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

Sonia Berrih-Aknin; Jacqueline Palace; Andreas Meisel; Kristl G Claeys; Srikanth Muppidi; Francesco Saccà; Fatemeh Amini; Mark Larkin; Casey Quinn; Jon Beauchamp; Glenn Philips; Femke De Ruyck; Joyce Ramirez; Sandra Paci

doi:10.1136/bmjopen-2022-068104

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

BMJ Open. 2023 May 11;13(5):e068104. doi: 10.1136/bmjopen-2022-068104.

Authors

Sonia Berrih-Aknin¹, Jacqueline Palace², Andreas Meisel³, Kristl G Claeys^{4

5}, Srikanth Muppidi⁶, Francesco Saccà⁷, Fatemeh Amini⁸, Mark Larkin⁹, Casey Quinn⁹, Jon Beauchamp¹⁰, Glenn Philips¹⁰, Femke De Ruyck¹⁰, Joyce Ramirez¹⁰, Sandra Paci¹⁰

Affiliations

¹ INSERM, Institute of Myology, Center of Research in Myology, Sorbonne Université, Paris, France.
² Department of Clinical Neurology, John Radcliffe Hospital, Oxford, UK.
³ Department of Neurology, Neuroscience Clinical Research Center, Charite Universitatsmedizin Berlin, Berlin, Germany.
⁴ Department of Neurology, KU Leuven University Hospitals, Leuven, Belgium.
⁵ Laboratory for Muscle Diseases and Neuropathies, Department of Neurosciences, KU Leuven, Leuven, Belgium.
⁶ Department of Neurology and Neurosciences, Stanford University, Stanford, California, USA.
⁷ Department of DNSRO, University of Naples Federico II, Napoli, Italy.
⁸ Vitaccess Limited, Oxford, UK fatemeh.amini@vitaccess.com.
⁹ Vitaccess Limited, Oxford, UK.
¹⁰ Argenx BV, Ghent, Belgium.

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.

Publication types

Observational Study
Research Support, Non-U.S. Gov't

MeSH terms

Adult
Anxiety / epidemiology
Female
Humans
Longitudinal Studies
Middle Aged
Myasthenia Gravis* / drug therapy
Myasthenia Gravis* / therapy
Prospective Studies
Surveys and Questionnaires

Associated data

ClinicalTrials.gov/NCT04176211