分析托法替尼治疗dcSSc难治性皮肤增厚的疗效。
分析了10例接受托法替尼（5mg，每日两次）治疗的dcSSc患者的数据。选择总共12例接受强化常规免疫抑制剂治疗的dcSSc患者作为历史比较组。临床相关的反应被定义为改良的Rodnan皮肤评分(mRSS)从基线降低>5点和≥25%。比较两组临床指标,评价托法替尼的疗效。
tofacitinib治疗后的第一个月，mRSS显著改善，mRSS的平均变化为-3.7（95％CI-5.52，-1.88；P=0.001），大于6个月时的比较[-10.0（95％CI-14.74，-5.26）vs-4.1（95％CI-7.49，-0.73），P=0.026]。Tofacitinib治疗的患者的反应时间明显短于比较者（通过对数秩检验P=0.015），在1个月和3个月时，总反应率为20％（2/10）vs0％（0/12）和60％（6/10）vs16.7％（2/12），分别。
我们的结果表明，托法替尼可能与强化常规免疫抑制剂一样有效，甚至更好，在伴有进行性皮肤厚度的难治性dcSSc患者中具有更快和更高的响应率。
To analyse the effectiveness of tofacitinib for the treatment of refractory skin thickening in dcSSc.
Data from 10 patients with dcSSc treated with tofacitinib (5 mg twice daily) were analysed. A total of 12 dcSSc patients treated with intensive conventional immunosuppressants were selected as the historical comparator group. A clinically relevant response was defined as a decrease in the modified Rodnan skin score (mRSS) of >5 points and ≥25% from baseline. Clinical indicators were compared between the two groups to evaluate the effect of tofacitinib.
The mRSS significantly improved the first month after tofacitinib treatment, with a mean change in the mRSS of -3.7 (95% CI -5.52, -1.88; P = 0.001) and greater than the comparators at 6 months [-10.0 (95% CI -14.74, -5.26) vs -4.1 (95% CI -7.49, -0.73), P = 0.026]. Tofacitinib-treated patients had a significantly shorter response time than the comparators (P = 0.015 by log-rank test), with overall response rates of 20% (2/10) vs 0% (0/12) and 60% (6/10) vs 16.7% (2/12) at 1 and 3 months, respectively.
Our results indicate that tofacitinib may be as effective as or even better than intensive conventional immunosuppressants, with a quicker and higher response rate in refractory dcSSc patients with progressive skin thickness.
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