Patient-reported outcome measures
fatigue, Cancer
K DEFICIENCY, VDS, CANCER
The PKDD is a self-administered, daily, 7-item PRO measure of the core signs and symptoms of PK deficiency in adults, with a 24-h recall period constituting the same day as administration (Table The PKDIA is a 12-item PRO measure, which assesses the impacts of PK deficiency experienced by adults on their HRQoL (e.g., feeling bothered by appearance, impact on the ability to do household activities and on moderate physical activity etc.). The recall period for all PKDIA questions is the past seven days. Items 1–8, 9b, 10, and 11b have response options coded to an 11-point NRS. Items 9 and 11 are two-part items where part To support convergent and discriminant validity analyses of the PKDD and PKDIA, additional PROs were employed that were hypothesized to be part of the nomological network of constructs identified as important to patients using Cronbach’s method, built on the concepts of Campbell and Fiske [The European Quality of Life Five-Dimensional Descriptive System (EQ-5D-5L), that measures mobility, self-care, usual activities, pain/discomfort, and anxiety/depression;The Functional Assessment of Cancer Therapy-Anemia (FACT-An), that assesses fatigue and anemia-related concerns;The mental health and physical functioning component summaries (MCS and PCS, respectively) of the 12-item short form health survey version 2.0 (SF-12v2);The patient global impression of severity (PGIS), a disease-specific, single-item measure that assesses the patient-reported severity of the effect of PK deficiency over one day, rated on a 5-point Likert scale VDS (severity categories: 0 = not a lot; 1 = a little; 2 = moderately; 3 = a lot; and 4 = very much).The PGIS rated at baseline was also used in the assessment of known-groups validity for the PKDD and PKDIA. For collection of all PRO data, patients were provided with an electronic diary (Signant Health ePRO platform) at screening to record responses to PRO measures within the appropriate recall timeframe throughout the trial.