Pomalidomide in Patients with Interstitial Lung Disease due to Systemic Sclerosis: A Phase II, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study
Pomalidomide in Patients with Interstitial Lung Disease due to Systemic Sclerosis: A Phase II, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study
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DOI：
10.3899/jrheum.161040
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发表时间：
2018-03-01
影响因子：
3.9
通讯作者：
Hough, Douglas R.
中科院分区：
文献类型：
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作者：
Hsu, Vivien M.;Denton, Christopher P.;Hough, Douglas R.
目标。评价泊马度胺（POM）对系统性硬化症（SSc）间质性肺疾病患者52周治疗期间用力肺活量（FVC）、改良罗德曼皮肤评分（mRSS）和胃肠道（GI）症状的安全性和有效性。23例诊断为SSc的成年患者随机分为1:1的POM：安慰剂（PBO）。与基线相比，第52周预测FVC%的平均变化-5.2和-2.8；mRSS -2.7和-3.7；加州大学洛杉矶分校硬皮病临床试验协会胃肠道（SCTC GIT 2.0）评分分别为0.1和0.0，分别为POM和PBO。在52周时，这3个主要终点均未达到统计学意义。由于招募方面的挑战，受试者招募提前终止。在中期分析中，该研究没有达到第52周的主要终点。因此，决定终止所有研究阶段。POM通常耐受性良好，其不良事件概况与POM在其他疾病中的已知安全性和耐受性概况一致。研究结果既非正面也非负面，因为纳入的受试者太少，无法得出有意义的结论。
Objective. To evaluate the safety and efficacy of pomalidomide (POM) on forced vital capacity (FVC), modified Rodnan skin score (mRSS), and gastrointestinal (GI) symptomatology over 52 weeks of treatment in patients with interstitial lung disease due to systemic sclerosis (SSc).Methods. Twenty-three adult patients diagnosed with SSc were randomized 1:1 POM:placebo (PBO).Results. Mean change at Week 52 from baseline in predicted FVC% -5.2 and -2.8; mRSS -2.7 and -3.7; and UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tract (SCTC GIT 2.0) score 0.1 and 0.0, with POM and PBO, respectively. Statistical significance was not achieved for any of these 3 primary endpoints at 52 weeks.Conclusion. Because of recruitment challenges, subject enrollment was discontinued early. In an interim analysis, the study did not meet its Week 52 primary endpoints. Therefore, a decision was made to terminate all study phases. POM was generally well tolerated, with an adverse event profile consistent with the known safety and tolerability profile of POM in other diseases. Study results were neither positive nor negative because too few subjects were enrolled to make meaningful conclusions.