Patient-reportedimpact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)

Abstract

Introduction: Myasthenia gravis (MG) is a rare, chronic, autoimmune disease, mediated by immunoglobulin G antibodies, which causes debilitating muscle weakness. As with most rare diseases, there is little patient-reported data with which to understand and address patient needs. This study explores the impact of MG in the real world from the patient perspective.

Methods and analysis: This is a 2-year prospective, observational, digital, longitudinal study of adults with MG, resident in the following countries: the USA, Japan, Germany, France, the UK, Italy, Spain, Canada and Belgium. The planned sample size is 2000. Recruitment will be community based, via patient advocacy groups, social media and word of mouth. Participants will use a smartphone application (app) to check eligibility, provide consent and contribute data. Planned data entry is as follows: (1) personal profile on enrollment-covering demographics, MG characteristics and previous care; (2) monthly event tracker-current treatments, healthcare visits, treatment-related adverse events, productivity losses; (3) monthly selection of validated generic and disease-specific patient-reported outcomes instruments: EQ-5D-5L, Myasthenia Gravis Activities of Daily Living, Myasthenia Gravis Quality of Life 15-item revised scale, Hospital Anxiety and Depression Scale and Health Utilities Index III. Analyses are planned for when the study has been running in most countries for approximately 6, 12, 18 and 24 months.

Ethics and dissemination: The study protocol has been reviewed and granted ethics approval by Salus IRB for participants resident in the following countries: Germany, the UK and the US. Local ethics approval is being sought for the following study countries: Belgium, Canada, France, Italy, Japan and Spain. Study results will be communicated to the public and participants via conference presentations and journal publications, as well as regular email, social media and in-application communication.

Trial registration number: NCT04176211.

Keywords: adult neurology; neuromuscular disease; qualitative research.

Conflict of interest statement

Competing interests: The principal investigator, ML, is CEO and owner of Vitaccess, which has been commissioned by argenx BV, to carry out the study. EB and HK are or were employees of Vitaccess. JB, TL and SP are employees of argenx BV. MFJ is a member of the EuroQol Group. SD, MFJ and SB-A are consultants and received honoraria from argenx BV for this study. KGC has received advisory board honoraria, speaker fees and funding for research from Alnylam, Biogen, CSL Behring and Sanofi-Genzyme; and travel reimbursement from Sanofi-Genzyme. KGC holds the Emil von Behring Chair in Neuromuscular and Neurodegenerative Disorders, sponsored by CSL Behring. HM has served as a consultant for argenx BV, Alexion Pharmaceuticals, Ra Pharmaceuticals and UCB Pharma and has received speaker honoraria from the Japan Blood Products Organisation and research support from the Ministry of Health, Labour and Welfare, Japan. RM has received speaking honoraria from Biomarin, Alexion and UCB, served on advisory boards for Alexion, argenx BV and UCB and received support for congress participation from Merck, Teva and Biogen. FS has received public speaking honoraria from Biogen, Mylan, Novartis, Roche, Sanofi and Teva; and served on advisory boards for Almirall, argenx BV, Avexis, Biogen, Forward Pharma, Merk, Novartis, Novatek, Pomona, Roche and Sanofi.

© Author(s) (or their employer(s)) 2021. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

Figures

Figure 1

Participant timeline. MG, myasthenia gravis; PRO, patient-reported outcome.