Patient-reported impact of myasthenia gravis in the real world
| Autor: | Sonia Berrih-Aknin, Emma Bagshaw, Trevor Leighton, Renato Mantegazza, Hara Kousoulakou, Sandra Paci, Mark Larkin, Sarah Dewilde, Hiroyuki Murai, Nancy Law, Mathieu F. Janssen, Kristl G. Claeys, Jon Beauchamp, Francesco Saccà |
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| Přispěvatelé: | Institut de Myologie, Centre National de la Recherche Scientifique (CNRS)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Association française contre les myopathies (AFM-Téléthon)-Sorbonne Université (SU), University Hospitals Leuven [Leuven], Fondazione 'Istituto Neurologico Nazionale C. Mondino', International University of Health and Welfare Hospital (IUHW Hospital), University of Naples Federico II, Erasmus University Medical Center [Rotterdam] (Erasmus MC), Psychiatry |
| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: |
Longitudinal study
Activities of daily living Patient advocacy 0302 clinical medicine Belgium Japan QUALITY-OF-LIFE Germany Activities of Daily Living Health care Longitudinal Studies Prospective Studies CRISIS 030503 health policy & services adult neurology General Medicine 3. Good health Observational Studies as Topic Treatment Outcome Italy Neurology Medicine [SDV.NEU]Life Sciences [q-bio]/Neurons and Cognition [q-bio.NC] France 0305 other medical science Life Sciences & Biomedicine Health Utilities Index Adult Canada medicine.medical_specialty HOSPITAL ANXIETY 03 medical and health sciences Medicine General & Internal Quality of life (healthcare) General & Internal Medicine Myasthenia Gravis MANAGEMENT medicine Humans Science & Technology business.industry neuromuscular disease Spain Family medicine Quality of Life Observational study business qualitative research 030217 neurology & neurosurgery |
| Zdroj: | BMJ Open BMJ Open, BMJ Publishing Group, 2021, 11 (7), pp.e048198. ⟨10.1136/bmjopen-2020-048198⟩ BMJ Open, Vol 11, Iss 7 (2021) BMJ Open, 11(7):e048198. BMJ Publishing Group |
| ISSN: | 2044-6055 |
| Popis: | International audience; Download PDFPDFNeurologyProtocolPatient-reportedimpact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG) Sonia Berrih-Aknin1, Kristl G Claeys2,3, Nancy Law4, Renato Mantegazza5,6, Hiroyuki Murai7, Francesco Saccà8, Sarah Dewilde9, Mathieu F Janssen10, http://orcid.org/0000-0001-7208-1303Emma Bagshaw11, Hara Kousoulakou11, Mark Larkin11, Jon Beauchamp12, Trevor Leighton12, Sandra Paci12 INSERM, Institute of Myology, Center of Research in Myology, Sorbonne Université, Paris, France Department of Neurology, University Hospitals Leuven, Leuven, Belgium Laboratory for Muscle Diseases and Neuropathies, Department of Neurosciences, KU Leuven, Leuven, Belgium Myasthenia Gravis Foundation of America Inc, Westborough, Massachusetts, USA Fondazione IRCCS, Istituto Nazionale Neurologico Carlo Besta, Milan, Italy Associazione Italiana Miastenia e Malattie Immunodegenerative, Milan, Italy Department of Neurology, International University of Health and Welfare, Narita, Japan DNSRO Department, University of Naples Federico II, Naples, Italy Services in Health Economics, Brussels, Belgium Section Medical Psychology and Psychotherapy, Department of Psychiatry, Erasmus MC, Rotterdam, Netherlands Vitaccess Limited, London, UK argenx BV, Ghent, BelgiumIntroduction 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. |
| Databáze: | OpenAIRE |
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