Adaptive randomized study of idarubicin and cytarabine versus troxacitabine and cytarabine versus troxacitabine and idarubicin in untreated patients 50 years or older with adverse karyotype acute myeloid leukemia
Adaptive randomized study of idarubicin and cytarabine versus troxacitabine and cytarabine versus troxacitabine and idarubicin in untreated patients 50 years or older with adverse karyotype acute myeloid leukemia
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DOI：
10.1200/jco.2003.11.016
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发表时间：
2003-05-01
影响因子：
45.3
通讯作者：
Estey, EH
中科院分区：
文献类型：
--
作者：
Giles, FJ;Kantarjian, HM;Estey, EH
目的：曲沙他滨作为单一药物或与阿糖胞苷(Ara-C)或伊达比星联合治疗难治性髓系白血病有效。对50岁或以上未经治疗、核型不良的急性髓系白血病(AML)患者进行了一项前瞻性随机研究，以评估以曲沙他滨为基础的诱导治疗方案。患者和方法：患者随机接受伊达比星和阿糖胞苷(IA)、阿糖胞苷(TA)和曲沙他滨和伊达比星(TI)治疗。采用贝叶斯设计，过继随机分配患者接受治疗。因此，尽管最初随机分为IA、TA或TI的机会均等，但成功率较高的治疗组逐渐获得了更大比例的患者。结果：34名患者接受了治疗。在5例患者后停止了对TI的随机化，在11例患者后停止了对TA的随机化。将成功定义为开始治疗后49天内发生的完全缓解(CR)，IA组成功率为55%(18例患者中有10例)，TA组为27%(11例患者中3例)，TL组为0%(5例患者中0例)。由于3个CRS发生在第49天之后，最终CR率分别为：IA 55%(10/18例)，TA 45%(5/11例)，TL 20%(5例/5例)。TA低于IA的概率为70%，TA的CR率比IA高20%的概率为5%。三种方案的存活率均相当。结论：在既往未经治疗的不良核型AML的老年患者中，曲沙他滨联合用药均不优于IA。
Purpose: Troxacitabine has activity in refractory myeloid leukemia, either as a single agent or when combined with cytarabine (ara-C) or with idarubicin. A prospective, randomized study was conducted in patients aged 50 years or older with untreated, adverse karyotype, acute myeloid leukemia (AML) to assess troxacitabine-based regimes as induction therapy.Patients and Methods: Patients were randomized to receive idarubicin and ara-C (IA) versus troxacitabine and ara-C (TA) versus troxacitabine and idarubicin (TI). A Bayesian design was used to adoptively randomly assign patients to treatment. Thus, although there was initially an equal chance for randomization to IA, TA, or TI, treatment arms with a higher success rate progressively received a greater proportion of patients.Results: Thirty-four patients were treated. Randomization to TI stopped after five patients and randomization to TA stopped after 11 patients. Defining success as complete remission (CR) that occurred within 49 days of starting treatment, success rates were 55% (10 of 18 patients) with IA, 27% (three of 11 patients) with TA, and 0% (zero of five patients) with Tl. Because three CRs occurred after day 49, final CR rates were 55% (10 of 18 patients) with IA, 45% (five of 11 patients) with TA, and 20% (one of five patients) with Tl. The probability that TA was inferior to IA was 70%, with a 5% probability that TA would have a 20% higher CR rate than IA. Survival was equivalent with all three regimens.Conclusion: Neither troxacitabine combination was superior to IA in elderly patients with previously untreated adverse karyotype AML.