Minimal manifestation status and prednisone withdrawal in the MGTX trial
Minimal manifestation status and prednisone withdrawal in the MGTX trial
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DOI：
10.1212/wnl.0000000000010031
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发表时间：
2020-08-11
期刊：
影响因子：
9.9
通讯作者：
Wolfe, Gil I.
中科院分区：
文献类型：
--
作者：
Lee, Ikjae;Kuo, Hui-Chien;Wolfe, Gil I.
目的 探讨胸腺切除的重症肌无力 (MG) 患者是否能更好地实现完全停用泼尼松的持续最低表现状态 (MMS)。 方法本研究是对 MG 胸腺切除随机试验（接受泼尼松治疗的非胸腺瘤性重症肌无力患者的胸腺切除试验 [MGTX]）数据进行事后分析。 MGTX 是一项多中心、随机、评估者盲法的 3 年试验，随后对乙酰胆碱受体 (AChR) 抗体阳性、无胸腺瘤的 MG 患者自愿延长 2 年。患者按 1:1 的比例随机分配至胸腺切除术加泼尼松组与单独泼尼松组。参与者入组时年龄为 18-65 岁，病程不超过 5 年。所有患者均每隔一天接受口服泼尼松增量至 100 毫克，直至达到 MMS，这促使只要维持 MMS，就需要标准化泼尼松逐渐减量。比较胸腺切除加泼尼松组与单独泼尼松组之间完全撤除泼尼松后持续 MMS（6 个月无 MG 症状）的实现率。结果 与单独泼尼松组相比，胸腺切除加泼尼松组中的 MG 患者更频繁地（64% vs 38%）和更快地实现持续 MMS 完全撤除泼尼松（中位时间 30 个月 vs 未实现中位时间，p < 0.001) 在 5 年的研究期间。单独使用泼尼松组中与泼尼松相关的不良症状更常见，并且随着泼尼松剂量的增加，痛苦程度也会增加。结论胸腺切除术通过增加完全撤除泼尼松后实现持续 MMS 的可能性，从而使 MG 患者受益。证据分类本研究提供了 II 类证据，表明对于具有 AChR 抗体的全身性 MG 患者，接受胸腺切除术加泼尼松的患者比接受胸腺切除术加泼尼松的患者更有可能实现持续 MMS 和完全撤除泼尼松。单独泼尼松。
ObjectiveTo examine whether sustained minimal manifestation status (MMS) with complete withdrawal of prednisone is better achieved in thymectomized patients with myasthenia gravis (MG).MethodsThis study is a post hoc analysis of data from a randomized trial of thymectomy in MG (Thymectomy Trial in Non-Thymomatous Myasthenia Gravis Patients Receiving Prednisone Therapy [MGTX]). MGTX was a multicenter, randomized, rater-blinded 3-year trial that was followed by a voluntary 2-year extension for patients with acetylcholine receptor (AChR) antibody-positive MG without thymoma. Patients were randomized 1:1 to thymectomy plus prednisone vs prednisone alone. Participants were age 18-65 years at enrollment with disease duration less than 5 years. All patients received oral prednisone titrated up to 100 mg on alternate days until they achieved MMS, which prompted a standardized prednisone taper as long as MMS was maintained. The achievement rate of sustained MMS (no symptoms of MG for 6 months) with complete withdrawal of prednisone was compared between the thymectomy plus prednisone and prednisone alone groups.ResultsPatients with MG in the thymectomy plus prednisone group achieved sustained MMS with complete withdrawal of prednisone more frequently (64% vs 38%) and quickly compared to the prednisone alone group (median time 30 months vs no median time achieved, p < 0.001) over the 5-year study period. Prednisone-associated adverse symptoms were more frequent in the prednisone alone group and distress level increased with higher doses of prednisone.ConclusionsThymectomy benefits patients with MG by increasing the likelihood of achieving sustained MMS with complete withdrawal of prednisone.Classification of evidenceThis study provides Class II evidence that for patients with generalized MG with AChR antibody, those receiving thymectomy plus prednisone are more likely to attain sustained MMS and complete prednisone withdrawal than those on prednisone alone.