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Patient-reportedimpact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG).
Berrih-Aknin, Sonia; Claeys, Kristl G; Law, Nancy; Mantegazza, Renato; Murai, Hiroyuki; Saccà, Francesco; Dewilde, Sarah; Janssen, Mathieu F; Bagshaw, Emma; Kousoulakou, Hara; Larkin, Mark; Beauchamp, Jon; Leighton, Trevor; Paci, Sandra.
  • Berrih-Aknin S; INSERM, Institute of Myology, Center of Research in Myology, Sorbonne Université, Paris, France.
  • Claeys KG; Department of Neurology, University Hospitals Leuven, Leuven, Belgium.
  • Law N; Laboratory for Muscle Diseases and Neuropathies, Department of Neurosciences, KU Leuven, Leuven, Belgium.
  • Mantegazza R; Myasthenia Gravis Foundation of America Inc, Westborough, Massachusetts, USA.
  • Murai H; Fondazione IRCCS, Istituto Nazionale Neurologico Carlo Besta, Milan, Italy.
  • Saccà F; Associazione Italiana Miastenia e Malattie Immunodegenerative, Milan, Italy.
  • Dewilde S; Department of Neurology, International University of Health and Welfare, Narita, Japan.
  • Janssen MF; DNSRO Department, University of Naples Federico II, Naples, Italy.
  • Bagshaw E; Services in Health Economics, Brussels, Belgium.
  • Kousoulakou H; Section Medical Psychology and Psychotherapy, Department of Psychiatry, Erasmus MC, Rotterdam, Netherlands.
  • Larkin M; Vitaccess Limited, London, UK emma.bagshaw@vitaccess.com.
  • Beauchamp J; Vitaccess Limited, London, UK.
  • Leighton T; Vitaccess Limited, London, UK.
  • Paci S; argenx BV, Ghent, Belgium.
BMJ Open ; 11(7): e048198, 2021 07 20.
Article in English | MEDLINE | ID: covidwho-1495461
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.
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Full text: Available Collection: International databases Database: MEDLINE Main subject: Activities of Daily Living / Myasthenia Gravis Type of study: Clinical Practice Guide / Observational study / Randomized controlled trials Limits: Adult / Humans Country/Region as subject: North America / Asia / Europa Language: English Journal: BMJ Open Year: 2021 Document Type: Article Affiliation country: Bmjopen-2020-048198

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Full text: Available Collection: International databases Database: MEDLINE Main subject: Activities of Daily Living / Myasthenia Gravis Type of study: Clinical Practice Guide / Observational study / Randomized controlled trials Limits: Adult / Humans Country/Region as subject: North America / Asia / Europa Language: English Journal: BMJ Open Year: 2021 Document Type: Article Affiliation country: Bmjopen-2020-048198