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杜克重症肌无力临床登记处:II. 结果分析。

The Duke Myasthenia Gravis Clinic Registry: II. Analysis of outcomes.

机构信息

Neuromuscular Division, Department of Neurology, Duke University Medical Center, Durham, North Carolina.

出版信息

Muscle Nerve. 2023 Apr;67(4):291-296. doi: 10.1002/mus.27794. Epub 2023 Feb 20.

Abstract

INTRODUCTION/AIMS: The Duke Myasthenia Gravis (MG) Clinic Registry contains comprehensive physician-derived data on patients with MG seen in the Duke MG Clinic since 1980. The aim of this study was to report outcomes in patients seen in the clinic and treated according to the International Consensus Guidance statements.

METHODS

This is a retrospective cohort study of patients initially seen after 2000 and followed for at least 2 years in the clinic. Treatment goal (TG) was defined as achieving MGFA post-intervention status of "minimal manifestations" or better; PIS was determined by the treating neurologist. Time-to-event analysis, including Cox proportional hazards modeling, was performed to assess the effect of sex, acetylcholine receptor antibody (AChR-Ab) status, age at disease onset, distribution (ocular vs generalized), thymectomy, and thymoma on the time to achieve TG.

RESULTS

Among the 367 cohort patients, 72% achieved TG (median time less than 2 years). A greater proportion of patients with AChR-Abs and thymectomy achieved TG and they did so sooner than patients without these antibodies or thymectomy. Otherwise, there were no significant differences in these findings within the tested subgroups. The disease duration at the first Duke Clinic visit was shorter in patients who achieved TG than in those who did not.

DISCUSSION

These results demonstrate outcomes that can be achieved in patients with MG treated according to the current Consensus Guidance statements. Among other things, they can be used to determine the added value and potential role of new treatment modalities developed since 2018.

摘要

简介/目的:杜克重症肌无力(MG)临床注册中心包含自 1980 年以来在杜克 MG 临床中心就诊的 MG 患者的综合医生来源数据。本研究的目的是报告根据国际共识指南声明在临床中心就诊和治疗的患者的结局。

方法

这是一项对 2000 年后首次就诊且在临床中心至少随访 2 年的患者进行的回顾性队列研究。治疗目标(TG)定义为达到 MGFA 干预后“最小表现”或更好的状态;PIS 由治疗神经科医生确定。进行生存分析,包括 Cox 比例风险模型,以评估性别、乙酰胆碱受体抗体(AChR-Ab)状态、疾病发病年龄、分布(眼型与全身型)、胸腺切除术和胸腺瘤对达到 TG 的时间的影响。

结果

在 367 名队列患者中,72%达到 TG(中位数时间不到 2 年)。AChR-Ab 阳性和胸腺切除术的患者达到 TG 的比例更高,并且比没有这些抗体或胸腺切除术的患者更早达到 TG。否则,在测试的亚组中,在这些发现中没有显著差异。与未达到 TG 的患者相比,达到 TG 的患者首次在杜克临床中心就诊时的疾病持续时间更短。

讨论

这些结果表明,根据当前共识指南声明治疗的 MG 患者可以取得这些结果。除其他外,它们可用于确定自 2018 年以来开发的新治疗方法的附加值和潜在作用。

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