单中心超晚发型重症肌无力队列的临床特点及短期预后分析

Clinical Features and Short-term Prognosis of A Single-center Ultra-late Onset Myasthenia Gravis Cohort

目的回顾性分析并总结单中心超晚发型重症肌无力(MG)的基线临床特点及短期预后情况。方法纳入2019年1月1日至2021年8月1日复旦大学附属华山医院MG数据库中登记确诊的361例MG患者,收集MG患者的人口学(性别、年龄)资料、临床特点、合并慢性基础疾病、用药等基线资料及MG评分进行回顾性分析。MG相关评分包括MG日常生活质量量表(MG-ADL),定量评分(MGFA-QMG)及MG生存质量(QoL-15)评分。根据发病年龄分为3组:早发型(18~49岁)组(172例)、晚发型(50~64岁)组(108例)及超晚发型(≥65岁)组(81例)。对超晚发型组的临床特点及短期预后进行统计学分析。结果361例MG患者中,男女性别比为149∶212,平均年龄(49.89±15.7)岁。与早发型组比较,超晚发型组的基线临床特点:(1)两组均以女性为主,但超晚发型组中男性比例显著增高(P=0.0001);(2)合并甲状腺功能亢进患者比例显著降低(P=0.013)。与超晚发型组高龄匹配的临床基线特点包括:BMI增高(P=0.0015),高血压病(P<0.0001)及糖尿病(P<0.0001)比例升高。基线期早发型组的MG相关评分(MG-ADL、MGFA-QMG及QoL-15)及治疗药物与超晚发型组比较差异无显著性。治疗后的短期(6个月)疗效:超晚发型组自身治疗前后对比可见MG-ADL评分中的延髓肌总分治疗后显著下降(P=0.0001),而QMG总分及其中的眼肌总分显著下降(P=0.0397及P=0.0079)。结论超晚发型MG与早发型MG患者比较,具有男性比例高、合并慢性基础疾病较多、合并甲状腺功能亢进患者比例低等基线临床特点,超晚发型组患者经短期治疗后的临床评分可有不同程度改善。

Aim To analyze retrospectively the baseline clinical features and the short-term prognosis of single-center ultra-late onset myasthenia gravis(MG).Methods The consecutive clinical data and the laboratory findings were collected and retrospectively reviewed in a total of 361 patients with confirmed MG diagnosis in the Huashan myasthenia gravis(MG)registered from January 1,2019 to August 1,2021.The patients were divided into 3 subgroups according to the onset age:early-onset MG(EOMG)(18 to 49 years old);late-onset MG(LOMG)(50 to 64 years old),and very late-onset myasthenia gravis(VLOMG,65 years old and beyond).The clinical variables included onset symptoms,antibody subtypes,disease course,BMI,autoimmune comorbidities,family history,thymoma concurrence,thymectomy,myasthenic crisis,chronic diseases,and immunosuppressants.MG relevant scores including MG activities of daily living(MG-ADL),MGFA-quantitative MG test(MGFA-QMG),MG Quality of Life 15(QoL-15)were analyzed retrospectively.MG-ADL and QMG score were collected repeatedly at 6 months to 1 year after immunotherapy.Results A total of 172 EOMG(47.7%),108 LOMG(29.9%),and 81 VLOMG(22.4%)patients were finally enrolled.41.27%(149/361)of patients were male and the average onset age for all patients was 49.89±15.7 years old.Compared with the patients with EOMG,VLOMG have a higher male proportion(34.88%vs.40.74%,P=0.0001),higher BMI(22.81±5.02 vs.24.48±4.6,P=0.0015),and a higher concurrence with hypertension and type 2 diabetes mellitus(5.81%vs.27.16%,P=0.0001 and 1.74%vs.14.81%,P=0.0001,respectively).In contrast,the concurrence of hyperthyroidism was lower(P=0.0098).The bulbar muscle domain assessed by changes in ADL score significantly improved in VLOMG at 6 months to 1 year after immunotherapy(P=0.0001).The overall QMG score and the ocular domain score were decreased significantly after the treatment(P=0.0397 and P=0.0079).Conclusion Patients with VLOMG have the distinct clinical features of a higher proportion of males,more chronic underlying diseases and a lower proportion of patients with hyperthyroidism.The clinical scores of patients in VLOMG could be improved after the short-term treatment.