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作者：CV Oddis,et al. 翻译：北医三院王文季 发布：孙琳 审核：刘畅
目的:为评估利妥昔单抗对成人和儿童肌炎病人的安全性和有效性而进行的一项随机双盲安慰剂对照试验。 方法:以难治性成人和青少年皮肌炎病人及成人难治性多肌炎病人为纳入对象，纳入标准包括成人肌无力和≥2项其他的核心组异常值，青少年皮肌炎要求≥3项核心组异常值，伴或不伴有肌无力。病人随机分为早期或晚期接收利妥昔单抗治疗组，纳入研究后可以接受激素及免疫抑制剂治疗，两组主要终点时间为达到国际肌炎评估和临床研究小组初步定义的获得改善时间（DOI）。次要终点为达到肌肉力量提高20%并且20%早期或晚期接收利妥昔单抗治疗组病人第8周获得改善。 结果: 在200名随机分配的病人中（包括76名多肌炎，76名皮肌炎，48名青少年皮肌炎患者），晚期（n=102）或早期（n=93）接收利妥昔单抗治疗2组中（P=0.74，log检验）195名病人达到获得改善的时间无明显差别，获得改善的中位时间分别为20.2周和20.0。次要终点在两组病人中也没有显著差异。通过44周临床试验，两组中161（83%）名随机病人达到获得改善，并且个体核心组异常值获得改善。 结论:尽管两组治疗在主要终点和次要终点间无显著差别，83%成人和青少年难治性肌炎患者获得改善。考虑不同的试验设计，B细胞清除疗法在治疗肌炎的作用需要进一步研究。
附原文： Objective To assess the safety and
efficacy of rituximab in a randomized, double-blind, placebo-phase trial in
adult and pediatric myositis patients. Methods Adults with refractory
polymyositis (PM) and adults and children with refractory dermatomyositis (DM)
were enrolled. Entry criteria included muscle weakness and ≥2
additional abnormal values on core set measures (CSMs) for adults.
Juvenile DM patients required ≥3 abnormal CSMs, with or without muscle
weakness. Patients were randomized to receive either rituximab early or
rituximab late, and glucocorticoid or immunosuppressive therapy was allowed at
study entry. The primary end point compared the time to
achieve the International Myositis Assessment and Clinical Studies Group
preliminary definition of improvement (DOI) between the 2 groups.The secondary end points were the time to achieve ≥20% improvement in muscle
strength and the proportions of patients in the early and late rituximab groups
achieving the DOI at week 8. Results Among 200 randomized patients (76 with PM, 76
with DM, and 48 with juvenile DM), 195 showed no difference in the time to
achieving the DOI between the rituximab late (n = 102) and rituximab early (n =
93) groups ( P = 0.74 by log rank test), with a median time to achieving a DOI
of 20.2 weeks and 20.0 weeks, respectively. The secondary end points also did
not significantly differ between the 2 treatment groups. However, 161 (83%) of
the randomized patients met the DOI, and individual CSMs improved in both
groups throughout the 44-week trial. Conclusion Although there were no
significant differences in the 2 treatment arms for the primary and secondary
end points, 83% of adult and juvenile myositis patients with refractory disease
met the DOI. The role of B cell–depleting therapies in
myositis warrants further study, with consideration for a different trial
design.
引自：Oddis C V, Reed A M, Aggarwal R, et al.
Rituximab in the treatment of refractory adult and juvenile dermatomyositis and
adult polymyositis: a randomized, placebo-phase trial.[J]. Arthritis &
Rheumatism, 2013, 65(2):314-24.
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