A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q
A randomized phase 3 study of lenalidomide versus placebo in RBC transfusion-dependent patients with Low-/Intermediate-1-risk myelodysplastic syndromes with del5q
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DOI：
10.1182/blood-2011-01-330126
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发表时间：
2011-10-06
期刊：
影响因子：
20.3
通讯作者：
Hellstrom-Lindberg, Eva
中科院分区：
文献类型：
--
作者：
Fenaux, Pierre;Giagounidis, Aristoteles;Hellstrom-Lindberg, Eva
这项III期、随机、双盲研究评估了来那度胺在205例红细胞（RBC）输注依赖型国际预后评分系统低/中1风险del 5 q31骨髓增生异常综合征患者中的疗效和安全性。患者在28天周期的第1-21天接受来那度胺10 mg/天（n = 69）或在第1-28天接受5 mg/天（n = 69）;或安慰剂（n = 67）。16周后允许交叉至来那度胺或更高剂量。与安慰剂组相比，来那度胺10 mg组和5 mg组更多的患者达到RBC-输血独立性（TI）≥ 26周（主要终点）（56.1%和42.6% vs 5.9%;均P <0.001）。未达到RBC-TI的中位持续时间（中位随访时间为1.55年），在未进展为急性髓性白血病（AML）的患者中，60%至67%的缓解仍在持续。细胞遗传学缓解率分别为50.0%（10 mg）和25.0%（5 mg; P = 0.066）。合并来那度胺组，3年总生存率和AML风险分别为56.5%和25.1%。RBC-TI ≥ 8周与死亡和AML进展或死亡的相对风险分别降低47%和42%相关（P = .021和.048）。安全性特征与既往报告一致。来那度胺在低/中1风险del 5 q骨髓增生异常综合征输血依赖患者中是有益的，并且具有可接受的安全性特征。该试验在www.clinicaltrials.gov上注册为#NCT00179621。（血。2011; 118（14）：3765-3776）
This phase 3, randomized, double-blind study assessed the efficacy and safety of lenalidomide in 205 red blood cell (RBC) transfusion-dependent patients with International Prognostic Scoring System Low-/Intermediate-1-risk del5q31 myelodysplastic syndromes. Patients received lenalidomide 10 mg/day on days 1-21 (n = 69) or 5 mg/day on days 1-28 (n = 69) of 28-day cycles; or placebo (n = 67). Crossover to lenalidomide or higher dose was allowed after 16 weeks. More patients in the lenalidomide 10- and 5-mg groups achieved RBC-transfusion independence (TI) for >= 26 weeks (primary endpoint) versus placebo (56.1% and 42.6% vs 5.9%; both P < .001). Median duration of RBC-TI was not reached (median follow-up, 1.55 years), with 60% to 67% of responses ongoing in patients without progression to acute myeloid leukemia (AML). Cytogenetic response rates were 50.0% (10 mg) versus 25.0% (5 mg; P = .066). For the lenalidomide groups combined, 3-year overall survival and AML risk were 56.5% and 25.1%, respectively. RBC-TI for >= 8 weeks was associated with 47% and 42% reductions in the relative risks of death and AML progression or death, respectively (P = .021 and .048). The safety profile was consistent with previous reports. Lenalidomide is beneficial and has an acceptable safety profile in transfusion-dependent patients with Low-/Intermediate-1-risk del5q myelodysplastic syndrome. This trial was registered at www.clinicaltrials.gov as #NCT00179621. (Blood. 2011; 118(14):3765-3776)