健康知识库

ORIGINAL ARTICLE

Randomized Trial of Thymectomy in Myasthenia Gravis

Gil I. Wolfe, Henry J. Kaminski, Inmaculada B. Aban, for?the MGTX Study Group*

N Engl J Med 2016; 375:511-522?August 11, 2016

DOI: 10.1056/NEJMoa1602489


BACKGROUND 背景

Thymectomy has been a mainstay in the treatment of myasthenia gravis, but there is no conclusive evidence of its benefit. We conducted a multicenter, randomized trial comparing thymectomy plus prednisone with prednisone alone.

胸腺切除术是治疗重症肌无力的重要措施，但有关其疗效尚没有确切的证据。我们进行了一项多中心、随机临床试验比较胸腺切除术联合强的松与单纯使用强的松的效果。


METHODS 方法

We compared extended transsternal thymectomy plus alternate-day prednisone with alternate-day prednisone alone. Patients 18 to 65 years of age who had generalized nonthymomatous myasthenia gravis with a disease duration of less than 5 years were included if they had Myasthenia Gravis Foundation of America clinical class II to IV disease (on a scale from I to V, with higher classes indicating more severe disease) and elevated circulating concentrations of acetylcholine-receptor antibody. The primary outcomes were the time-weighted average Quantitative Myasthenia Gravis score (on a scale from 0 to 39, with higher scores indicating more severe disease) over a 3-year period, as assessed by means of blinded rating, and the time-weighted average required dose of prednisone over a 3-year period.

我们对经胸骨胸腺切除术联合隔日强的松与单纯隔日使用强的松治疗进行了比较。入选的患者年龄在18至65岁，重症肌无力为全身性且无胸腺瘤，病程不足5年，美国重症肌无力基金临床II至IV级（临床分级I至V级，级别越高，病情越严重），循环中乙酰胆碱受体抗体水平升高患者入选。主要预后指标为3年期间定量重症肌无力评分（评分0-39分，分值越高病情越重）的时间加权均值（通过设盲评估），以及3年期间强的松用量的时间加权均值。


RESULTS 结果

A total of 126 patients underwent randomization between 2006 and 2012 at 36 sites. Patients who underwent thymectomy had a lower time-weighted average Quantitative Myasthenia Gravis score over a 3-year period than those who received prednisone alone (6.15 vs. 8.99, P
2006年至2012年期间，36个中心共计126例患者接受随机分组。与单纯使用强的松的患者相比，接受胸腺切除术的患者在3年期间定量重症肌无力评分时间加权均值较低(6.15 vs. 8.99, P