【摘要】 目的 分析尼达尼布在中国系统性硬化症相关间质性肺病(SSc-ILD)患者中的有效性与安全性。方法 SENSCIS是一项多国家、多中心、前瞻性、随机双盲、安慰剂对照Ⅲ期临床研究，本研究对其中22例中国患者进行亚组分析。入选患者以1∶1的比例随机接受尼达尼布(口服150 mg,一日两次)或安慰剂治疗，治疗时长为52至100周。主要终点为52周时用力肺活量(FVC,mL)年下降率，关键次要终点为第52周时改良版Rodnan皮肤评分(mRSS)自基线的绝对值改变，及第52周时圣乔治呼吸问卷(SGRQ)总分自基线的绝对值改变。结果 22例中国患者(尼达尼布组9例、安慰剂组13例)中，20例为女性。尼达尼布和安慰剂组患者的平均年龄分别为(50.8±17.3)岁和(51.5±13.3)岁。尼达尼布组52周时经校正的FVC平均年下降率低于安慰剂组[分别为(70.6±79.2)mL/年和(-112.2±62.2)mL/年，校正差异(182.77±100.62)mL/年，95%置信区间(CI-14.90,380.45)]。最常见的不良反应是腹泻。结论 在SENSCIS研究中国亚组分析中，尼达尼布组患者52周时FVC下降率的减缓趋势与整体人群一致。中国患者最常见的不良事件为腹泻，未发现新的安全性问题。更多还原

【Abstract】 Objective To fevaluate the efficacy and safety of nintedanib in Chinese systemic sclerosis patients with interstitial lung disease(SSc-ILD). Methods SENSCIS is a multinational, multicenter, prospective, randomized, double-blind, placebo-controlled, phase Ⅲ trial. We conducted a subgroup analysis of 22 Chinese patients in the SENSCIS study.Eligible patients were randomly assigned in 1∶1 ratio, to receive nintedanib(150 mg, twice daily) or placebo for 52-100 weeks. The primary endpoint was the annual rate of decline in forced vital capacity(FVC,mL) over 52 weeks. The key secondary endpoints were the absolute change from baseline in the modified Rodnan skin score(mRSS) and the absolute change from baseline in the St. George’s Respiratory Questionnaire(SGRQ) total score at week 52.Results A total of 22 Chinese patients were analyzed(nintedanib: 9 patients; placebo: 13 patients),and 20 patients were females. The mean age of patients in the nintedanib and placebo group were(50.8±17.3) years and(51.5±13.3) years, respectively. The adjusted mean annual rate of decline in FVC over 52 weeks was(70.6±79.2) mL/year in nintedanib group and(-112.2±62.2) mL/year in placebo group [adjusted difference(182.77±100.62) mL/year, 95% confidence interval(-14.90,380.45)]. Diarrhea was the most common adverse event over 52 weeks. Conclusions The trend of annual rate of decline in FVC over 52 weeks in Chinese population is consistent with that in the global population. The most common adverse event in Chinese population is diarrhea, and no new safety events are detected.更多还原