People Diagnosed with Myasthenia Gravis have Lower health-related quality of life and Need More Medical and Caregiver Help in Comparison to the General Population: Analysis of Two Observational Studies

S. Dewilde*, G. Phillips, S. Paci, F. De Ruyck, N. H. Tollenaar, M. F. Janssen

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

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Abstract

Introduction: Myasthenia gravis (MG) is a neuromuscular disease causing extreme muscular fatigue, triggering problems with vision, swallowing, speech, mobility, dexterity, and breathing. This analysis intended to estimate the health-related quality-of-life impact, the medical burden, and the need for caregiver help of people diagnosed with MG. Methods: MyRealWorld-MG (MRW) is an observational study among adults diagnosed with MG in 9 countries. The General Population Norms (POPUP) observational study enrolled representative members of the general population in 8 countries. In both digital studies, respondents entered personal characteristics and provided data on medical conditions, EQ-5D-5L, HUI3, MG-Activities of Daily Living (MG-ADL), sick leave, caregiver help, and medical care utilization. Results: In MRW (n = 1859), 58.4% of respondents had moderate-to-severe MG. Average utility values were lower in MRW versus POPUP (0.739 vs. 0.843 for EQ-5D-5L; 0.493 vs. 0.746 for HUI3), and declined with more severe disease (0.872, 0.707, 0.511 EQ-5D-5L utilities and 0.695, 0.443, 0.168 HUI3 utilities for mild, moderate, and severe MG, respectively). Taking sick leave in the past month was 2.6 times more frequent among people diagnosed with MG compared to the general population (34.4% vs. 13.2%) and four times more people diagnosed with MG reported needing help from a caregiver (34.8% vs. 8.3%). Use of medical care was twice as likely in MRW in comparison with POPUP (51.9% vs. 24.6%). Conclusion: This direct comparison of people diagnosed with MG and the general population using two large international studies revealed significant negative impact of MG. Results were consistent across all outcomes, in all countries.

Original languageEnglish
Pages (from-to)4377-4394
Number of pages18
JournalAdvances in Therapy
Volume40
Issue number10
Early online date25 Jul 2023
DOIs
Publication statusPublished - Oct 2023

Bibliographical note

Funding Information:
The study design, data collection, data analysis and manuscript writing was funded by Argenx BV, Ghent, Belgium. No other grant or award is applicable. Argenx BV also funded the rapid service and Open Access fee.

Funding Information:
The authors would like to thank all the participants of both studies for sharing their anonymized data and thank Vitaccess and the Scientific Advisory Board for providing access to the MyRealWorld-MG data. The study design, data collection, data analysis and manuscript writing was funded by Argenx BV, Ghent, Belgium. No other grant or award is applicable. Argenx BV also funded the rapid service and Open Access fee. Sarah Dewilde and Mathieu F Janssen contributed to the design of the study, analyzed the data, interpreted the findings, and wrote the manuscript. Nafthali Tollenaar contributed to the interpretation of the findings and the manuscript writing. Glenn Phillips, Sandra Paci and Femke De Ruyck designed the study, provided strategic advice, interpreted the findings, and reviewed the manuscript. Sarah Dewilde, the principal investigator of the study, Nafthali Tollenaar and Mathieu F Janssen have been commissioned by argenx BV and received honoraria to design the study, analyze and report the data. Glenn Phillips, Sandra Paci and Femke De Ruyck are employees of argenx BV, the sponsor of the study. Ethical approval was obtained in all countries for both studies from Salus IRB (US, UK, Germany, Netherlands, Denmark), CPP (France), KULeuven (Belgium), Veritas IRB (Canada), local ethical approval (Spain, Italy), and MINS-IRB (Japan) [17] and the authors received permission to access the data. Informed consent to collect data, analyze and report anonimzed and aggregate data was obtained from all participants from both studies. The study was performed in accordance with the Declaration of Helsinki. Anonymized, aggregated study data is available upon reasonable request through the corresponding author (for POPUP) and through the Scientific Advisory Board (for MyRealWorld-MG).

Publisher Copyright:
© 2023, The Author(s).

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