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[国际项目——MGTX研究]

[International project--MGTX study].

作者信息

Yoshikawa Hiroaki

机构信息

Health Service Center, Kanazawa University.

出版信息

Rinsho Shinkeigaku. 2007 Nov;47(11):880-2.

Abstract

MGTX Study is a multi-center, international, single-blinded, randomized trial to determine whether extended transsternal thymectomy (ETTX) for patients with myasthenia gravis receiving the prednisone protocol confers added benefits to the prednisone protocol alone. The aims of the study are answering following three questions. 1) Does ETTX combined with prednisone result in a greater improvement in myasthenic weakness, compared to prednisone alone? 2) Does ETTX combined with prednisone result in a lower total dose of prednisone, thus decreasing the likelihood of concurrent and long-term toxic effects, compared to prednisone alone? 3) Does ETTX combined with prednisone enhance quality of life by reducing adverse events and symptoms associated with the therapies, compared to prednisone alone? Primary outcome measure is consists with 1) Comparison of the prednisone protocol alone to prednisone protocol plus ETTX, based on the clinical response to therapy measured over the 3 year trial period by the Area Under the Quantitative Myasthenia Gravis (QMG) Weakness Score (AUQMG), and 2) Testing the difference in the total prednisone used over the 3 year trial period measured by pill count from blister packs (Area Under the Prednisone Dose Time Curve, AUDTC), conditional on the results of comparing AUQMG.

摘要

MGTX研究是一项多中心、国际性、单盲、随机试验,旨在确定对于接受泼尼松方案治疗的重症肌无力患者,扩大经胸骨胸腺切除术(ETTX)是否能为单独使用泼尼松方案带来额外益处。该研究的目的是回答以下三个问题。1)与单独使用泼尼松相比,ETTX联合泼尼松是否能使肌无力症状有更大改善?2)与单独使用泼尼松相比,ETTX联合泼尼松是否能降低泼尼松的总剂量,从而降低同时出现和长期毒性作用的可能性?3)与单独使用泼尼松相比,ETTX联合泼尼松是否能通过减少与治疗相关的不良事件和症状来提高生活质量?主要结局指标包括:1)根据在3年试验期内通过重症肌无力定量(QMG)肌无力评分曲线下面积(AUQMG)衡量的治疗临床反应,比较单独使用泼尼松方案与泼尼松方案加ETTX;2)在比较AUQMG结果的条件下,通过泡罩包装的药丸计数测量3年试验期内使用的泼尼松总量的差异(泼尼松剂量时间曲线下面积,AUDTC)。

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