Zhang Yingjin, Xue Changwen, Wu Chao, Yang Wenyu, Zou Yao, Guo Ye, Chen Yumei, Chen Xiaojuan, Zhu Xiaofan, Zhang Li
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Blood Sci. 2025 Apr 7;7(2):e00225. doi: 10.1097/BS9.0000000000000225. eCollection 2025 Jun.
The use of cytarabine (Ara-C) in treating acute promyelocytic leukemia (APL) is controversial. This study was conducted to demonstrate the effect of treatment with or without Ara-C on long-term event-free survival (EFS) or overall survival (OS). All patients received all-trans retinoic acid (ATRA) + arsenic trioxide (ATO) induction therapy, followed by the course of idarubicin (IDA) and ATO, then were randomly allocated to 2 groups for consolidation therapy, with patients in the daunorubicin (DNR) group received DNR, in the DNR + Ara-C (DA) group received DNR + Ara-C. Maintenance therapy consisted of oral ATRA, 6-mercaptopurine, and methotrexate for 1.5 years. Thirty patients in DA group and 35 patients in DNR group, all achieved complete remission. At follow-up, there was 1 death and 3 relapses in DNR group, compared to none in DA group. There was no statistically significant difference in EFS ( = 0.140) and OS ( = 0.398) between 2 groups, with EFS being 100% in DA group and 91.4% ± 0.047 in DNR group, and OS being 100% in DA group and 97.1% ± 0.028 in DNR group. Our study found no prognostic significance of Ara-C, this may be related to the small sample size. We still recommend the addition of Ara-C during treatment, which has a more positive impact on early remission and late prognosis of patients.
阿糖胞苷（Ara-C）用于治疗急性早幼粒细胞白血病（APL）存在争议。本研究旨在证明使用或不使用Ara-C治疗对长期无事件生存期（EFS）或总生存期（OS）的影响。所有患者均接受全反式维甲酸（ATRA）+三氧化二砷（ATO）诱导治疗，随后进行伊达比星（IDA）和ATO疗程，然后随机分为2组进行巩固治疗，柔红霉素（DNR）组患者接受DNR，DNR+阿糖胞苷（DA）组患者接受DNR+阿糖胞苷。维持治疗包括口服ATRA、6-巯基嘌呤和甲氨蝶呤，为期1.5年。DA组30例患者和DNR组35例患者均实现完全缓解。随访时，DNR组有1例死亡和3例复发，而DA组无。两组之间的EFS（=0.140）和OS（=0.398）无统计学显著差异，DA组的EFS为100%，DNR组为91.4%±0.047，DA组的OS为100%，DNR组为97.1%±0.028。我们的研究发现Ara-C无预后意义，这可能与样本量小有关。我们仍建议在治疗期间加用Ara-C，这对患者的早期缓解和晚期预后有更积极的影响。
