Discussion
fatigue, anxiety/depression
DISEASE
Efgartigimod had a rapid impact on HRQoL, with significant improvements as early as the first week of treatment in each TC. These improvements were consistent across multiple measures and were similar for the effect seen in TC 1 and TC 2. The observed benefits on HRQoL were reproducible and durable.At baseline and despite being on stable treatment at screening, participants in this study had fairly low EQ-5D-5L health utility scores (0.62–0.66), highlighting the potential burden of gMG. The majority reported issues with mobility, usual activities, and self-care and a considerable proportion reported pain/discomfort and anxiety/depression. These baseline levels underscore the need for more-effective treatment options for this patient population.The study did not include formal data analysis of AChR-Ab− participants due to the small sample size; the study was not powered to demonstrate statistically significant differences between the efgartigimod and placebo groups in the AChR-Ab− population. However, results for MG-QOL15r and EQ-5D-5L were similar for the overall efgartigimod group (AChR-Ab+ and AChR-Ab−), indicating that there were no substantial differences between the AChR-Ab+ and AChR-Ab− populations.Substantial and consistent improvements were seen in disease-specific MG-QOL15r scores. The MG-QOL15r apprises a clinician of how a patient has evaluated their level of satisfaction/dissatisfaction with current MG manifestations and offers an efficient and standardized assessment of physical, social, and psychological domains that is specific to patients with MG [Improvements were seen across all dimensions captured in the EQ-5D-5L for the efgartigimod group compared with little or no improvement for the placebo group. The maximum mean difference from TC baseline in EQ-5D-5L utility score was seen at 4 weeks in both TC 1 and TC 2 for the efgartigimod group (TC 1, 0.158; TC 2, 0.187) and was higher than the estimated minimally important difference cut point for the EQ-5D-5L utility scores (range: 0.03–0.05) [The pattern of improvements in HRQoL corresponded well with the change in IgG level, as shown in Fig. We evaluated whether the benefits of efgartigimod were consistent among key patient demographic characteristics known to impact HRQoL and found consistent treatment benefit. Certain patient demographic criteria, including female sex and increasing age, have been identified as drivers of HRQoL in MG [Strong correlations have been reported between symptom control and HRQoL benefit [Given such findings, HRQoL measures are becoming increasingly important for evaluating the benefit of therapeutic interventions. They have historically been undervalued both in clinical studies of gMG and in clinical practice, where treatment intent has been primarily focused on preventing hospitalization. These measures, however, better assess the full burden of disease experienced by individuals with gMG; HRQoL measures reflect patients’ perspective on the effectiveness of treatment and disease burden and can be used to foster patient-centric care in clinical practice. Regulatory bodies have also recognized the importance of patient-reported outcome measures, including HRQoL, and are increasingly using these data to support label claims and reimbursement decisions. The consistent and substantial achievement of clinical and HRQoL end points in this study demonstrates the broader benefit of treatment with efgartigimod beyond relief of immediate signs and symptoms of gMG.A limitation of this study is that although fatigue is an important aspect of gMG, it is not fully captured by the assessment tools utilized. This might explain the difference seen between the EQ-5D-5L VAS scores, which represent participants’ perceived overall health, and EQ-5D-5L utility scores, which represent perceived problems in specific dimensions. Findings were based on clinical study data with limited data/follow-up and a specific dosing schedule. The study design evaluated duration of effect after each TC, which did not allow extrapolation of HRQoL impact to long-term real-life scenarios wherein patients are treated according to neurologic evaluation. Future studies using real-world data will be needed to further validate the benefit on HRQoL of efgartigimod in gMG.