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

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 t...

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Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8292816/
https://www.ncbi.nlm.nih.gov/pubmed/34285010
http://dx.doi.org/10.1136/bmjopen-2020-048198
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author 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
author_facet 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
author_sort Berrih-Aknin, Sonia
collection PubMed
description 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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spelling pubmed-82928162021-08-13 Patient-reported impact 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 BMJ Open Neurology 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. BMJ Publishing Group 2021-07-19 /pmc/articles/PMC8292816/ /pubmed/34285010 http://dx.doi.org/10.1136/bmjopen-2020-048198 Text en © 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. https://creativecommons.org/licenses/by-nc/4.0/This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) .
spellingShingle Neurology
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
Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
title Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
title_full Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
title_fullStr Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
title_full_unstemmed Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
title_short Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)
title_sort patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (myrealworld mg)
topic Neurology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8292816/
https://www.ncbi.nlm.nih.gov/pubmed/34285010
http://dx.doi.org/10.1136/bmjopen-2020-048198
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