Physician- and patient-reported perspectives on myasthenia gravis in Europe: a real-world survey

Orphanet Journal of Rare Diseases

Research

Keisha Golden¹  Jonathan DeCourcy¹  Gregor Gibson¹  Christian Taylor¹  Anna Scowcroft²  Milada Mahic²  Ali Bozorg³ 

[1] Adelphi Real World, Bollington, UK;UCB Pharma, 208 Bath Road, SL1 3WE, Slough, UK;UCB Pharma, Morrisville, NC, USA;

关键词: Myasthenia gravis;    Real-world data;    Diagnosis;    Symptoms;    HCRU;    Disease management;    Quality of life;   

DOI  :  10.1186/s13023-023-02727-0

 received in 2022-09-23, accepted in 2023-05-14,  发布年份 2023

来源: Springer

【 摘 要 】

BackgroundMyasthenia gravis (MG) is a rare, chronic, debilitating, unpredictable, and potentially life-threatening neuromuscular disease. There is a lack of real-world data on disease management that could be used to further understand and address unmet patient needs and burden. We aimed to provide comprehensive real-world insights in the management of MG in five European countries.MethodsData were collected using the Adelphi Real World Disease Specific Programme™ in MG, a point-in-time survey of physicians and their patients with MG in France, Germany, Italy, Spain, and the United Kingdom (UK). Physician- and patient-reported clinical data were collected, including demographics, comorbidities, symptoms, disease history, treatments, healthcare resource utilization (HCRU), and quality of life outcomes.ResultsIn total, 144 physicians completed 778 patient record forms from March to July 2020 in the UK, and from June to September 2020 in France, Germany, Italy and Spain. Mean patient age at symptom onset was 47.7 years, with a mean time from symptom onset to diagnosis of 332.4 days (10.97 months). At diagnosis, 65.3% of patients were classified as Myasthenia Gravis Foundation of America Class II or above. Mean number of symptoms reported at diagnosis per patient was five, with ocular myasthenia reported in at least 50% of patients. At time of survey completion, the mean number of symptoms reported per patient was five and ocular myasthenia and ptosis were each still present in more than 50% of patients. Acetylcholinesterase inhibitors were the most commonly prescribed chronic treatments in all countries. Of 657 patients treated with chronic treatment at the time of the survey, 62% continued to experience moderate-to-severe symptoms. On average, 3.1 healthcare professionals (HCPs) were involved in patient management, 6.2 consultations were made per patient with any HCP over the last 12 months, and 178 (22.9%) patients were hospitalized in the last 12 months. Overall, HCRU and disease management were similar across all countries.ConclusionsOur findings demonstrated the high burden of MG despite current treatment options for patients with MG.

【 授权许可】

CC BY   
© The Author(s) 2023

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