大剂量阿糖胞苷巩固治疗核心结合因子相关急性髓系白血病的疗效及安全性分析

目的:探讨大剂量阿糖胞苷(HDAraC)巩固治疗核心结合因子相关急性髓系白血病(CBF-AML)的疗效和安全性。方法:回顾分析本院血液科骨髓移植病区2007年至2012年收治的CBF-AML患者应用HDAraC巩固化学治疗(化疗)的临床资料。评估患者总生存(OS)率、无病生存(DFS)率及复发率,并评价其治疗安全性,包括血液学不良反应及胃肠道、中枢神经系统等非血液学不良反应。结果:入选14例患者(男9例、女5例),中位年龄36.5(15~58)岁;AML-M2b 7例,AML-M4Eo 7例。共进行38个疗程HDAraC治疗。中位随访32.3个月,预期5年OS率为67.4%±14.0%,5年DFS率为44.6%±14.4%,5年复发率56.3%±14.4%。截止末次随访,10例患者存活,1例死于血小板减少所致颅内出血,3例死于疾病复发。7例患者复发,其中1例为复杂染色体异常,2例AML-M2b伴c-kit基因突变。29个疗程可评价不良反应:所有患者均出现Ⅳ度粒细胞减少和Ⅳ度血小板降低以及粒细胞缺乏性发热,经治疗无严重感染致死病例,1例因血小板减少致颅内出血死亡。患者均有Ⅰ~Ⅱ度胃肠道不良反应。未见中枢神经系统、皮肤等不良反应。结论:HDAraC应用于CBF-AML缓解后巩固化疗的效果和安全性令人满意,值得临床推广应用。

Outcome of consolidation chemotherapy with high dose cytarabine for core binding factor acute myeloid leukemia

Objective To assess the efficacy and safety of high-dose cytarabine （HDAraC） as consolidation chemotherapy for core binding factor acute myeloid leukemia （CBF-AML）. Methods Patients with CBF-AML in their first complete remission （CR1） after induction chemotherapy and then undergoing consolidation chemotherapy with HDAraC from 2007 to 2012 were enrolled. Overall survival （OS）, disease free survival （DFS） and relapse incidence （RI） were assessed, and therapy-related adverse events, including hematological toxicity and non-hematological toxicity such as gastrointestinal tract, central nervous system （CNS） adverse effects were analyzed. Results Of the 14 patients enrolled, there were 9 males and 5 females with a median age of 36.5 （15-58） years. Seven patients were diagnosed as AML-M2b and the other 7 as AML-M4Eo. All patients underwent 2 to 4 cycles of HDAraC chemotherapies for a total of 38 courses with a median follow-up of 32.3 months. The estimated 5-year OS, DFS and RI were 67.4%±14.0%, 44.6%±14.4%, and 56.3%±14.4%, respectively. At the last follow-up, 10 patients were alive, 1 died from intracranial hemorrhage due to thrombocytopenia and 3 died of disease relapse. Overall, 7 patients experienced relapse, of whom 2 AML-M2b patients having c-kit gene mutation, 1 having complex chromosomal aberrations and other 4 having no other chromosomal aberrations or gene mutations. All patients had grade Ⅳ myelosuppression after HDAraC chemotherapy and experienced grade IV neutropenia and thrombocytopenia, but no patient with neutropenia died of infection after appropriate treatment. All patients had grade Ⅰ-Ⅱ gastrointestinal adverse effects but no adverse reactions of skin and CNS were documented. Conclusions HDAraC is a promising and safe regimen for post-remission consolidation in patients with CBF-AML.