Department of Medicine, Beth Israel Deaconess Medical Center, Boston, MA, USA.
Division of Hematological Malignancies, Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA, USA.
Leuk Res. 2015 Feb;39(2):204-10. doi: 10.1016/j.leukres.2014.11.031. Epub 2014 Dec 12.
Clofarabine, a second-generation nucleoside analog, has clinical activity in relapsed or refractory acute myelogenous leukemia (AML) and higher-risk myelodysplastic syndromes (MDS). However, there are few data evaluating performance of clofarabine in populations of patients not enrolled in clinical trials. We reviewed outcomes for 84 patients treated with clofarabine for relapsed or refractory AML or MDS, either with clofarabine as monotherapy (n=19) or in combination with cytarabine (n=65). Using International Working Group (IWG) response criteria, the overall response rate (ORR) of all treated patients was 21%, with a complete response rate with either complete or incomplete hematopoietic recovery (CRR=CR+CRi) of 14%. For combination therapy, ORR was 22% with CRR of 18%, and monotherapy patients had an ORR of 21% with CRR of 11%. Although limited by small numbers, subgroup analysis did not reveal variation in response rates when comparing different risk factors. The 30-day mortality was 21% and median survival was 3 months; a subset of 12 patients who were able to go to transplant had an 18-month median survival. Clofarabine's efficacy in a "real-world" setting appears to be less than has been reported in clinical trials, and treatment is associated with a high early mortality rate.
氯法拉滨是一种第二代核苷类似物，在复发或难治性急性髓系白血病（AML）和高危骨髓增生异常综合征（MDS）中具有临床活性。然而，评估氯法拉滨在未入组临床试验的患者人群中的表现的数据较少。我们回顾了 84 例接受氯法拉滨治疗的复发或难治性 AML 或 MDS 患者的结局，其中氯法拉滨单药治疗（n=19）或联合阿糖胞苷治疗（n=65）。根据国际工作组（IWG）反应标准，所有治疗患者的总体反应率（ORR）为 21%，完全缓解率（CRR=CR+CRi）为 14%。对于联合治疗，ORR 为 22%，CRR 为 18%，单药治疗患者的 ORR 为 21%，CRR 为 11%。尽管受到样本量较小的限制，但亚组分析并未发现不同危险因素比较时反应率的差异。30 天死亡率为 21%，中位生存期为 3 个月；12 例能够进行移植的患者亚组的中位生存期为 18 个月。氯法拉滨在“真实世界”环境中的疗效似乎低于临床试验报告的疗效，并且治疗与高早期死亡率相关。
