Bonar Kerina, Boudiaf Nada, Zaremba Piotr, Tarancón Thais, Zhou Jiachen, Jacob Saiju
UCB, Slough, UK.
UCB, Warszawa, Poland.
J Neuromuscul Dis. 2025 Jan-Feb;12(1):22143602241308194. doi: 10.1177/22143602241308194.
Myasthenia gravis (MG), a chronic and unpredictable autoimmune disease, is associated with multiple comorbidities and high disease burden.
To assess the disease burden, healthcare resource utilisation (HCRU), and treatment patterns of patients with newly diagnosed MG in England.
Data from Clinical Practice Research Datalink GP practices linked to the Hospital Episode Statistics database were used. Eligible patients had ≥1 diagnostic code for MG, with the first MG diagnostic code recorded between 01 January 2010 and 31 December 2019. Non-MG controls were selected if they had no recorded MG diagnosis and ≥12 months of data. Controls were matched for age, sex and GP practice in a maximum ratio of 5:1.
Mean follow-up duration was 2.8 and 3.1 years for the MG and non-MG cohorts, respectively. In the MG cohort, 56% of patients were male, with a mean age of 67 years at baseline. Incidence rates of all comorbidities assessed during follow-up were higher in the MG cohort than in controls. Almost two-thirds of MG patients experienced ≥1 myasthenic exacerbation during follow-up; incidence rates (95% confidence interval) of MG exacerbations and crises were 50.0 (44.7-55.9) and 1.3 (0.8-2.0) per 100 person-years, respectively. Visits to non-neurology specialists and outpatient clinics were the most common instances of HCRU overall, each being more frequent in the MG cohort than for controls. In the first year of follow-up, acetylcholinesterase inhibitors (AChEIs) and corticosteroids were used by 56.0% and 50.2% of MG patients, respectively; the use of AChEIs declined thereafter.
Despite treatment, there is a high disease burden for patients with newly diagnosed MG in England, with high rates of MG exacerbation and HCRU use. Thus, there is a need for targeted treatments with sustained efficacy and improved safety to adequately manage MG symptoms and reduce MG-related disease burden.
重症肌无力（MG）是一种慢性且不可预测的自身免疫性疾病，与多种合并症及高疾病负担相关。
评估英国新诊断MG患者的疾病负担、医疗资源利用（HCRU）及治疗模式。
使用来自临床实践研究数据链全科医生诊所并与医院事件统计数据库相链接的数据。符合条件的患者有≥1个MG诊断代码，首个MG诊断代码记录于2010年1月1日至2019年12月31日之间。若未记录MG诊断且有≥12个月的数据，则选择非MG对照。对照按年龄、性别和全科医生诊所进行匹配，最大比例为5:1。
MG队列和非MG队列的平均随访时长分别为2.8年和3.1年。在MG队列中，56%的患者为男性，基线时平均年龄为67岁。随访期间评估的所有合并症的发病率在MG队列中均高于对照。近三分之二的MG患者在随访期间经历了≥1次重症肌无力加重；MG加重和危象的发病率（95%置信区间）分别为每100人年50.0（44.7 - 55.9）和1.3（0.8 - 2.0）。总体而言，就诊于非神经科专科医生和门诊诊所是最常见的HCRU情况，在MG队列中每种情况都比对照更频繁。在随访的第一年，分别有56.0%和50.2%的MG患者使用了乙酰胆碱酯酶抑制剂（AChEIs）和皮质类固醇；此后AChEIs的使用有所下降。
尽管进行了治疗，但英国新诊断MG患者的疾病负担仍然很高，MG加重率和HCRU使用率都很高。因此，需要有持续疗效和更高安全性的靶向治疗，以充分控制MG症状并减轻MG相关疾病负担。
