Patient-reported impact of myasthenia gravis in the real world: findings from a digital observational survey-based study (MyRealWorld MG)

Abstract

Objectives: This study aims to explore the impact of myasthenia gravis (MG) - in terms of treatments, side effects, comorbidities, psychological health and work or study- in the real world from a patient perspective.

Design and participants: This is a prospective, observational, digital, longitudinal study. Adults diagnosed with MG residing in the USA, Japan, Germany, the UK, Italy, Spain or Canada were eligible to participate in the study. There were no other exclusion criteria. Participants used a bespoke smartphone application to confirm eligibility, provide consent and enter data about their MG into a profile, a tracker to record MG-related events and a series of patient-reported outcome instruments. 1693 participants completed at least 1 survey and were included in this analysis.

Results: Results are presented as a percentage of respondents to each survey question. The study population was largely female (69% of 1586 respondents), with an average age of 49.9 years (SD 14.8). In the previous 12 months, 83.7% of 1412 respondents confirmed that they had received one or more routine treatments for MG, and 67.1% of 255 respondents confirmed that they had experienced a side effect in the previous month. Commonly experienced comorbidities reported by 966 respondents were thyroid problems, hypertension and anxiety, experienced by 37.5%, 31.4% and 28.0% of respondents, respectively.According to 889 respondents to the Hospital Anxiety and Depression Scale survey, 52.7% and 43.2% had a score indicative of at least mild anxiety and mild depression, respectively. Of 257 respondents, 33.0% reported experiencing a work or study impact in the past month.

Conclusions: This analysis of baseline characteristics of the MyRealWorld MG study population indicates that, despite current treatments, patients experience notable burden. Further scheduled analyses will develop a longitudinal picture of MG burden.

Trial registration number: NCT04176211.

Keywords: adult neurology; neurology; neuromuscular disease.

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. FA and CQ are employees of Vitaccess. JB, GP, FDR, JR and SP are employees of argenx BV, the sponsor of the study. SB-A is a consultant and receives honoraria from argenx BV for this study. AM has received speaker honoraria, consulting fees or financial research support from Alexion, argenx BV, Grifols, Hormosan Pharma, Janssen, Octapharma and UCB. He serves as Chairman of the medical advisory board of the German Myasthenia Gravis Society. KGC has received advisory board honoraria, speaker fees and funding for research from Alnylam Pharmaceuticals, 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. FS has received public-speaking honoraria from Almirall, Biogen, Mylan, Novartis, Roche, Sanofi and Teva Pharmaceuticals, and served on advisory boards for Almirall, argenx BV, AveXis, Biogen, Forward Pharma, Lexeo Therapeutics, Merck, Novartis, Novatek Pharmaceuticals, Pomona, Roche, Sanofi and Takeda.

© Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

Figures

Figure 1

Routine treatment regimens in the 12 months prior to study registration (% of 1412 responding participants—note that participants could select more than one treatment). AChEI, acetylcholinesterase inhibitor; NSISTs, non-steroidal immunosuppressive therapies.

Figure 2

Rescue treatment regimens in the 12 months prior to study registration (% of 1376 responding participants—note that participants could select more than one treatment). IVIG, intravenous immunoglobulin; PLEX, plasma exchange.

Figure 3

Symptoms attributed to side effects in the 30 days prior to study registration (% of 171 responding participants—note that participants could select more than one symptom).

Figure 4

Comorbid conditions reported at study registration (% of 1495 responding participants—note that participants could select more than one condition). COPD, chronic obstructive pulmonary disease.