急性早幼粒细胞白血病治疗的远期疗效

Long-term therapeutic outcome of children with acute promyelocytic leukemia

目的评估儿童急性早幼粒细胞白血病(APL)治疗的远期疗效。方法诱导治疗均应用全反式维甲酸(ATRA),同时联合化疗或同时联合三氧化二砷(As2O3)和化疗,直至骨髓形态学缓解。完全缓解(CR)后给予3～5个疗程的巩固化疗,使分子生物学缓解后再予以ATRA+As2O3+6巯基嘌呤(6MP)及氨甲喋呤(MTX)维持治疗,总疗程2年。所有患儿治疗前、治疗后每3个月及停药随访期间每3～6个月定期行骨髓形态学、染色体及PML-RARα融合基因检测。结果17例APL患儿,获CR 16例(94%),CCR 12例(70%),随访7～122个月,平均35个月。4例复发(25%)。其中2例曾获骨髓形态学和分子生物学缓解,另外2例曾获骨髓形态学缓解而分子生物学始终未缓解。结论系统性的ATRA+As2O3联合化疗治疗方案使大部分APL患儿获得长期生存,PML-RARα融合基因定期监测是判断疗效及预后的重要指标。

Objectives To analyze the long-term therapeutic outcome of patients with acute promyelocytic leukemia （APL） in children. Methods All 17 APL patients were treated with all-trans retinoic acid （ATRA） as induction therapy until achieved complete remission （CR）, together with chemotherapy or combined with arsenic induction and chemotherapy; followed by 3 - 5 courses of consolidation chemotherapy until PML-RARα fusion gene negative were proved. Then they received ATRA, As2O3, 6-MP, MTX maintenance therapy. Total courses was 2 years. All patients were examined for bone marrow morphology, karyotype and PML-RARα fusion gene before, every three months after chemotherapy, and every three to six months after treatment was completed. Karyotype and PML-RARα fusion gene were analyzed by R-banding, and RT-PCR respectively. Results Among all the 17 patients, 16 （94%） patients achieved CR, 12 （70%） patients achieved continuous CR （CCR）, The mean follow-up was 35 （7 - 122） months. Four （4） patients relapsed （25%） . Two of them showed continuous positive PML-RARα fusion gene and the other 2 patients transformed from negative into positive. Conclusions Most APL patients treated with serial therapy of combined ATRA and arsenic induction and chemotherapy can achieve a higher CCR, regularly cytogenetic detections can be regarded as an important marker for prognosis of APL.