全反式维甲酸联合三氧化二砷治疗初发急性早幼粒细胞白血病的近期疗效观察

目的　观察全反式维甲酸 (ATRA)联合三氧化二砷 (As2 O3 )治疗初发急性早幼粒细胞白血病 (APL)的完全缓解 (CR)率和融合基因PML RARα转阴情况。方法　ATRA 2 5mg·m- 2 ·d- 1 、As2 O30 .16mg·kg- 1 ·d- 1 联合治疗初发APL直至CR。根据外周血白细胞计数、维甲酸综合征以及肝功能变化调整ATRA和As2 O3 的剂量。观察CR率、获得CR和融合基因PML RARα转阴所需的时间、不良反应及近期缓解时间。结果　 31例初发APL患者早期死亡 2例 ,2 9例获CR ,CR率 93.5 %,获得CR的平均时间为 (2 5 .1± 3.9)d。 6 6 .5 %患者在治疗开始后出现白细胞升高 ,6 5 .5 %出现肝功能异常 ,多在As2 O3减量或停用后 1周内恢复。所有患者初发时均为PML RARα阳性 ,CR时 10 .3%转阴 ,巩固治疗后检测的 13例中 10例 (77.0 %)转阴。至今 2 9例获CR的患者仍处CR状态 (1～ 8个月 )。结论　ATRA联合As2 O3 治疗初发APL疗效好 ,不良反应少。长期CR时间需要进一步观察。

Clinical observation of the short-term efficacy of the treatment with combination of all-trans retinoic acid (ATRA) and arsenic trioxide (As2O3) in newly diagnosed acute promyelocytic leukemia (APL)

OBJECTIVE: To study whether all-trans retinoic acid (ATRA) combined with arsenic trioxide (As(2)O(3)) in acute promyelocytic leukemia (APL) treatment could further improve the clinical and molecular remission rate. METHOD: Thirty one newly-diagnosed APL patients of whom 15 were males, 16 females and median age 35.4 years entered into the study. They were treated with ATRA 25 mg x m(-2) x d(-1) combined with As(2)O(3) 0.16 mg x kg(-1) x d(-1) until complete remission (CR). The doses were adjusted according to white blood cell (WBC) counts, occurrence of RA syndrome and the status of liver function. CR rate, time of reaching clinical and molecular remission and side effects were observed. RESULT: Two patients died 2 approximately 3 days after the treatment due to intracranial hemorrhage, and 29 (93.5%) achieved CR. The average time for achieving CR was 25.1 +/- 3.9 days. Hyperleukocytosis emerged in 66.5% and hepatic damages in 65.5% of the patients, they were ameliorated within one week after reduction of the As(2)O(3) dose or its suspension. The PML/RAR alpha fusion gene that was positive in all 29 patients before treatment turned negative only in 3 cases (10.3%) after obtaining CR (CR1) and in 10/13 cases (77%) after consolidation treatment. Up to now (1-8 months follow-up), all 29 patients remain in CR1. CONCLUSION: ATRA combined with As(2)O(3) in de novo APL treatment can yield a high CR rate without intolerable side effects. Long term effect needs further observation.