioral Science Core and Head of the Psychosocial Support and Research Program at the pediatric oncology branch of the National Cancer Institute. As both a clinician and behavioral scientist, Dr. Wiener has dedicated her career to the fields of oncology and pediatric HIV/AIDS. At NCI Pediatric Oncology Branch, she developed a robust clinical and research program that has focused on critical clinical issues such as parental coping, lone parenting, transnational parenting, emotional consequences of medically required isolation, sibling and sibling donor experiences, graph versus host disease, and end-of-life planning. Dr. Wiener has also dedicated a substantial part of her career to applying knowledge from her clinical experience and psychosocial studies to create innovative resources such as books, workbooks, therapeutic games and an advance care planning guide for children, adolescents, and young adults. Each of these resources are distributed worldwide and widely utilized in pediatric centers. Dr. Wiener has published extensively, co-edited the *Pediatric Psycho-Oncology: A Quick Reference on the Psychosocial Dimensions of Cancer Symptom Management*, New York, NY: Oxford University Press, 2015 and *Pediatric Psychosocial Oncology: Textbook for Multi-disciplinary Care*, Springer International Publishing, 2016. She has been part of the leadership team that has developed the first evidence-based psychosocial standards of care for children with cancer and their family members.

#### **Maureen E. Lyon**

Maureen E. Lyon is a Clinical Health Psychologist at Children's National Hospital and a Professor of Pediatrics at George Washington University School of Medicine and Health Sciences. As both a clinician and principal investigator, Dr. Lyon has focused her research on advance care planning interventions for persons living with serious illness, including HIV, cancer, and ultra-rare diseases. Dr. Lyon completed three National Institutes of Health 5-year clinical trials of FAmily CEntered (FACE) advance care planning; a 2-year pilot trial of FACE for family caregivers of children with ultra-rare diseases; and a study of gender effects on advance care planning outcomes. Dr. Lyon adapted the FACE protocol for Spanish speaking persons. She co-edited the book Teenagers, HIV, AIDS: Insights from Adolescents Living with the Virus in 2006 with Dr. Lawrence D'Angelo. Dr. Lyon led community based participatory research demonstrating the benefits of advance care planning.

## *Editorial* **Special Issue: Psychosocial Considerations for Children and Adolescents Living with a Rare Disease**

**Maureen E. Lyon 1,2,\* and Lori Wiener 3**

	- Bethesda, MD 20892, USA<EMAIL_ADDRESS>
#### **1. Introduction**

This Special Issue of the journal *Children* constitutes an opportune moment to reflect on the psychosocial needs of children living with rare diseases and of their families. As medical advances, treatments, and developments have enabled many of these children to survive infancy and to live into adulthood, progress brings with it concerns and opportunities to enhance the psychosocial quality of life of children living with rare diseases, and of their families.

In August of 2021, we released a call for papers whereby healthcare providers could share their experience and research on the psychosocial needs of children living with rare diseases, and the needs of their families. Our call resulted in 13 accepted peer-reviewed submissions. The manuscripts covered a diverse range of topics and contributions from around the globe, including Asia (Taiwan), Australia, Europe (Germany, Italy, Sweden, and The Netherlands) and the United States.

We acknowledge that, as is often the case with Special Issues and their time constraints, the manuscripts within this Special Issue do not cover or represent all the potentially important contributions to the topic. However, as international perspectives are shared, we hope this Special Issue leads to future research collaborations. It is also our hope that the data presented in this Special Issue will ultimately reduce the systemic and structural inequities that place children with rare diseases at unfair, unjust, and avoidable disadvantages with respect to their quality of life and that of their families.

This Special Issue reflects the current state of psychosocial research, which is primarily qualitative in nature. There are no scientifically rigorous randomized clinical trials to create an evidence base of effective psychosocial interventions for the provision of care to children with rare diseases and to their families; nevertheless, the papers within this Special Issue provide a reflection on the state of the science, including ideas about future research and practice. In this next section we share observations about the contributions made by each of the 13 articles, which cover a diverse range of topics.

#### **2. Contributions to the Special Issue**

Belzer, Wright, Goodwin, Singh, and Carter provide a thorough, thoughtful, and comprehensive overview of psychosocial considerations for the child with a rare disease, including recommendations and a call to action [1]. Of particular importance, the authors note the experience of stigma and social isolation amidst the medicalization of homes and family lives, and the need for care coordination. The authors call for a focus on the intersectionality of identities (e.g., gender, race, and poverty), experiences, and care models. The impact of the social determinants of health, known to contribute to inequity outcomes, has not been fully characterized; this is, in part, because of small sample sizes. The importance of the child voice (when possible), of the family as part of the care and

**Citation:** Lyon, M.E.; Wiener, L. Special Issue: Psychosocial Considerations for Children and Adolescents Living with a Rare Disease. *Children* **2022**, *9*, 1099. https://doi.org/10.3390/ children9071099

Received: 15 July 2022 Accepted: 18 July 2022 Published: 21 July 2022

**Publisher's Note:** MDPI stays neutral with regard to jurisdictional claims in published maps and institutional affiliations.

**Copyright:** © 2022 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https:// creativecommons.org/licenses/by/ 4.0/).

research team, of gender differences in caregiving, of sibling caregiving, of access barriers in the community, of electronic health record functionality with documentation, and of the transition to adult healthcare is also reviewed here and further explicated in the articles within this Special Issue.

Each rare disease is unique with respect to the specific medical and psychosocial needs associated with the condition. Additionally, many studies only include the parent perspective because the child's rare disease involves communication and/or neurocognitive disorders that preclude the child's participation. Sharping and colleagues identify the unmet needs of the parents of children with urea-cycle disorders residing in Germany [2]. Using the validated Parental Need Scale for Rare Diseases Questionnaire, 59 parents reported on the needs of 24 children. Close to half of the parents reported a need for information on available services, and one-third on the need for additional information on the development of their children. More than two-thirds reported a need for additional support such as support groups or psychological counseling. The authors conclude that the findings underscore the importance of family-centered approaches to care. We concur that the template they used for their assessment of family burden could be used for children with other rare diseases, increasing the replicability of study findings, as children with rare diseases and their families share many commonalities in their need for knowledge about their or their children's conditions and desire for support.

Similarly, through data collected via a structured psychosocial interview and the Distress Thermometer/Problem Checklist, Lockridge and colleagues [3] found that patients (*n* = 63) with Multiple Endocrine Neoplasia 2 (MEN2B) and Medullary Thyroid Carcinoma (MTC) identified the need for information about available services and education about MTC as high-priority. MEN2 is a genetic cancer syndrome for which there are limited data pertaining to the quality of life and psychosocial experiences of persons affected. Over half of the pediatric patients reported experiencing attention challenges and difficulty concentrating. While pediatric and adult patients identified pain as interfering with their mood and daily activities, the parents of pediatric patients reported mood shifts as most concerning, thus highlighting the importance of both the child voice and parental perspectives. The children and parents agreed that they would want to meet others with this rare condition. The study suggests that the psychological impact of living with MEN2 and MTC extends beyond changes in physical attributes, daily life limitations, and pain and; therefore, it speaks to opportunities for educational and mental-health intervention and further research.

Beckwith-Wiedemann syndrome (BWS) is a rare overgrowth disease and is not usually associated with intellectual delay. In a cross-sectional exploratory study in Italy, assessing psychosocial difficulties in preschool-age children with BWS, Butti and colleagues [4] administered two standardized questionnaires to 30 parents—the Child Development Inventory and the Child Behavior Checklist. The authors found that overall, BWS was not associated with specific behavioral problems; however, at the individual level, almost a quarter of the sample had scores in the borderline range on the withdrawal scale, and half had scores within in the borderline or critical range in the social domain. Increasing age was associated with higher behavioral and developmental difficulties. Social withdrawal problems were independent of developmental difficulties in the social domains. The authors speculate that children with BWS might become more aware of their condition as they begin spending more time with their peers in social contexts outside the family. They recommend that children with BWS receive routine psychosocial assessment of emotional and psychosocial development as they enter kindergarten and elementary school. This could have beneficial effects on the national health system in Italy, reducing costs associated with the long-term consequences of neglected emotional–behavioral problems.

In a mixed-methods study, Chu and colleagues [5] explored gender differences in parenting stress, health outcomes, and illness perceptions among 100 family caregivers (42 men and 58 women) caring for children with genetic or rare diseases in Taiwan. Measures included the Pediatric Inventory for Parents (PIP) to assess caregiver distress, the

Center for Epidemiological Studies Depression Scale Short Form (CES-D Sort Form) to assess caregiver depression, and the Satisfaction with Life Scale (SWLS) to assess life satisfaction [5]. Open-ended questions were theoretically informed, using Leventhal's Common-Sense Model of Illness Representation. Consistent with prior research, most female caregivers served as the primary caregiver and provided more caregiving, while experiencing high levels of parenting distress and depressive symptoms compared with male caregivers. The authors identified a gender discrepancy in illness perception (negative consequences requiring disease control vs. quality of life), which may have contributed to the higher levels of stress and depressive symptoms in female caregivers than in males.

The standardized documentation of psychosocial concerns is the first step in improving the ability of healthcare providers to identify and intervene in psychosocial concerns and their risk factors. The documentation of psychosocial distress and its antecedents in children with rare diseases is often not captured in the medical record, as illustrated in McCarthy and colleagues' article [6]. The medical records of patients with rare or life-limiting chronic conditions (*n* = 60) being followed by a pediatric complex care coordination program in the United States were reviewed. The authors extracted both structured data elements and narrative text from the most recent visit with the clinician. Topics related to psychosocial distress were documented in notes, including child and parent emotional problems, parent social support, sibling emotional or physical problems, family structure, and financial concerns. However, 35% of the notes lacked any mention of psychosocial concerns and mention of parents' emotional health or concerns was largely absent. Risk factors and vulnerabilities of the family system (i.e., financial, sibling) were also rarely captured. The authors emphasized the need for universal psychosocial screening using structured, evidence-based tools, systematically entered into the medical record as a way to contribute to an integrated medical and behavioral service model.

Among nonhuman primates, siblings are "helpers at the nest." Therefore, it is not surprising that the siblings of children with rare diseases also function in this role, as demonstrated in the studies of Wawrzynski and colleagues [7] in the United States and Kreicbergs and colleagues in Sweden [8]. Their studies highlight the need for social support for the siblings as well as the patients. In semi-structured interviews of siblings aged 12–17 years, Wawrzynski and colleagues constructed ecomaps of support networks, including types of support and of support provider. Support networks ranged from 2–10 individuals, with mothers, fathers, close friends, and siblings, with and without cancer being major supports, in that order. We concur that this foundational knowledge of sibling networks will contribute to the design of interventions to improve support for the siblings of children with rare diseases, including cancer.

Little is known about the need for information and the involvement of the siblings of children with palliative care needs. Kreicbergs and colleagues, using four standardized communication tools (See–Hear–Do pictures, including the empty body as a separate element, Bear cards, and words originating from previous sibling research), conducted a conventional content analysis of the responses of nine siblings aged 6–14 years [8]. Most striking was that these siblings expressed an awareness that their brother or sister would die—"not if, but when". School was perceived as a place for leisure, friends, and learning. Relentless feelings of guilt and self-blame, as well as themes of loss and separation were elicited. Nevertheless, these siblings also felt they were part of a special, happy family.

Several other papers address palliative care needs for children and adolescents living with rare conditions. Aoun and colleagues assessed the support needs of 28 parents whose children were receiving pediatric palliative care (*n* = 20 with non-cancer diagnosis, *n* = 8 with cancer diagnosis) [9]. With the study conducted in Australia, the authors used structured telephone interviews upon parents' completion of an intervention using the Carer Support Needs Assessment Tool (CSNAT), a process for assessing the palliative care needs of children and their families. The interviews were audio-recorded and transcribed verbatim. The authors found that the parents appreciated a systematic approach in engaging them in conversations about both their needs and solutions to address them. Similar to

other studies, the interviews elicited the following themes: caregiving challenges; perceived gaps in psychosocial care and feelings of isolation; and validation and empowerment when participating in the CSNAT intervention, which helped them identify strategies and receive support in response to their needs. Nevertheless, parents were left wanting practical psychosocial and emotional support. Aoun and colleagues recommend that palliative care services build stronger partnerships with supportive community networks through compassionate community volunteer models of care to address the non-clinical needs of families whose children are receiving end-of-life palliative care.

The question of whether or not pediatric advance care planning (pACP) matters to the parents of children with rare diseases, particularly for those children who are unable to participate in decision making, is only beginning to be explored. Fratantoni and colleagues beta tested a pACP intervention with six families [10]. Their article describes a qualitative analysis of structured interviews examining what parents thought mattered most to their child and what they would want their doctor to know. Five themes emerged that might guide future interventions: getting out and moving freely; feeling included and engaged; managing symptoms and disease burden; coordinating care among the many care team members; and managing today and planning for the future. The parents strived to be effective advocates on their children's behalf.

Brunetta and colleagues conducted a systematic review of the literature on pACP, with a focus on how to operationally define age-appropriate pACP for children living with a life-limiting condition [11]. They identified 18 unique tools. These tools primarily assessed the preferences of the children and their families concerning their goals for care and end-of-life treatment preferences. In most studies, the children were adolescents who were able to participate in decision making. This article is well-organized, beginning with evidence from randomized control trials, observational studies, mixed-methods studies, qualitative studies, and descriptive studies. The authors identify six factors influencing ageappropriate care from the literature: willingness to participate; decision-making capacity; a child's understanding of their own medical process; cognitive impairment; the development of a social identity (defined as an awareness of self and others that influences children's preferences and goals in pACP); and legal responsibilities. The authors call for a more explicit explanation for the choice of age. For example, in adapting adult models for adolescents, it is important to address how the adaptations meet the developmental needs or capacities of the children studied. The authors also call for future studies to specify race and ethnicity.

Two studies addressed the needs of adolescents and young adults living with a chronic or rare condition. A quality-improvement study assessing the needs of adolescents and young adults (*n* = 89) with neurofibromatosis type 1, cancer, primary immunodeficiencies, or sickle cell disease, and of their caregivers (*n* = 37), was conducted by Allen and colleagues [12]. The subjects completed a survey developed for this study to identify a range of informational and service-related needs. Consistent with the other studies in this Special Issue, there was an overwhelming desire for information about their specific disease. The authors conclude that this is a critical and largely unmet component of care which requires the development and implementation of targeted educational and psychosocial interventions. Considering most adolescents and young adults have access to smartphone apps and web-based services, the authors suggest that future research should utilize digital technologies to expand services and address informational needs.

The transition to adulthood for youth living with chronic illnesses is complex. Sandquist and Lyon—in partnership with Davenport and Monaco, who are parents of children with rare diseases—provide a useful international review of the literature on challenges specific to the transition to adulthood for youth living with rare diseases [13]. Their review found that transitional support is lacking, particularly for maturing psychosocial needs. Many programs that do exist assume the young person can participate in decision making and live independently, which may not be the case for many young people with rare diseases. The parents of children with neurological conditions that impair decision making and/or independent living are often surprised to discover that they need to establish legal guardianship over their children when their child becomes a legal adult and prove their competency as caregivers to the government. The barriers and challenges to transition to adult care are identified, including the need for programmatic support. The authors conclude that a large portion of children with rare diseases are underserved and experience health disparities in the transition.

While the papers published in this Special Issue provide important new knowledge, more work is required in several areas. Discovering effective approaches to improving the quality of life of children with rare diseases and of their families necessitates addressing the social determinants of health, which, in turn, should inform clinical practice and policy. We need to examine the systemic and structural problems that contribute to health disparities and consider ascertaining psychosocial needs through new systems and models of care. Macro-level interventions at the population or community-health level can help meet the psychosocial challenges that persistently affect children living with rare diseases and their families. Finally, we recognize that, along with the caveats of what is still needed, there are some wonderful programs available for children, adolescents, and young adults living with rare conditions. Unfortunately, it is not possible to list them all here.

**Author Contributions:** Writing—original draft preparation, M.E.L.; writing—review and editing, L.W. All authors have read and agreed to the published version of the manuscript.

**Funding:** This work was funded, in part, by the Intramural Research Program of the National Cancer Institute; the National Institutes of Health (Lori Wiener); and, in part, by the NINR/NIH (National Institute of Nursing Research/National Institutes of Health) Award # R21NR019340 (Maureen Lyon). The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

**Acknowledgments:** We would like to thank the authors who worked so diligently to present their work and respond so thoughtfully to the reviewers' comments and suggestions.

**Conflicts of Interest:** The authors declare no conflict of interest.

#### **References**

## *Article* **The Social Networks and Social Support of Siblings of Children with Cancer**

**Sarah E. Wawrzynski 1,2,\*, Melissa A. Alderfer 3,4, Whitney Kvistad 5, Lauri Linder 1,2, Maija Reblin 6, Jia-Wen Guo 2 and Kristin G. Cloyes 2**

**Abstract:** Siblings of children with cancer need support to ameliorate the challenges they encounter; however, little is known about what types and sources of support exist for siblings. This study addresses this gap in our understanding of the social networks and sources of support for adolescents with a brother or sister who has cancer.  During semistructured interviews, siblings (ages 12–17) constructed ecomaps describing their support networks. Data were coded for support type (emotional, instrumental, informational, validation, companionship) and support provider (e.g., mother, teacher, friend). Network characteristics and patterns of support were explored. Support network size ranged from 3 to 10 individuals (M = 6 ± 1.9); siblings most frequently reported mothers as sources of support (n = 22, 91.7%), followed by fathers (n = 19, 79.2%), close friends (n = 19, 79.2%) and siblings (with or without cancer) (n = 17, 70.8%). Friends and brothers or sisters most often provided validation and companionship while instrumental and informational supports came from parents. This study provides foundational knowledge about siblings' support networks, which can be utilized to design interventions that improve support for siblings of children with cancer.

**Keywords:** cancer; childhood cancer; adaptation; psychological; neoplasm; oncology; sibling; social support; social adjustment

#### **1. Introduction**

A pediatric cancer diagnosis causes disruptions within the family including shifting of roles, finances, and resources. The focus on the needs of the diagnosed child often leaves siblings feeling anxious, alone, and distracted [1,2]. Siblings may experience poor psychosocial adjustment, including poor school functioning, cancer-related traumatic stress, and poorer quality of life [3–5]. Due to distress and challenges with adjustment among siblings of children with cancer [4,6], supportive care, including providing education and psychological supports, for siblings is recommended as a standard of care in pediatric oncology [7].

Social support is broadly defined as the provision of assistance, comfort, or resources to individuals that alleviate stress and assist in coping [8]. The stress-buffering hypothesis of social support [9] has been extensively explored and suggests that social support offers resources and promotes coping to buffer stress. Social support is well established as a key factor in health outcomes and adjustment in children and adolescents [10–13].

**Citation:** Wawrzynski, S.E.; Alderfer, M.A.; Kvistad, W.; Linder, L.; Reblin, M.; Guo, J.-W.; Cloyes, K.G. The Social Networks and Social Support of Siblings of Children with Cancer. *Children* **2022**, *9*, 113. https:// doi.org/10.3390/children9010113

Academic Editors: Lori Wiener and Maureen E. Lyon

Received: 18 December 2021 Accepted: 13 January 2022 Published: 15 January 2022

**Publisher's Note:** MDPI stays neutral with regard to jurisdictional claims in published maps and institutional affiliations.

**Copyright:** © 2022 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https:// creativecommons.org/licenses/by/ 4.0/).

Few studies have examined siblings' perspectives of their social support or associations between support and adjustment [14,15]. A recent scoping review suggested that social support is indeed helpful to siblings; however, the most important sources and types of helpful support for siblings of children with cancer remain unclear [16].

The purpose of this study was twofold. First, we aimed to advance our understanding of social support among siblings by identifying sources and types of support within their social networks. Second, we aimed to identify how support sources and types of support given to siblings alleviates the "hardest things" they have encountered since the cancer diagnosis of their brother or sister through their own narratives.

#### **2. Materials and Methods**

#### *2.1. Participants*

All study procedures were approved by the Institutional Review Board at the University of Utah (protocol #00124303). Eligible participants were healthy adolescent siblings (age 12–17) of children in active treatment for cancer or off treatment but diagnosed within the last two years. Our sampling was purposive to ensure representation of varied ages and genders of participants because developmental age and gender are known to influence perceptions of support [10,17]. Siblings were English-speaking, nonbereaved, and living in the home of the child with cancer at least 50% of the time. Table 1 provides participant, family, and cancer diagnosis demographics.

**Table 1.** Participant Demographics.

1 Two families did not wish to disclose income.

#### *2.2. Screening and Recruitment*

Two methods were used to recruit participants for this study. First, we used the electronic health records (EHR) at Primary Children's Hospital, a quaternary pediatric oncology center serving the Intermountain West of the United States, to identify pediatric patients diagnosed with cancer within the last two years. Second, we partnered with SuperSibs, a program of Alex's Lemonade Stand Foundation, to identify families with eligible siblings. SuperSibs is a free program providing comfort and care mailings to siblings of children with cancer. Parents identified through both sources received emails inviting participation in a study of sibling social networks and support. They were then contacted by phone to answer any questions and determine interest in participation. Interested caregivers completed a screening survey to confirm sibling eligibility. If eligible, parental permission and demographic information were collected via a Research Electronic Data Capture (REDCap) [18] link, along with contact information for the target sibling. Eligible siblings were then sent study information and invited to participate. Interviews were conducted after documentation of assent.

#### *2.3. Data Collection*

Participants completed audio-recorded interviews that lasted 20–47 (*M* = 30) minutes and took place via Zoom [19]. Participants worked with the researcher to build an ecomap [20], a visual representation of their social network, by identifying up to 10 people they perceived as a source of support throughout their brother or sister's cancer experience. Participants described characteristics of their social support networks (e.g., relationship, age, and closeness) and interactions with members (e.g., frequency, type of support) to complete the ecomap (see Appendix A, Table A1). Participants were asked about the kind of support each person provided to them using a set of terms and examples formulated in lay language developed and tested in previous work [21]. However, they were also allowed to freely describe specific or recent examples of the support received from each source they mentioned in their ecomap without using the terms provided.

Participants were also asked to describe what they felt was the "hardest thing" for them since their brother or sister's diagnosis along with any support they felt helped in dealing with or alleviating stress related to their identified "hardest thing".

#### *2.4. Data Integration and Analysis*

Demographic and ecomap data were summarized using descriptive statistics (mean, percentage) which were generated using SPSS version 26 [22]. Audio recordings of participant interviews were transcribed verbatim. Transcripts were then de-identified and imported into Dedoose [23] for management and coding. Participant demographic data were imported as case descriptors and linked with interview data.

To ensure theoretical and empirical coverage of the data, analysis took place in two stages of deductive and inductive coding. First, we used a deductive approach applying codes and definitions derived from theoretical constructs, interview questions, and review of the ecomap data (e.g., relationship and interaction characteristics, types of perceived support) [24]. This preliminary deductive coding scheme was refined by four members of our research team (SEW, WK, MA and KGC) by coding two interviews together. Primary coders (SEW and WK) then independently coded a series of interviews, discussing inconsistencies in coding and refining use of the code book after each one until reliability was established by achieving a Kappa within the "substantial" range [25], at 0.86 after four transcripts. Coders then independently coded the rest of the transcripts resolving discrepancies at weekly meetings.

Next, an inductive open coding approach with thematic analysis was undertaken to ensure novel content was captured and integrated into the coding [26]. Throughout the coding process, reflexive and analytic memos were recorded directly into Dedoose alongside the data to enhance the description and understanding of the data [27,28]. In the second phase of coding, the authors reviewed and discussed the codes and memos to develop themes and summarize the data. Codes and subcodes were organized by conceptual similarity, subsuming the initial codes within emergent thematic categories.

#### **3. Results**

#### *3.1. Sample Demographics*

Twenty-four siblings between the ages of 12 and 17 were included in this study (Table 1). Half were male and half were female. Most siblings were white, but nearly one-third represented racial or ethnic minorities. All siblings came from traditional (mother, father, siblings) or blended (divorced and remarried or cohabitating adults and their children) two-parent homes; however, families varied regarding income, parental education, cancer diagnosis, and time since diagnosis.

#### *3.2. Sources of Support*

Ecomaps indicated that siblings' cancer social support networks ranged from 3 to 10 individuals. A total of 162 individuals were identified as sources of support in our sample's ecomaps, with each individual providing one to three types of support. Siblings reported an average closeness rating of each supporter at 4.5 (SD 0.78, range 1–5) with 5 indicating the greatest perceived closeness to the individual. On average, siblings' social networks were primarily made up of family members (71.6%, range 33–100%). Figure 1 shows the type of relationships included in each sibling's ecomap network by percent. Mothers were identified as a source of support by nearly all participants. The next most frequently mentioned sources of support were close friends and fathers, then a brother or sister in the home. One-third of siblings (33.3%) mentioned a teacher, school counselor, or coach, and 29.1% included their family pet in their support network. Several siblings mentioned a group of individuals as a single source of support in their ecomap, for example, a sports team, their local community, or a neighboring family who offered them important supports throughout the cancer journey (Figure 1).

#### *3.3. Types of Social Support Received*

Siblings identified support within all six deductively derived domains, including emotional, informational, instrumental, companionship, and validation support. Two additional types of support, appraisal support and indirect support, were identified via inductive coding. In total, N = 383 examples of support were identified across all interviews; what follows is a summary of the specific supports reported by siblings within each domain of support. Percent of support provided their most frequent sources are also included. Definitions for each type of support and exemplary quotes are noted in Table 2.

Emotional support was the most frequently identified type of support siblings reported (N = 144/383, 37.6%) receiving from their social network members. Examples of emotional support among siblings often related to encouragement and "check-ins" where the identified source of support would ask how the sibling was doing or make themselves available to the sibling to talk or answer questions. Some siblings had difficulty identifying a specific example of how emotional support had been given but articulated instead that the source was "just there for them", giving them a sense of presence and availability. Emotional support was provided across all types of sources of support; however, data matrices showed that emotional support was most frequently provided by friends (n = 35, 24.3%), mothers (n = 25, 17.4%), healthy siblings (n = 18, 12.5%), and fathers (n = 17, 11.8%).

**Figure 1.** Percentage and number of siblings reporting each mentioned source of support.

**Table 2.** *Cont.*

Companionship was the second most frequently reported type of support among siblings (N = 80/383, 20.9%). Companionship primarily served the purpose of distraction for siblings. Companionship allowed siblings to feel normal, have fun and escape the cancer experience. This was in the form of sports activities, "hanging out" with friends, or a one-on-one trip to the store with a parent or older sibling. While parents and other sources of support were identified as providing companionship, friends (n = 22, 27.5%) and healthy siblings (n = 20, 25%) were the most frequently identified sources of companionship.

Siblings also identified instrumental support (N = 53/383, 13.8%) in a variety of forms. Several siblings mentioned receiving meals or having a place to stay while their parent(s) were at the hospital. Sometimes instrumental support was help with homework, sports, or facilitating sibling participation in their interests or activities. Instrumental support was

most frequently provided by fathers (n = 14, 26.4%), followed by mothers (n = 11, 20.8%) and aunts (n = 8, 15.1%). Instrumental support provided by parents also included acting as a link to other sources or types of support. For example, parents were able to connect siblings to teachers, therapists, or extended family in the sibling's social network who provided support.

Informational support was the next most identified support (N = 50/383, 13.1%). Informational support most often related to someone providing information about cancer, its treatment, or the effects of treatment. Several siblings mentioned someone providing information that supported them in dealing with specific challenges such as tips on interacting with the child with cancer or help with homework. Informational support was most frequently identified as being provided by mothers (n = 16, 32%), fathers (n = 13, 26%), and healthy siblings (n = 8, 16%). Teachers and aunts were also mentioned by some.

Validation was identified as having someone who understands you or your experience; this was typically related to the cancer experience (N = 32/383, 8.4%). Validation was most frequently provided by healthy siblings (n = 8, 25%) and friends (n = 7, 21.9%).

We also noted two additional types of support described by participants that did not fit our initial coding approach. First, appraisal support was identified, comprised of comments or behaviors from individuals in the siblings' network that assisted in the sibling's self-evaluation or their appraisal of their social situation. Appraisal support (N = 15/383, 3.9%) was occasionally related to cancer, but most often related to feedback or affirmation given to the sibling unrelated to cancer, such as praise for an accomplishment or help with typical adolescent interpersonal issues. Appraisal support was provided by fathers, healthy siblings, and the children with cancer with the same frequency (n = 3, 20%) followed similarly by mothers and friends (n = 2, 13.2%).

Second, indirect support occurring at the family level was identified (N = 9/383, 2.3%). This type of support was not directed at the sibling specifically but provided support targeted at alleviating family stressors, which provided siblings with the added benefit of feeling more secure in their situation. Examples of this included community fundraisers, GoFundMe campaigns, care of their family needs, or the care their brother or sister was receiving for their cancer. Siblings who identified these supports conveyed that these types of support helped them feel loved, watched over, or provided a sense of comfort regarding their worries about their brother or sister with cancer. Indirect support was identified as coming from medical professionals (n = 3, 33.3%), fathers (n = 2, 22.2%), and community (n = 2, 22.2%) most often. Frequencies of reported support by gender are noted in Table 3. Chi-square analysis on gender differences in reported support was significant, (X2 (df = 6) = 15.21, *p* = 0.019; Cramer's V = 0.20) and appraisal support was noted to be the type of support contributing significance.

**Table 3.** Frequency of Social Supports Reported by Gender.

#### *3.4. Hardest Things and Most Helpful Supports Reported by Siblings*

Sibling reports of the "hardest thing" they had dealt with related to their brother's or sister's cancer aligned with their reports of the most helpful supports and the inductive themes identified in our analysis. In Table 4, we provide examples of what siblings identified as the hardest things, their most helpful supports, along with the overarching themes noted in the data. Specifically, we identified involvement in family and care, distraction, creating connections and presence, and understanding as most relevant in addressing their identified challenges.

**Table 4.** Hardest and most helpful supports reported by siblings.

**Table 4.** *Cont.*

Seeing their brother or sister sick or their parent's distress was most frequently reported as the "hardest thing". Siblings reported struggling with being treated differently by parents, friends, or others. Siblings felt more alone and limited in their interactions with friends or normal routines and their social networks provided a sense of security, integration, and normalcy that was important to the siblings in coping with their identified challenges.

Siblings identified their own ability to provide support to their family as important. Siblings took on caregiving activities out of a seeming desire to be a part of the family through the cancer trajectory. Siblings discussed the importance of spending time with their brother or sister with cancer, seeing improvements in their health, or even their joy however brief. Siblings also wanted to be a support to parents. One sibling even mentioned seeking to understand their mothers' challenges through an aunt in their social network. Overall, siblings conveyed that assisting with the care of other healthy siblings or their brother or sister with cancer provided siblings with a sense of integration, purpose, and visibility within their family.

Distraction, often occurring through companionship, allowed siblings to feel close to their family and gain a sense of normalcy while dealing with the distress and changes caused by cancer. Concurrently, instrumental support, such as transportation or money, was important to provide the means for siblings to spend time with friends and engage in extracurricular activities, which further supported distraction from cancer.

Most siblings reported that members of their social network were aware of their challenges and were available to them. This created meaningful connections and a presence felt by siblings. Checking in and knowing people were watching out for them were often expressed as important emotional supports. Even when discussing other forms of support, the emotional significance of the time or support an individual provided was simultaneously expressed by siblings.

Finally, siblings expressed understanding as important. This occurred in two ways. First, siblings wanted "real" information. Siblings could see the distress of their parents, siblings, and others. Information that helped siblings grasp the situation, gain perspective, and feel grounded and was identified as one of the most important supports in dealing with their brother or sister's cancer. Second, siblings felt understanding related to their feelings and challenges helped them feel seen. This type of understanding was identified in

things like having extra time to do a homework assignment and leeway in their emotions and processing of the situation.

#### **4. Discussion**

Previous work has identified that siblings of children with cancer are at risk for poor adaptation, difficulties in school, and altered relationships with members of their social networks [4,6]. Barriers to supporting siblings have also been identified [29,30], and structured support may not be available to many siblings. COVID-19 has further limited access to supportive services [31]. Notably, no siblings in our study mentioned receiving support or information from the oncology team or hospital for themselves, and when formal services from a therapist were obtained for siblings, it was reported as having occurred as a result of a parent or teacher concern and support.

In this study, we aimed to advance our understanding of social support among siblings and to fill a gap in the literature by characterizing sibling social support networks and identifying the sources and types of support they find helpful. We identified that existing, informal supports were most meaningful and helpful to siblings during their brother or sisters' cancer and that this support most often came from sources closest to them. Siblings identified specific examples of support across a variety of social support domains. The examples of support received from their networks were relatively typical for adolescents [32,33], contributed to the siblings' sense of security, and made them feel cared for during the stressful experience of cancer within the family. These findings are consistent with other research demonstrating siblings challenges [15] and their desire to be seen and involved when a brother or sister has cancer [1,2].

In this study, we also aimed to identify through narratives how the sources and types of support given to siblings alleviate the "hardest things" they have encountered since their brother or sister's cancer diagnosis. Sibling social networks were primarily made up of family members and close friends, highlighting the importance of support within close relationships. Unfortunately, lack of awareness of sibling support needs within the family is a recognized barrier to sibling support [30]. Based on our findings, siblings seem to benefit from meaningful connections formed when others regularly check in on their well-being and allow them space to express their specific needs. In turn, as others learn of siblings' needs, they may be better able to provide congruent support or seek out appropriate professional support when needed.  In addition, while most support came from parents, others such as extended family or community can support siblings (and parents) by providing these types of support.

Siblings in our study expressed being acutely aware of the challenges that their diagnosed brother or sister and parents faced and the implications of this on themselves. Siblings also indicated that their parent and family members' well-being was important and contributed to their own sense of emotional security and coping. Previous research has documented similar findings noting that pre-existing family challenges, inadequate resources, or poor parental coping can contribute to poor adjustment to cancer in all family members including siblings [34,35]. Clinicians can routinely assess for these psychosocial issues within families, stratify risk, and improve health equity using tools like the Psychosocial Assessment Tool [36].

Important sibling supports were often related to being seen, involved, or part of the family. Our overarching theme of being "involved" may be more about siblings leveraging their own power to create or enhance cohesion and connection between themselves and their important supporters, rather than a desire for increased responsibility at home or in the care of the child with cancer. These findings align with family systems theory [37] and suggest that family focused interventions may be the most impactful for siblings because positive changes within their relationships with their most important social network members—family members—may enhance intervention effects.

Clinicians treating children with cancer can use our findings to offer additional evidence and guidance to parents about keeping adolescent siblings involved, supported, and connected as they navigate the cancer trajectory. Our findings point to helpful support coming from siblings' existing and informal social networks, available to siblings in their day-to-day activities, outside of structured hospital and community-based interventions. Previous research has demonstrated that siblings of children with other chronic illness experience similar emotional and psychological challenges [38,39] to those of children with cancer. Our reported findings should be compared to those reported by siblings of other childhood illnesses and may be applicable and useful in supporting other sibling groups.

This is among the first studies to report on the social networks of siblings, and our findings should be interpreted with caution. While participant selection was purposive, the sample was relatively small and under-represents the racial and ethnic diversity that is prevalent in the general population of adolescents in the United States [40]. In addition, many children who participated were recruited from SuperSibs, a program that recognizes the needs of siblings of children with cancer. These families may be more aware and in tune with sibling support needs. Finally, our sample was entirely composed of two parent families; the known challenges for single parent families [41] were not integrated into our findings. Our findings may in fact represent a "best case", as participants often expressed having adequate supportive resources.

It is important to note that siblings with supportive resources may still have unmet social support needs if the support they receive is mismatched to their specific challenges. These mismatches of support and need may play a role in poor or ineffective adjustment to the cancer experience. Furthermore, while emotional support was the most frequently reported type of support, it may not be the most needed; rather it may be the most easily offered or cognitively accessible to this age group. More work is needed to determine the specific support needs of individual siblings and how to leverage the supports available to them to promote their healthy adjustment. Future studies could undertake a more traditional social network analysis examining how support, cohesion, or the heterogeneity of their network influences sibling outcomes. Lastly, our research noted some differences in reports of appraisal support by gender, and other research suggests that cultural influences play a role in what supports are desired [42]. Additionally, this generation is the most diverse generation in US history [40] (race, ethnicity, orientation and gender identity), and that should be accounted for in research. Future research efforts should further examine and confirm if specific types of support are more relevant to specific demographic groups or socioeconomic aspects of families.

**Author Contributions:** Conceptualization, S.E.W., K.G.C., M.A.A., L.L., M.R. and J.-W.G.; methodology, S.E.W., K.G.C., M.A.A., L.L., M.R. and J.-W.G.; validation, S.E.W., K.G.C., M.A.A. and W.K.; formal analysis, S.E.W., K.G.C., M.A.A., W.K.; data curation, S.E.W., W.K.; writing—original draft preparation, S.E.W.; writing—review and editing, S.E.W., K.G.C., M.A.A., W.K., L.L., M.R. and J.-W.G.; supervision, S.E.W., K.G.C., M.A.A., L.L., M.R. and J.-W.G.; project administration, S.E.W., K.G.C., M.A.A.; funding acquisition, S.E.W., K.G.C., M.A.A., L.L., M.R. and J.-W.G. All authors have read and agreed to the published version of the manuscript.

**Funding:** Research reported in this publication was supported by the National Institute of Nursing Research of the National Institutes of Health under Award Number F31NR018987 (Wawrzynski, S.E., PI) and K01NR016948 (Guo, J.-W., PI) as well as the National Center for Advancing Translational Sciences of the National Institutes of Health under Award Number UL1TR002538. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. Ms. Wawrzynski was also supported in part by Sigma Theta Tau, Gamma Rho chapter through a dissertation research award.

**Institutional Review Board Statement:** The study was conducted according to the guidelines of the Declaration of Helsinki and approved by the Institutional Review Board of University of Utah (protocol #00124303, 31 October 2019).

**Informed Consent Statement:** Parental permission and informed child assent and were obtained for all subjects involved in the study.

**Data Availability Statement:** The data presented in this study are available upon reasonable request from the corresponding author. The data are not publicly available due because participants of this study did not agree for their data to be shared publicly.

**Acknowledgments:** We would like to thank the siblings who contributed to this study as well as the Sibling Partnership for Advocacy, Research, and Care (SPARC), and SuperSibs powered by Alex's lemonade Stand Foundation who facilitated our connection to participants. We would also like to thank Missy Lusk and Katie Holcomb at Ryan's Case for Smiles, a non-profit that supports families who have a child with a serious illness, for their final review of this manuscript.

**Conflicts of Interest:** The authors have no funding or conflict of interest to disclose.

#### **Appendix A**

**Table A1.** Interview Guide.

#### **ECO-MAPPING INTERVIEW GUIDE**

In this interview we are going to talk about the people in your life that provide you with support and help you.

 This can be people that you see in person every day, or people that you text, talk or chat with online. We're going to make a list of people you feel have supported you the most throughout your sibling's cancer diagnosis and treatments.

Together, we are going to make a diagram of your relationships with these people. Each circle will represent a person that you feel supports you. We are going to put your name in the center circle.

Next let's add the names of the people and relationships you have been thinking about.

We are going to talk about each person on your map here. I'm going to ask you a few questions about each person, including their relationship with you, and how they support you. As we go, you can make changes to the map, by adding or subtracting people from your map.

*For each person identified in the siblings eco-map ask the following questions:*

Now that you've thought about the people and support you have received, I am going to ask you a little bit more about your experiences as a sibling of someone with cancer.

Can you think of anything else that you think is important for us to know about your support system or what might be helpful to other siblings like you?

#### **References**

## *Article* **"It Is a Whole Different Life from the Life I Used to Live": Assessing Parents' Support Needs in Paediatric Palliative Care**

**Samar M. Aoun 1,2,3,\*, Roswitha Stegmann 3, Renee Deleuil 4, Suzanne Momber 4, Lisa Cuddeford 4, Marianne B. Phillips 4, Maureen E. Lyon 5,6 and Fenella J. Gill 4,7**

**Abstract:** Aims: This feasibility study aimed to systematically identify and address the support needs of parents of children with life-limiting illnesses and to assess whether the systematic approach was acceptable and relevant to parents. Methods: The CSNAT (Paediatric) intervention consisted of two assessment visits with the paediatric palliative care team, 2–8 weeks apart, comprising conversations about sources for support in a tertiary children hospital in Western Australia (2018–2019). Audiorecorded telephone interviews were conducted with parents, and inductive thematic analysis was undertaken. Results: All 28 parents who were involved in the intervention agreed to be interviewed. Five themes summarised their experience: caregiving challenges, perceived gaps and feelings of isolation; the usefulness and practicality of the systematic assessment; emotional responses to selfreflection; feelings of validation and empowerment; and received supports responsive to their needs. Conclusions: Parents appreciated the value of this systematic approach in engaging them in conversations about their needs and solutions to address them. While clinical service support was affirmed by parents, they were left wanting in other areas of practical, psychosocial, and emotional support. Palliative care services need to build stronger partnerships with supportive community networks through compassionate communities volunteer models of care to address the non-clinical needs of these families.

**Keywords:** palliative care; end-of-life care; equity; public health approach; compassionate communities; caregiving; parents; psychosocial support

#### **1. Background**

Paediatric palliative care (PPC) begins with the diagnosis of a child's life-limiting illness and focuses on improving the quality of life for the child and their family [1–4]. PPC addresses physical, psychological, social, and spiritual needs for the child and their family. Families with seriously ill children face multiple and complex challenges that are individual to that family's circumstances. Receiving individualised supportive care from health professionals and the building of effective and trusting relationships have been reported to be essential components of PPC [5], yet there may be a gap between the identified ideal approach and the actual experiences of children and their families [6].

Families' support needs are not always adequately addressed by health professionals. Reported unmet needs include a lack of access to psychological support for parents and

**Citation:** Aoun, S.M.; Stegmann, R.; Deleuil, R.; Momber, S.; Cuddeford, L.; Phillips, M.B.; Lyon, M.E.; Gill, F.J. "It Is a Whole Different Life from the Life I Used to Live": Assessing Parents' Support Needs in Paediatric Palliative Care. *Children* **2022**, *9*, 322. https://doi.org/10.3390/ children9030322

Academic Editor: Linda S. Franck

Received: 6 February 2022 Accepted: 22 February 2022 Published: 1 March 2022

**Publisher's Note:** MDPI stays neutral with regard to jurisdictional claims in published maps and institutional affiliations.

**Copyright:** © 2022 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https:// creativecommons.org/licenses/by/ 4.0/).

siblings of the child with a life-limiting illness [6]. Other unmet needs include a lack of access to home support and to educational supports and resources [7]. Despite the known unmet needs, there have been very few reports of a systematic approach to assess and evaluate parent caregiver support needs [8,9] and no reports of using evidence-based tools to routinely help identify needs or review whether support needs were met [7].

The Carer Support Needs Assessment Tool (CSNAT) is a validated tool designed to identify and address the individual support needs of caregivers of adults with life-limiting conditions [10]. The CSNAT approach involves the creation of an action plan in response to the needs assessment, with ongoing further reviews and follow-ups. The 14-item CSNAT covers two domains that address enabling the caregiver to care as well as direct support for the caregiver. The adapted 16-item paediatric version, CSNAT (Paediatric) [11], was modified from the adult version for use with parent caregivers in the PPC setting.

While the benefits of implementing the assessment of caregiver needs into routine practice has been demonstrated in adult settings, this had not been shown in the paediatric setting. We undertook a pilot study to trial the use of the CSNAT (Paediatric) in an Australian PPC setting [12]. In designing the pilot study, we recognised that, in addition to testing initial CSNAT (Paediatric) implementation outcomes, it was also important to examine the feasibility to understand whether the CSNAT (Paediatric) was appropriate for further testing and whether it can be recommended for implementation into routine care [13].

Feasibility studies can address a number of focus areas and commonly include acceptability from the perspectives of individual recipients and from those involved in implementing the intervention [14,15].

#### **2. Objectives**

The objective of this qualitative study was to assess the acceptability of using the CSNAT (Paediatric), a systematic approach to caregiver needs assessment, from the perspective of parents in a paediatric palliative care setting in Western Australia.

#### **3. Methods**

A steering group guided the development and implementation of the project and included researchers, health professionals (HPs—palliative care consultants and specialist nurses), and two parents with lived experience of receiving paediatric palliative care services. A two-hour training session for HPs was conducted by the researchers and regular 8-weekly team meetings took place for the duration of the project to identify and discuss implementation and data collection issues. The project was approved by the Human Research Ethics Committee of the Child and Adolescent Health Service (RGS0000000772) and La Trobe University Research Ethics Committee. Participants provided written informed consent prior to the start of data collection. The standards for reporting qualitative research were followed [16].

#### *3.1. Setting*

Paediatric palliative care services in Western Australia are co-ordinated from the specialist children's hospital and provide care for children with life-limiting diseases and their families.

#### *3.2. Participants*

Parents of children aged 18 years and younger receiving palliative care who could speak, read, and understand English were invited to participate (2018–2019). Parents of children who were assessed to be within 6–8 weeks of dying were excluded.

Parents participated in semi-structured telephone interviews to obtain feedback regarding their experience using the CSNAT (Paediatric). The brief interview guide was developed and pre-tested with two parents to ensure clarity of the following questions: How easy or difficult was it for you completing the CSNAT (Paediatric)? Can you please describe in what ways completing this assessment approach was helpful in getting the

support you needed? In what ways did the experience of identifying your needs affect what you did yourself? In what ways did you feel that your needs as a caregiver were acknowledged and listened to in a way that was distinct from the needs of [child's name] for whom you provide care? What improvements if any could be made to this assessment approach?

#### *3.3. Description of the Intervention*

The adult version of CSNAT is a validated, evidence-based tool used to systematically identify family caregiver support needs during their relative's end-of-life care [17]. The tool is a caregiver-led, supportive intervention facilitated by the HP. The CSNAT uses a screening format and is structured around 14 broad domains. The domains fall into two distinct groups: those enabling the caregiver to care and those that enable more direct support for the caregiver. It is brief but comprehensive and enables caregivers to identify the domains in which they require further support, which can then be discussed with health professionals through an action plan that needs to be regularly reviewed. The CSNAT (Paediatric) has two additional domains, and its detailed description is reported in [12].

#### *3.4. Recruitment and Data Collection*

Eligible parents with a child known to paediatric palliative care services and attending a clinical appointment were identified by the HP, provided with information about the study, and invited to participate.

The CSNAT (Paediatric) was completed during a scheduled clinical appointment or over the telephone when deemed by the HP to be appropriate. Parents were given the option to complete the CSNAT (Paediatric) on their own or in the presence, and/or with the assistance, of the HP. Parents completed it for a second time 2–8 weeks later, either at a clinical appointment or over the telephone with the HP. Interviews took place approximately 2 weeks after the second assessment had been completed.

#### *3.5. Data Analysis*

Interviews were audio-recorded and transcribed verbatim. Transcripts were imported into NVivo version 12 software for data management (QSR International Pty Ltd., Melbourne, Australia). Inductive thematic analysis was undertaken using the six phases described by Braun and Clarke [18]. Initial coding was carried out independently by two co-authors, one being the interviewer. Transcriptions were read and re-read to identify key words and phrases that were then grouped into categories labelled with codes. To enhance the credibility of the findings, the interviewer participated in the analysis process so that consideration of the nonverbal context was assured. To further ensure the trustworthiness of our findings, transferability is established by our description of the study's setting and participants.

#### **4. Results**

#### *4.1. Participant Characteristics*

In total, 33 parents agreed to be enrolled in the project, and 28 of them completed the intervention: there were 8 parents of children with cancer (code C in quotes) and 20 with non-cancer diagnoses (code NC in quotes). Most parent caregivers were female (93%) and aged from 27 to 55 years old, with a mean of 41.7 years (SD = 8.4). In addition, 82% of parents were married/de facto married and 14% were separated/divorced, and 75% of parents had an Australian background, of which one was of Aboriginal descent. Approximately 80% of parents lived with their child in the metropolitan area. Children's ages ranged from under one to 18 years, with a median of 10 years. The median time from diagnosis was 64 months, the median time since the child first became unwell was 94 months, and the median time interval following referral to palliative care was 23 months. Other characteristics are described in more detail in [12].

Reasons for declining to participate or not completing the study were due to the child rapidly deteriorating or because the parent was feeling overwhelmed.

#### *4.2. Parents' Feedback*

The interviews explored parents' experiences using the CSNAT (Paediatric), its usefulness to them, and the support they received. All participating 28 parents agreed to be interviewed by the research officer over the telephone. The median length of interview was 16 min (range 6–45) with a mean duration of 17.6 min (SD = 7.92).

Five themes were identified: (1) caregiving challenges, gaps, and feelings of isolation elicited by the assessment process, (2) the practicality and usefulness of systematic assessment, (3) the self-reflection evoking emotional responses, (4) the validation and empowerment experienced by being asked about their needs, (5) receiving support responsive to their needs. Table 1 summarises the five themes and their sub-themes.

**Table 1.** Summary of themes and subthemes.

#### **Theme 1.** *Caregiving challenges and gaps in support elicited by the assessment process***.**

Feedback on the assessment process triggered parents to describe their long, challenging journey through the course of the disease and the impact it had on them mentally and the perceived lack of support for their own needs.

*I actually ended up, I had a major breakdown, about 18 months into this. My marriage had already broken down and there were issues at work, a stressful job. So, with this, with [child name] I think once we went through the process of him having the surgery, chemo, radiation, more chemo, when it was sort of getting to the end of that and I realised, "oh gosh" and I recognised the fact that I was falling into a deep hole despite having, you know, medication and gone to a psychologist.* (P07-C)

*So, there is none of that is, none of that care services are free [for parents]. You have to go and do it externally. But sometimes you even wonder if it actually would be helpful if it was done as part of the overall care for the child, because it's really if the parents are not coping the child is not going to get what they need either.* (P27-NC)

As parents, they were putting themselves last *"Because I felt it wouldn't be fair for the focus be on me when my child is so ill"* (P10-C), although for them major life changes happened: *"It's changed my life, it is a whole different life from the life I used to live"* (P19-NC); *"That side of the whole caring role, the parent's role, their life stops"* (P13-NC). Therefore, some

assessment questions did not seem relevant to them when internal or external support was not forthcoming:

*When it comes to the questions where you are getting time for yourself and looking after your own needs etcetera. Obviously, we don't really get to do that because we are parents at the same time.* (P4-NC)

*Like at the moment I have absolutely no time for doing things for myself or socialising or anything. So, you know, there is just nothing. I tried to go out the other night and [child name] had a really bad night and I had to come home. And it was the first time in six months, I think, I am gone out. So, this was just, you know, it was a bit of a disaster. Yeah. It's a bit disappointing.* (P14-NC)

A number of parents of children with complex needs commented that the assessment process would not be useful because of entrenched systematic inadequacies:

*So, I would say, no it hasn't helped. But that has nothing to do with the survey. Because of outside providers I am struggling with. Because my thing is the whole transition* ... *I am still fighting the same fights with the same people. And getting the same frustration.* (P25-NC)

*Sometimes a lack of understanding of the day-to-day needs that we have and it's almost like it's so much red tape you have to get through and it is almost like there is no common sense at the other end.  So, frustration, you know, when it appears black and white to us.* (P28-NC)

The perceived lack of guidance, information, and feelings of isolation with no one to turn to for other types of support compounded negative feelings, *"The focus is much more there on the clinical side, rather than the psychological and social side"* (P11-C):

*Yeah, big gaps in, I am going to say in care. Because the child gets medical support which is amazing. There is absolutely no fault there. but there is no guidance for parents, like, you know, especially in the early days of a diagnosis.* (P27-NC)

*Yeah, parent struggle because there is no one in your current circle to talk to. So, when you often become friends with other families in a similar situation which is great. But those families can't really deal with your problems either because they've got their own. You don't want to burden other family with your worries because they are feeling the same.* (P30-NC)

*You do need lots of hands on, lots of people to come in, you know, such as the OT, the social worker, the doctor, the nurse, whether it's physio required as well. I think we just sort of find out various other things just by chance, sometimes by stumbling across something, or another parent, perhaps in the waiting room talking about a particular thing.* (P10-C)

#### **Theme 2.** *Practicality and usefulness of the systematic assessment***.**

Parents described that the CSNAT (Paediatric) and its structured format comprehensively acknowledged their support needs. They experienced the assessment as straightforward and relevant. The systematic assessment also highlighted issues that might otherwise have been forgotten, especially in a stressful situation. Parents expressed this with various comments, such as:

*This really ticks all the boxes that need to be discussed. Because, obviously sometimes you can only think of one thing or the first thing that is most pertinent to you, you and your family, and the person you are caring for and other things get forgotten*. (P23-NC)

The CSNAT (Paediatric) improved communication between parent and the palliative care service providers or team. Parents described that the assessment opened a discussion between them and the HPs and enabled the parents to identify their needs and to articulate them.

*It was helpful because it did open up discussions with the team and highlighted some of the areas that I have been struggling with.* (P14-NC)

Parents acknowledged the impact of their child's illness and the parental caring role on the family unit, as well as the necessary solutions.

*It highlighted the fact that I probably need to look at him going into respite care maybe once a month. And that is something I always not wanted to do up until now. But it probably highlighted the fact that for my other children it's a necessity, so they can have some breathing room as well* ... *So, it helped me to recognise that is something that we need to do.* (P14-NC)

#### **Theme 3.** *Emotional responses to self-reflection***.**

#### - *Subtheme 3.1: Prompted self-reflection in a positive way*

The systematic assessment prompted the parent to pause and reflect; *"it made me stop and think"* (P25-NC) was a common experience. The questions offered the opportunity for a pause in the daily routine and for them to re-assess the caring situation. Parents commented positively on how this experience affected them.

*I think it was helpful because it just made me think more about [child name]'s needs, my needs and if they are being addressed and how they are being addressed. So, sometimes we just don't stop to think about all these questions or issues that are raised through this program. And I think it's a good process. So especially beneficial for me as a caregiver and a parent.* (P22-NC)

#### - *Subtheme 3.2: Elicited feelings of confrontation*

For other parents, this triggered a sense of confrontation, and they admitted that *"it's hard and when you're reading the questions you have to try and think honestly about how you feel inside"* (P09-C). The parent explained that the avoidance of this confrontation represented a way of coping with the necessary daily life tasks.

*Well, it's just the whole thing. When you read the questions, it's not like you are trying to blank out the illness, it's just, just hard dealing with your feelings and worries, I suppose. I am no different to everybody else, you try to bury your feelings and worries because you still have to go on with other things.* (P09-C)

Reflecting on their needs enabled the parents to take a different perspective, which was expressed as *"just made you think outside the box"* (P12-NC). This process helped them to analyse and understand their thoughts and feelings.

*the questions open up different pathways for your thoughts as well. I think. So, you know it helps you to sort of break down your own thoughts and to, you know, go more into depths of why you are thinking the way you are thinking.* (P08-C)

One parent felt that the process of reflection helped in her search for meaning in the serious illness of her child and spoke of positive feelings that emerged despite the sadness.

*At the end of the day I actually realised that even though I am in that situation I actually still feel blessed and that there is actually a lot more that's positive that's coming out of this. And it's not all just bad and sad and so and so. No, I really think it was good for me, personally for me, myself.* (P02-C)

Likewise, participating in the research was seen as an opportunity to help other parents in a similar situation and comforted the parents that their situation and care experience contributed to something meaningful.

*I see that as a positive in providing information to you. So, that hopefully that feeds back and it provides help to others. And that's my ultimate goal. I know in the end, that, we might not be able to help [child name]. But, you know, the end goal for me is, if I can, if something good comes out of this it is going to be that we've helped inform others about our situation and about what areas are lacking and what can be improved.* (P08-C)

#### **Theme 4.** *Validation and empowerment***.**

Parents appreciated the added comprehensiveness of the assessment and found that it allowed for a different level of acknowledgement and validation of their needs, including their caregiver role:

*This is on a completely different level. And this is about acknowledging and diving deeper and getting me to do some self-reflection. And, and knowing and acknowledging the issues that I've got. I think it's been invaluable; I think it is fantastic and very different.* (P24-NC)

This systematic assessment also encouraged the parents to develop their own strategies, and they adhered to the mutual action plan they had discussed during the assessment.

*But I did start one thing that we discussed on there, which was the meditation. So, I definitely started doing that after the assessment.* (P21-NC)

*I haven't seen this psychologist for three months. And I thought I didn't need to. And then when I went and saw her, I realised how much I had to talk about. So, I can't leave it that long I have to definitely check in with her every now and again.* (P21-NC)

The acknowledgment received during the conversation with the HPs not only made parents' needs visible, but also legitimised their own needs. Parents' self-confidence to ask for help increased as they described that they felt reassured to ask, for instance, *"for more respite under the new NDIS [National Disability Insurance Scheme] rollover thing. I have asked for that. So, I put my needs forward with them. And asked for more respite"* (P05-NC).

In addition to taking time for themselves, parents also intended to take care of their own medical needs: *"Just made me realise that some parts like her care I might need to find some support with, just like with time for myself and my medical needs and things like that"* (P31-NC).

Some parents mentioned how they engaged in advocacy and that this had been reinforced by the CSNAT (Paediatric) experience. They turned to politicians to raise awareness of their support needs and to increase support for themselves and other families caring for a child with a life-limiting illness.

*I think we just try and look at other avenues to see if we could get things ourselves to speed up the process. I've even tried to ring members of parliament and the radio station just to, yeah things like that, just to bring to light the issues what we have. Because sometimes I think if we don't speak people don't understand.* (P28-NC)

#### **Theme 5.** *Received supports responsive to their needs***.**

The information and assistance they received from the HPs alleviated parents' concerns and created a sense of empowerment. They felt better prepared:

*It's sort of prepared me for what's going to come and had a plan in place.* (P07-C)

*having talked to [HP name] and the doctor we know we have plans in action if we need it. So, I feel a little bit more comfort with that.* (P11-C)

*I guess by having gone through the process and being able to talk to [HP name] about some of my concerns around transition allowed me to have a conversation about what is a clear plan. Who can I turn to in between the process? It helped me to put some steps into place, I guess. Just through having those conversations.* (P15-NC)

The improved confidence increased parents' sense of coping and managing *"I actually do feel stronger about most of the things that I thought I would fail in"* (P02-C) and *"It made me realise that I do a lot and I am taking on a lot and I am managing it quite well"* (P07-C).

The HPs answered parents' questions, liaised with other services, provided advocacy, and arranged required referrals according to what was identified and discussed in the CSNAT (Paediatric) conversation and action plan. Parents found the provided support to be very helpful:

*She has been able to answer a lot of the questions I had and find the answers that I needed, by going through that form. So, it's been very helpful. And I am very thankful that you've done it.* (P11-C)

*Obviously, [HP name] and [HP name], anything that I had issues with or that is, you know, I needed help through what we've worked out from the questionnaire that they have been able to help, which has been amazing.* (P23-NC)

The following comment seems to sum it all by reflecting on the uniqueness of this approach, which is truly centred on caregivers:

*Well, I just want to say, thank you very much, really. Because I think that's something that is, all the year that we had, so many things, we got bombarded with people who want to do surveys and from the health department or, you know, I get calls all the time. But this one seems to be the one right for us and that made it, again, reassuring that somebody is out there trying to understand, or trying to find out where we are coming from.* (P08-C)

#### **5. Discussion**

This study examined the feasibility of using the CSNAT (Paediatric) in the paediatric palliative care setting from the perspectives of parent caregivers of children with lifelimiting illnesses. All study participants agreed to be interviewed, and it is apparent that parents appreciated the value of this systematic approach in engaging them in conversations about their needs, priorities, and solutions. They felt it gave them the opportunity to consider and express needs and identify what is important to them in a timely manner where possible. Parents welcomed the focus on their individual situation and hoped that this research would increase the awareness of service providers and policy makers to create a positive change.

The use of the CSNAT (Paediatric) facilitated the identification of the parents' support needs, where over 60% reported the following needs: having time for yourself in the day (direct support for the caregiver); practical help in the home (direct support for the caregiver); knowing what to expect in the future when caring for your child (enabling support for patient); financial, legal, or work issues (direct support for the caregiver); knowing who to contact if you are concerned about your child (enabling support for patient); looking after your own health (direct support for caregiver) [12]. It is worth noting that parents needing direct support for themselves featured high on this list, as is supported by the quotes in this article.

#### *5.1. Gaps in Supportive Networks*

While clinical services and the support they can offer was affirmed by parents, these parents were left wanting in other areas of practical, psychosocial, and emotional support. This is echoed by a recent independent review of adult palliative care services in WA that used a cross-sectional consumer survey of quality indicators to respond to the six priorities of the WA End of Life for developing and improving palliative care services across WA. Of the six priorities, quality indicators for Priority Four (families and carers are supported) lagged behind the others [19]. Family carers reported not being well supported before and after bereavement by palliative care services. Emotional support and the ability to discuss worries and fears were not adequate, and 40% felt that they did not receive as much support as they wanted from palliative care services during the patient's illness, and 50% did not after the patient's death [20]. By way of contrast, responses to Priority Six (the community is aware and able to care) gave high ratings to the care provided by informal networks. Over 90% of respondents relied on the community (family/friends/neighbours/community organisations) to support them before and after bereavement and reported that this informal support was helpful in attending to practical, social, emotional, and spiritual support needs [20].

Another key finding of the independent review was the lower standard of care for noncancer conditions across all six priorities, dubbed as one of the 'loser' groups in palliative care service delivery, thus highlighting the inequity in care [20]. This finding was also echoed in this paediatric study, where there were higher levels of unmet needs for the non-cancer group, as reported in our first article from this study [12].

While the independent review focused on adult palliative care, it is worth replicating the consumer survey on quality indicators for the paediatric population. In fact, the more recent Western Australian Paediatric Strategy for End-of-Life and Palliative Care (2021–2028) has reiterated the need to consolidate the two priorities related to family carers and the community for the paediatric population [21]. Building blocks that will help to achieve Priority Four include the use of standardised assessment tools, ensuring the child's family has equitable access to support and respite, and services are urged to look at ways to improve opportunities for consumers to support consumers. Priority Six has building blocks around engaging the community to care, including volunteer models and the development of compassionate communities.

Hence, where our study group was left wanting is within the realm of support from their naturally occurring social informal networks or circles of care [22]. If these informal networks or the inner circles of care are not operational or activated, as expressed in parents' quotes, then compassionate communities models of end-of-life care need to be bolstered (as recommended by the Paediatric Strategy). This can be achieved by training volunteers from the community to seek the practical and social support the family needs to care for the dying from any age group [23]. Volunteers help families tap into the outer circles of care in their community to enhance their social networks in a sustainable way. A local example is the Compassionate Connectors program, a partnership between the South West Compassionate Communities Network and the WA Country Health Services, where the program has been translated into routine practice by the health service: this is a case of a successful partnership between formal and informal networks [23].

Compassionate communities, as part of the public health approach to end-of-life care, offer the possibility of solving the inequity in the difference in the provision of care by enhancing the naturally occurring supportive networks surrounding the patient and family and through palliative care services building stronger partnerships with supportive networks to transform end-of-life care at home [24,25]. Experts in the field have lamented the reality that, *"although palliative care looks beyond the patient to the family, it rarely looks beyond the family to the community or sees the whole person as an individual-in-community"* [26] (p. 133).

#### *5.2. Limitations*

We acknowledge the lack of diversity in the study sample where parents were primarily Caucasian women from a metropolitan area.

#### **6. Conclusions**

Assessing needs is a first step in acknowledging areas where parents need support. Formal and informal networks need to work together to provide a platform of psychosocial, emotional, and existential support that is sustainable in people's own communities. Given that equity in end-of-life care provision is a goal of government policy [27], inequity arising from disease type should be addressed within the health system. Inequity arising from inadequate social support must be addressed by local communities. Both aspects are taken into account by a public health approach to palliative and end-of-life care. Hence, the distinctive focus of this public health approach is that it views the community as an equal partner in the long and complex task of providing quality healthcare at the end of life [20].

**Author Contributions:** Conceptualization, S.M.A., F.J.G., M.B.P., L.C., R.D., S.M.; methodology, S.M.A., F.J.G., M.B.P., L.C.; validation, S.M.A., R.S., F.J.G., M.B.P., L.C., R.D., S.M.; formal analysis, S.M.A., R.S., F.J.G., M.B.P., L.C., R.D., S.M., M.E.L.; investigation, S.M.A., R.S., F.J.G., M.B.P., L.C., R.D., S.M., M.E.L.; resources, S.M.A., R.S., F.J.G., M.B.P., L.C., R.D., S.M.; data curation, S.M.A., R.S., F.J.G., M.B.P., L.C., R.D., S.M.; writing—original draft preparation, S.M.A., R.S., F.J.G.; writing review and editing, S.M.A., R.S., F.J.G., M.B.P., L.C., R.D., S.M., M.E.L.; supervision, S.M.A.; project administration, S.M.A., F.J.G.; funding acquisition, S.M.A. All authors have read and agreed to the published version of the manuscript.

**Funding:** The authors disclosed receipt of financial support for the research from the Health Department of Western Australia through the WA Cancer and Palliative Care Network and supported in-kind by La Trobe University, Perth Children's Hospital, and Perron Institute.

**Institutional Review Board Statement:** The study was conducted according to the guidelines of the Declaration of Helsinki and approved by the Human Research Ethics Committee of the Child and Adolescent Health Service (RGS0000000772) and La Trobe University Research Ethics Committee.

**Informed Consent Statement:** Participants provided written informed consent prior to the start of data collection.

**Data Availability Statement:** Ethical approval precludes the data being used for another purpose or being provided to researchers who have not signed the appropriate confidentiality agreement. Specifically, the ethical approval specifies that all results are in aggregate form to maintain confidentiality and privacy and precludes individual level data being made publicly available. All aggregate data for this study are freely available and included in the paper. Interested and qualified researchers may send requests for additional data to Samar Aoun at<EMAIL_ADDRESS>
**Acknowledgments:** We gratefully acknowledge the participants (parents and health professionals) for contributing to this research project and the consumer representatives for their advice. The CSNAT is a copyrighted tool which requires a license for its use. For details about accessing the CSNAT and the licensing process, please visit http://csnat.org, accessed on 5 February 2022.

**Conflicts of Interest:** The authors declare no conflict of interest.

#### **References**

## *Article* **Family-Centered Advance Care Planning: What Matters Most for Parents of Children with Rare Diseases**

**Karen Fratantoni 1,2, Jessica Livingston 1, Sandra E. Schellinger 3, Samar M. Aoun 4,5,6 and Maureen E. Lyon 1,7,\***

**Abstract:** Few studies have described the goals and wishes of parents caring for their children with rare diseases, specifically when children are unable to communicate their preferences directly. The purpose of this study was to describe the parent's understanding of their child's illness, goals of care, and what mattered most to their child from the parent's perspective. Six families completed a feasibility study of the FAmily CEntered (FACE)-Rare pACP intervention. Qualitative content analysis was performed on transcripts of videotaped responses to the Respecting Choices Next Steps pACP Conversation facilitated conversation guide about the goals of care. Codes were grouped into themes, with direct participant quotations representing the themes. Five themes emerged: getting out and moving freely; feeling included and engaged; managing symptoms and disease burden; coordinating care among many care team members; and managing today and planning for the future. In the context of pACP, families reported that what mattered most to their children included the freedom of movement and human connection and engagement, while parents strived to be effective caregivers and advocates for their child with a rare and severely disabling disease.

**Keywords:** rare disease; advance care planning; decision-making; family caregiver; palliative care; psychosocial care; communication; pediatric

#### **1. Introduction**

Children with rare, life-limiting diseases face challenges to their physical and psychosocial health. A child diagnosed with a rare disease may require total life-long care, and a parent often provides that care for the duration of their child's life. If a child cannot communicate, a parent must also try to determine their desires. There is no roadmap for this journey, leaving parents often feeling stressed or isolated [1]. Even though the parent is usually the most involved in the child's care, there is a lack of literature on a parent's experience caring for their child with a rare disease [2]. Few studies have described the goals and wishes of parents caring for their children with rare diseases, especially when children are unable to communicate their preferences directly.

There is a need for more research regarding parents' experiences and desires when caring for children with rare diseases. In addition, specific information about their children's medical conditions is often lacking, making it difficult to manage symptoms [3,4]. Often, little is known about future care needs [5–7]. General knowledge about the disease and its consequences is lacking [7], which may result in inadequate care [8]. There is also a gap in research focused on parents' experience with the healthcare system [9], although recent

**Citation:** Fratantoni, K.; Livingston, J.; Schellinger, S.E.; Aoun, S.M.; Lyon, M.E. Family-Centered Advance Care Planning: What Matters Most for Parents of Children with Rare Diseases. *Children* **2022**, *9*, 445. https://doi.org/10.3390/ children9030445

Academic Editor: María Fernández-Hawrylak

Received: 22 February 2022 Accepted: 18 March 2022 Published: 21 March 2022

**Publisher's Note:** MDPI stays neutral with regard to jurisdictional claims in published maps and institutional affiliations.

**Copyright:** © 2022 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https:// creativecommons.org/licenses/by/ 4.0/).

studies focus on pathways to psychosocial care at a system level [10,11]. Overall, parents of children with rare diseases report that there is a lack of coordination of care [2]. In a survey administered toward 46 families of children with rare diseases in Australia, parents felt that the care of their children could be improved if there was better coordination of care and interaction between multiple providers [3].

Pediatric Advance Care Planning (pACP) is a process that facilitates discussions with parents and potentially with children about future medical treatment, care, and decisions if the child's condition becomes life-threatening. It also helps families prepare for situations that may occur, and it can improve the family's understanding of the child's prognosis. Pediatric ACP is a core component of palliative care for children living with chronic conditions [12], and it should be offered to all parents of and children living with rare diseases.

The FACE-Rare intervention integrated two evidence-based processes: the Carer Support Needs Assessment Tool (CSNAT-Paediatric) Approach [13,14] and Respecting Choices® Next Steps pACP conversation [15]. Quantitative outcomes of both aims were reported previously [16].

The aim of this article is to highlight the qualitative findings from the Respecting Choices® Next Steps pACP session during which parents engaged in facilitated conversations about their understanding of their child's illness, goals for their child, and what mattered to their children from parental perspectives.

#### **2. Methods**

From October 2017 to January 2018, we recruited and enrolled parents of nonverbal children with rare diseases to participate in a feasibility study of a 4-session needs assessment and pACP intervention. Complete methodological details of the FACE-Rare intervention have been previously described [16]. Parents who were ≥18 years old, had a child with a rare disease, and spoke English were invited to participate. Participants' children were between the ages of 1 and 24 years old, had a diagnosed rare disease, were not admitted to an intensive care unit during the study, were unable to participate in medical decision making due to age or disability, were not in foster care, and did not have an active Do Not Resuscitate Order. Children with the primary diagnoses of autism, rare cancers, HIV, sickle cell, cystic fibrosis, down syndrome, lupus, and muscular dystrophy were excluded, as previously published studies have investigated their disease-specific needs. Written informed consent was obtained from the parents, along with a signed waiver of assent for their child. The study was approved by the Institutional Review Board of Children's National Hospital in Washington, DC, which is a quaternary hospital and the study site.

#### *2.1. Interviews*

Table 1 illustrates the FACE-Rare study protocol timeline, which provides the context for the study results. The 4-session intervention was administered in person at the study site or by telemedicine. This study only reports on the qualitative data that emerged from Sessions 3 and 4, which consisted of structured goals of care conversation using the Respecting Choices Next Steps pACP conversation guide and a pACP document [15]. The structured conversation guide and pediatric Advance Care Plan are proprietary and are made available upon the completion of training and certification by Respecting Choices, a Division of C-TAC Innovations [15].

**Table 1.** FACE-Rare Study Protocol Timeline.

Respecting Choices is designed to be culturally sensitive to persons with disabilities [17]. This session was facilitated by two trainers from Respecting Choices, including one of the authors (SS). Interviews were videotaped, transcribed verbatim, and deidentified (using pseudonyms). A psychology graduate student/research volunteer transcribed and deidentified the videotapes. Transcriptions were then verified by the last author (ML). This study reports on the data that emerged from Sessions 3 and 4, which comprised responses to the Respecting Choices Next Steps pACP conversation guide and pACP care plan.

#### *2.2. Data Analysis*

The authors (K.F., J.L., and S.E.S.) performed conventional content analysis [18] on the transcribed Respecting Choices conversations using an iterative process. Four iterations occurred over a 4-month period from 4 March 2019 to 2 June 2019. For researcher characteristics, please refer to Biographies at the end of this article.

J.L. and K.F. individually read the transcripts and assigned codes to transcripts. Codes were then jointly reviewed with S.E.S. and grouped into code families. Similar code families were combined into themes. Disagreements were resolved by consensus during monthly conference calls (K.F., J.L., S.E.S., and M.E.L.). Representative quotations illustrate key themes. Data were managed using hardcopy files. We used the Standards for Reporting Qualitative Research (SRQR) to guide our report [19].

#### **3. Results**

Eight parents were approached: One declined participation, and one was lost to follow-up after Session 1. Six parents completed Sessions 1, 2, 3, and 4. All participants were female, mean age 40 years (standard deviation = 7.7 years), 57% Caucasian, 29% black or African American, and 14% biracial. Two fathers participated with the mothers in Session 4 for the creation of an advanced care plan. The children ranged in age from 2 to 12 years with a mean age of 7 years. Five children had seizure related comorbidities, and all were technology dependent. Five used a wheelchair and feeding tube/pump, and one had a home ventilator.

#### **4. Key Themes**

Five themes emerged throughout the structured interview, which not only followed specific questions. See Tables 2–6. The five themes about what matters most for parents of children with rare diseases were as follows: (1) getting out and moving freely; (2) feeling included and engaged; (3) managing symptoms and disease burden; (4) optimizing coordinated care among many team members; and (5) planning for the future. See Tables 2–6 for subthemes and illustrative quotations.

**Table 2.** Theme 1 Getting Out and Moving Freely.

 He really likes it if he's in his wheelchair so that he can explore the new place.

But not too loud."

*Disabilities can impede full inclusion*

P5: "Hope ... a world that is more inclusive. When leaving the house, because we do not have a child that is ambulatory, [it's] hard to find a place that we can change a diaper because changing tables are only for infants and at 100 pounds, I can't put her up on the changing table and yet if I put her on the floor, I can't get her up from the floor. So, we're limited between or changing her in the back seat of the car, and she's an 11-year-old little girl, and so it limits her being included in a world the way we want her to be, and from us being in the world that we want."

**Table 3.** Theme 2 Feeling Included and Engaged.

#### **Theme 2 Illustrative Quotations**

*Interact with others* P1: "She needs to be able to have some form of communication. She needs to have her sisters." P5: " . . . being able to be with her peers but making accommodations to make it happen." *Engaged in enjoyable activities* P1: "Being engaged.  She likes the swiping aspect. She can swipe and kind of navigate her way around." P6: "Being held by someone and a massage"

**Table 4.** Theme 3 Managing Symptoms and Disease Burden.

#### **Theme 3 Illustrative Quotations**

*Limit unnecessary interactions with the healthcare system*

P1: "Stay away from some of the orthopedic surgeries that she needs. I'm trying to hold off as long as I can."

P1: "Stays healthy . . . I need her to not have the cold or flu because things kind of snowball . . . keep her healthy and out of the healthcare system."

*Limit the progression of disease*

P3: "Eliminate the chronic eye issue."

P7: "Worry about there being more issues ... he had an ASD [Atrial Septal Defect] that was repaired a few years ago. But he started getting followed by a cardio myopathy clinic so, I get worried that something may turn up . . . like a new condition."

*Seizure control*

P1: "So, my ultimate hope is much better seizure management ... when we get better seizure management, then we're better able to maximize speech therapy and receptive expression."

P3: "I'd love it if we'd reduce a lot of his seizure medication one day. Yeah, they sedate him . . . we've seen alertness change with medication, so it sure would be nice if we could reduce it someday."

*Optimize Quality of Life*

P1: " . . . be as comfortable as possible. Her comfort would be of utmost concern"

P7: "Address those baseline needs ... I think he has a lot of little things where, very seemingly little things, that bother him and so he's not comfortable. And that affects his ability to kind of grow and progress and learn."

*Minimize medications*

P3: "Minimize how many medications he's on and how we give them to him, so it maximizes his abilities and alertness."

*Self-Injuries*

P7: "He's banging his head on things. We're worried about getting him too strong. You know, getting too big and too strong for me to be able to take care of everything . . . unintentional injuries, him injuring himself, something that will injure him."

**Table 5.** Theme 4 Optimizing Coordinated Care among Many Team Members.

#### **Theme 4 Illustrative Quotations**

*Optimal communication and coordination among team members*

P1: "That there's better communication among our healthcare providers . . . nice if doctors got together once or twice a month to discuss . . . so they can get a good snapshot of where you are."

#### **Table 5.** *Cont.*

#### **Theme 4 Illustrative Quotations**

P6: "And this idea of, we'll do the procedure, and we'll work out the home care and the nursing after ... that's not going to work for us because there's not going to be a place for us after. And those are the types of things that every thought, thoughts, you really have to forecast, way down the road. And that's a lot of the concern, who's going to do that? Who's going to be willing to own that and am I going to get team members that are really going to work with us?"

*Preparing for transition*

P3: "I guess looking ahead with [his] age, parents like us have a discussion with the transition to adult care so I brought that up to his physicians periodically, and they assured me, don't worry about him, especially with a rare disease, you know. He's, he's not going to be sent away and he's at a children's hospital, I mean I know but he's not your typical patient that can go anywhere. A huge concern for when we are away and we've had to seek medical care, we seek help, other hospitals are just not equipped for him and we've had doctors tell us, we're just not equipped to helping [him]. And so, that just makes your heart sink and you always want him to be in the hands of someone who knows him best and have already done such good already in managing him."

**Table 6.** Theme 5 Planning for the Future.

#### **Theme 5 Illustrative Quotations**

#### *Hopes for the future*

P2: "So, I want him to thrive as much as he can, for as long as he can, but I don't want him to hang on just for me or for my husband or parents. I want his life to be his life and that for him, to leave hopefully or a little more peacefully."

P6: "I hope that we're able to try to be able to give her the best possible life possible and that she knows that we do our best to make that happen and that she feels loved and she feels acknowledged as an individual and that she knows, despite all of it, that we think she is a wonderful, miraculous little girl. And that she lives the best life and that we're able to support that."

P7: " ... stability and being able to be happy and enjoy each other ... not stressed out worrying about who will take care of him or working or money or things like that."

*Fears*

P5: "If that day comes ... . I kind of like want to be there and to hold her hand and to let her know it's okay. I don't want [her] to go through that alone and I think for me, that's my biggest fear, is her having to go through that alone and suffer."

P7: "When I'm not here anymore, who's going to take care of him?"

*Uncertainty of what might happen next-that the family may not be able to handle*

P2: "Every ED visit is more like-fear oriented . . . I fear every hospitalization is one step closer to something horrible and disease progressing out of what we can manage right now."

P3: " ... we have everything so well managed and so there's always there's that little concern with, what if a new challenge arises and the current method of treatment doesn't meet the needs, can't take care of the challenges. So, whole team is very proactive with keeping that under control. Just you know, his medical needs never outweigh the available treatment."

#### *4.1. Theme 1: Getting out and Moving Freely*

As illustrated in Table 2, parents emphasized the importance of encouraging independence for their children and providing opportunities to participate in activities. Physical exploration, optimizing mobility, and offering new experiences were all essential while simultaneously ensuring their safety. Creating and being a part of a supportive and inclusive environment were necessary to accomplish this goal, yet families were often reminded of the barriers to full inclusion.

#### *4.2. Theme 2: Feeling Included and Engaged*

As illustrated in Table 3, parents want their children to feel connected to the outside world beyond their diagnosis and medical care. They desire meaningful interaction with others and opportunities to engage in enjoyable activities.

#### *4.3. Theme 3: Managing Symptoms and Disease Burden*

As illustrated in Table 4, parents wanted to limit unnecessary interactions with the healthcare system, recognizing that their children with rare diseases have regular and frequent medical appointments, hospitalizations, and emergency department visits. Slowing the progression of disease and controlling symptoms are integral to a good quality of life. Many families mentioned the desire for seizure control, as seizures were disruptive to daily life and occupy attention that could be directed to other impactful therapies and interventions.

#### *4.4. Theme 4: Optimizing Coordinated Care among Many Care Team Members*

As illustrated in Table 5, parents wanted to optimize good care team communication means better care for children with rare diseases, especially at times of transition.

#### *4.5. Theme 5: Managing Today and Planning for the Future*

As illustrated in Table 6, parents expressed their hopes for the future: for their children to feel complete, whole, not a burden, thrive, and experience life to its fullest. Parents shared their fears about what the future may bring and their uncertainties about their ability to handle the challenges.

#### **5. Discussion**

To the best of our knowledge, this is the first study to examine parents' goals, values, and hopes for their child with a rare disease in the context of a structured familycentered pACP intervention. After completing the goals of care conversations with a trained/certified facilitator, all parents returned to complete an advanced care plan document [16]. Five themes emerged. With respect to living well, parents reported that their severely disabled children enjoyed getting out and moving freely, even though all but one of the children were wheelchair bound. Moreover, interactions with others so that they could feel included and engaged were also important to their children. Parents highlighted their caregiving role in managing symptoms and disease burden, coordinating care among many team members, and coping one day at a time by balancing their hopes and fears for their medically fragile child. Seizures were a common symptom associated with rare diseases [20], which caused significant distress.

These findings are consistent with a scoping review that identified five themes with respect to the support needs of parent caregivers of children with a life-limiting illness [21]. The themes identified were support for communication; choice; information; practical information; and social, psychological, emotional, and physical needs. Unmet needs included support for siblings; respite care; out-of-hours care; and psychological, home, and educational support [21]. A focus group with adults living with a rare diagnosis also highlighted the value of participation in society [7].

Our family-centered approach, which included parents in the development phase of the protocol, is consistent with calls for a family-centered framework relative to pACP for children with medical complexities [22]. There is also agreement that pACP should be an ongoing process from the time of diagnosis for seriously ill children [23–25], which is especially important for children living with prognostic uncertainty. In our clinical experience, long-term relationships and trust develop between patients and families and their providers. As the medical condition progresses, a disconnect may develop between a parents' beliefs, values, hopes, goals, and their child's quality of life and developing a holistic treatment plan. The FACE-Rare intervention did not require the pACP facilitator to be well-known to the parents, as has been recently recommended [22,25]. Our findings are consistent with our previous trials of a three-session FACE pACP model, which demonstrated that the initial pACP conversations about the goals of care with parents of seriously ill children and the completion of an initial advance care plan can be successfully conducted by trained/certified facilitators that are not known to the parents, who are referred by their treatment team [16,26,27]. A summary of these conversations and the documents were then emailed by the facilitator to the treating clinician, and the facilitator places the documents in the medical record, laying the groundwork for future pACP conversations with their health care provider. Thus, FACE-Rare demonstrated families' willingness to engage in goals of care conversations with a trained/certified nurse facilitator who was unknown to them.

The FACE-Rare intervention is consistent with findings from focus groups with parents of children with medical complexities and their health care providers (HCPs) [22]. HCPs and parents expressed the desire that the patient and family be at the center of pACP discussions. HCPs noted the importance of taking time to recognize, understand, and support diversity and individuality between families. Parents also explained that the best pACP conversations were the ones in which they felt involved, respected, and accepted, which is similar to our findings. Parents identified topics that they felt should be included in pACP discussions, which were included in the FACE-Rare model: (1) quality of life, (2) beliefs and values, and (3) hopes and goals. In our study, the parents noted that their child's quality of life was often underestimated by HCPs, thus highlighting the importance of asking parents about their child's quality of life at baseline rather than making inferences based on their clinical status when admitted to the hospital, which is consistent with findings from a focus group with parents of children with medical complexities [22]. The focus groups with parents and HCP also thought a family's values and belief system was foundational to pACP discussions, allowing HCP to better tailor care to each individual family [22], as was accomplished in the FACE-Rare intervention. Focus group parents and HCP also indicated that pACP discussions should include conversations surrounding their hopes and goals for their child because this process provided opportunities to collaboratively work toward and/or reframe hopes and goals [22]. Thus, study findings highlight the importance of incorporating parents' hopes and values for what it means for their child to live well, prior to the completion of an advanced care plan. Understanding a parent's focus on what is most important is possible with the FACE-Rare intervention. This approach may improve a parents' ability to advocate on their child's behalf and assist care team members to provide a person-centered care approach that matches the goals and values, with the needed care and resources as their child's condition changes.

#### **6. Limitations**

Research participants were selected to beta test the study protocol and to gain preliminary information on feasibility. Thus, we did not use sampling saturation as a criterion for our sampling strategy. This means that other themes might have emerged if we had continued sampling, which may limit generalizability. The cohort was small and from a single site. A larger pilot trial is ongoing to test the initial efficacy of FACE-Rare with 30 parents of children with ultra-rare diseases who are unable to participate in medical decision making [28].

The sex of the parent may have introduced bias. The primary caregiver in the home may depend on multiple factors, including parental employment; medical insurance; and the size, gender, and weight of the child. Clinically, many fathers have reported discomfort when caring for their female children with rare diseases once their child reach puberty age.

Moreover, information that was not reported is also important. No parents reported service providers' negative responses as a barrier for accessing services or assistance for their children, as has been reported in two small qualitative studies [5,6].

#### **7. Conclusions**

This study begins to close a gap in our knowledge [29] of parents' goals of care for their children living with a serious illness who, in aggregate, constitute a significant proportion of pediatric inpatients with life-limiting conditions in tertiary and quaternary pediatric hospitals [30]. Children with rare diseases are part of a heterogeneous group and are often excluded from research [31], thereby creating a health disparities. Collecting qualitative data on patient and family member goals and wishes is a pivotal part of quality care. With information about the goals and wishes of the patients and families being discussed, accurate and appropriate recommendations for palliative and end-of-life care from the care team can be provided. Ongoing research will determine if the FACE-Rare pACP process of decision making for parents [27] adds benefits to clinical care and family well-being [32], as has been true with the FACE pACP model with adolescents with cancer and HIV and their families [26,27].

**Author Contributions:** Conceptualization, K.F., J.L., S.M.A. and M.E.L.; methodology, K.F., S.E.S. and M.E.L.; analysis, K.F., J.L., S.E.S. and M.E.L.; writing—original draft preparation, K.F. and J.L.; writing—review and editing, K.F., J.L., S.E.S., S.M.A. and M.E.L. All authors have read and agreed to the published version of the manuscript.

**Funding:** This research received no external funding.

**Institutional Review Board Statement:** This study was conducted according to the guidelines of the Declaration of Helsinki and was approved by the Institutional Review Board (IRBear #8808 on 24 October 2017 of Children's National Hospital in Washington, DC, USA).

**Informed Consent Statement:** Written informed consent was obtained from the parents. A waiver of assent was obtained for their children.

**Data Availability Statement:** Only transcribed data can be obtained, as the original source videotapes contain personal information. Contact the last author at<EMAIL_ADDRESS>for additional information.

**Acknowledgments:** We are most grateful to all participants for taking the time to share their experiences and perceptions. We thank Christopher Lin, BA, the doctoral student in psychology who transcribed the videotapes.

**Conflicts of Interest:** The authors have no financial relationships relevant to this article to disclose.

#### **References**

## *Article* **The Needs of Adolescents and Young Adults with Chronic Illness: Results of a Quality Improvement Survey**

**Taryn Allen, Stephanie Reda, Staci Martin, Phoebe Long, Alexis Franklin, Sima Zadeh Bedoya, Lori Wiener and Pamela L. Wolters \***

> Pediatric Oncology Branch, National Cancer Institute, Bethesda, MD 20892, USA<EMAIL_ADDRESS>(T.A<EMAIL_ADDRESS>(S.R<EMAIL_ADDRESS>(S.M<EMAIL_ADDRESS>(P.L<EMAIL_ADDRESS>(A.F<EMAIL_ADDRESS>(S.Z.B<EMAIL_ADDRESS>(L.W.) **\*** Correspondence<EMAIL_ADDRESS>
**Abstract:** Adolescent and young adults (AYAs) with chronic illnesses cope with complex issues that require unique psychological support and healthcare services to reduce psychosocial difficulties, improve disease management, and facilitate positive transitions to adult care. Engaging patients and caregivers can help providers understand the specific needs of this population and identify the perceived areas of support. The purpose of this quality improvement initiative is to assess the needs of AYAs with chronic medical conditions at a large government research hospital. Eightynine AYA patients (age = 23.5 years; range 13–34) with neurofibromatosis type 1, cancer, primary immunodeficiencies, or sickle cell disease, and a sample of caregivers (*n* = 37, age = 52 years; range: 41–65), completed an anonymized survey that assessed their preferences for a wide range of informational and service-related needs. The results indicate an overwhelming desire for information about general health and wellbeing and disease-specific medical knowledge. The most endorsed item was the need for more information about an individual's medical condition (72%), which was a primary concern across disease, racial, and gender groups. Demographic and disease-specific needs were also identified. Thus, providing information to AYA patients and caregivers is a critical and largely unmet component of care, which requires the development and implementation of targeted educational and psychosocial interventions.

**Keywords:** adolescents and young adults; healthcare needs; chronic illness; AYA transition

#### **1. Introduction**

Large numbers of adolescents and young adults (AYAs) live with chronic medical conditions [1]. The vast majority (90%) of AYAs with chronic illness will survive into older adulthood [2,3] and this percentage will continue to increase, given the continued medical advances and improvements in survival rates [4,5]. Unfortunately, relatively less attention has been focused on AYAs with chronic health conditions, compared to children and adults; thus, the needs of patients within this age band may be less well understood and, as a result, less well addressed by the broader healthcare system.

Among the AYAs with medical conditions, the developmental period is marked by unique physical and psychosocial experiences that co-occur with chronic health needs. Under healthy circumstances, there is a normative increase in social and school or work responsibilities, and AYAs begin to differentiate themselves from caregivers and move closer to their peer group [6]. In addition, adolescence and young adulthood are associated with identity development and substantial physical change. Many of these developmental experiences can be impacted by a chronic illness. For example, on a social level, AYAs with chronic health conditions may need to rely on parents/caregivers to help manage disease morbidities, provide financial support, or handle logistical/pragmatic challenges [7,8], which constrains their level of independence. Further, disease morbidities (e.g., pain and disfigurement), adverse treatment side effects, physician visits, and/or inpatient

**Citation:** Allen, T.; Reda, S.; Martin, S.; Long, P.; Franklin, A.; Bedoya, S.Z.; Wiener, L.; Wolters, P.L. The Needs of Adolescents and Young Adults with Chronic Illness: Results of a Quality Improvement Survey. *Children* **2022**, *9*, 500. https://doi.org/10.3390/ children9040500

Academic Editor: Mark A. Connelly

Received: 3 March 2022 Accepted: 29 March 2022 Published: 2 April 2022

**Publisher's Note:** MDPI stays neutral with regard to jurisdictional claims in published maps and institutional affiliations.

**Copyright:** © 2022 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https:// creativecommons.org/licenses/by/ 4.0/).

hospitalizations impede typical social endeavors, such as extracurricular activities, routine socializing, or dating [9,10]. In addition, the developing adolescent brain [11], as well as disease- and treatment-related cognitive impairments, may limit the ability of AYAs to function independently and manage their complex healthcare needs [12]. The disruption caused by chronic health challenges during the AYA period directly impacts developmental processes and creates vulnerability into adulthood [13].

Given the challenges faced by AYAs with chronic illnesses, it is not surprising that research reports lower levels of socioemotional health compared to same-aged individuals without chronic health needs. Poorer social-emotional functioning is evident across AYA populations, including high rates of anxiety and depression in youths with sickle cell disease (SCD) [14] and increased feelings of self-consciousness in youths with lupus [15]. In addition, AYAs with neurofibromatosis type 1 (NF1) have higher rates of emotional and behavioral problems, as well as a diminished quality of life, compared to the AYAs of the general population [16]. Taken together, these examples suggest that AYAs with chronic illness face significant burdens, which make it that much more important that this population receive targeted support during the transitional period.

#### *Current Research on the Unmet Needs of the AYAs with Chronic Illness*

There is a clear need to provide tailored support and services to AYAs with chronic health conditions. Unfortunately, designated support and services for this population are often unavailable [17,18]. Unmet healthcare needs among AYAs with chronic medical conditions are associated with reduced quality of life [19] and put them at risk of negative behavioral health outcomes, such as nonadherence to medication, missed medical appointments, and poor health outcomes [20–22]. However, quality of life may be improved both during treatment and after discharge with the addition of specific, tailored informative processes and social services [23]. These services aid the transition from being an AYA who is fully dependent on others for their medical care to an independent adult who manages their own healthcare needs.

Specifically, studies suggest that AYAs want wide-ranging information and guidance from their providers on major life concerns, ranging from brain fog to fertility to finances [19,24]. An equally important unmet need is social support. Studies show that these patients would like to make social connections early on in their care and that many of them report loneliness and disconnection from the AYA community [24,25]. Further, the provision of developmentally appropriate information and activities that enhance social connections within care systems are important needs that remain unmet [26]. Despite the identification of these problems and potential solutions, gaps in AYA services persist.

To best plan for the growing AYA population at our institution, we engaged patients and caregivers in the process to determine the programmatic services that are needed for this age group at the National Institutes of Health through an anonymous survey. Gathering stakeholder input early on is critical for understanding the relevant concerns and will aid in decision-making about the development and implementation of programs and services. Thus, the first aim of this quality assurance study was to examine the needs and services of AYAs with chronic illnesses (i.e., cancer, SCD, NF1, and primary immunodeficiencies) and their caregivers. The secondary aims were to explore the differences between AYAs with different diagnoses as well as among AYAs of different demographic groups, including age, sex, and race. Moreover, this paper aims to highlight the key areas for improving transitional care and services and provides recommendations for those who treat AYAs with chronic illnesses.

#### **2. Materials and Methods**