A randomized phase 3 study of tipifarnib compared with best supportive care, including hydroxyurea, in the treatment of newly diagnosed acute myeloid leukemia in patients 70 years or older
A randomized phase 3 study of tipifarnib compared with best supportive care, including hydroxyurea, in the treatment of newly diagnosed acute myeloid leukemia in patients 70 years or older
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DOI：
10.1182/blood-2009-01-198093
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发表时间：
2009-08-06
期刊：
影响因子：
20.3
通讯作者：
Howes, Angela J.
中科院分区：
文献类型：
--
作者：
Harousseau, Jean-Luc;Martinelli, Giovanni;Howes, Angela J.
这项3期、多中心、开放标签研究评估了替吡法尼与最佳支持治疗（BSC）（包括羟基脲）相比作为新诊断、新发或继发性急性髓性白血病老年患者（>= 70岁）一线治疗的疗效和安全性。共入组457例患者，其中24%的患者年龄≥ 80岁。替吡法尼600 mg口服，每日两次，连续给药前21天，28天为一个周期。主要终点是总生存期。替吡法尼组的中位生存期为107天，BSC组为109天。总生存期的风险比（替吡法尼vs BSC）为1.02（分层对数秩检验的P值为0.843）。本研究中替吡法尼的完全缓解率（8%）低于先前观察到的完全缓解率，但中位持续时间相似，均为8个月。最常见的3级或4级不良事件是两组中的血细胞减少症，在替吡法尼组中观察到的感染（39%对33%）和发热性中性粒细胞减少症（16%对10%）。本试验在www.clinicaltrials.gov上注册为#NCT00093990。（血。2009; 114：1166-1173）
This phase 3, multicenter, open-label study evaluated the efficacy and safety of tipifarnib compared with best supportive care (BSC), including hydroxyurea, as first-line therapy in elderly patients (>= 70 years) with newly diagnosed, de novo, or secondary acute myeloid leukemia. A total of 457 patients were enrolled with 24% 80 years of age or older. Tipifarnib 600 mg orally twice a day was administered for the first 21 consecutive days, in 28-day cycles. The primary end-point was overall survival. The median survival was 107 days for the tipifarnib arm and 109 days for the BSC arm. The hazard ratio ( tipifarnib vs BSC) for overall survival was 1.02 ( P value by stratified log-rank test, .843). The complete response rate for tipifarnib in this study (8%) was lower than that observed previously, but with a similar median duration of 8 months. The most frequent grade 3 or 4 adverse events were cytopenias in both arms, slightly more infections (39% vs 33%), and febrile neutropenia (16% vs 10%) seen in the tipifarnib arm. The results of this randomized study showed that tipifarnib treatment did not result in an increased survival compared with BSC, including hydroxyurea. This trial was registered at www.clinicaltrials.gov as #NCT00093990. (Blood. 2009; 114: 1166-1173)