分析儿童急性早幼粒细胞性白血病(acute promyelocytic leukemia, APL)的临床特点及患儿的生存情况。

回顾性总结分析了四川大学华西第二医院血液科2000年10月至2007年1月收治的31例APL患儿的临床特点和生存情况。

本组患儿平均年龄为7. 8岁，男女患儿性别比为1.38：1. 00。其主要临床表现包括出血(96.8%)、肝大(61. 3%)、发热(58.0%)、贫血(55.0%)、脾大(41. 9%)和感染(35. 0%)等。26例患儿接受正规化疗，随访时间为4〜62个月，中位随访时间为36个月。本组患儿完全缓解率(CR)为88.5%。完全缓解率高低与初诊时外周血WBC计数和年龄相关。Kaplan-Meier曲线生存率分析表明，本组患儿预期1年、3年和5年无事件生存率(event-free survival, EFS)分别为84. 6%，72. 4%和54. 8%。

出血是儿童APL最常见和显著的临床表现，发生率高达97%。经全反式维甲酸结合蒽环类药物为主的化疗方案正规治疗，本组患儿完全缓解率与国内外文献报道结果相当，但生存率(尤其是长期生存率)低于国外文献的报道结果。根据患儿年龄、初诊时外周血WBC计数和幼稚细胞比例等临床预后因素，采用个体化治疗方案，对于提高生存率具有重要指导意义。

To study the clinical features of childhood acute promyelocytic leukemia (APL) and to analyze the survival and prognostic factors.

The clinical features of 31 children with newly diagnosed APL hospitalized in the department of pediatric hematology and oncology of the West China Second University Hospital Sichuan University during October 2000 to January 2007 were analyzed retrospectively. Follow up periods were 4 to 62 months, with median of 36 months. Survival rates were calculated using Kaplan-Meier statistical method, and rate comparison by X² test.

The average age at diagnosis of our children with APL was 7. 8 years, with a slight male predominance (M : F=1. 38 : 1. 00). Common clinical presentations in decreasing order were bleeding(96. 8%), hepatomegaly(61. 3%), fever(58. 0%), anemia(55. 0%), splenomegaly(41. 9%) and infection(35. 0%). With ARTA and/or daunorubicin-based remission induction therapy, rate of complete remission (CR) was 88. 5%. 1-year, 3-year and 5-year event-free survival (EFS) rates were 84. 6%, 72. 4% and 54. 8%. Older patients(>10 years) and those with higher initial WBC count (>10× 10⁹/L) had poorer survival rates.

Bleeding occurs in the majority of our cases, and some even has DIC before chemotherapy, though with lower incidence than their adult counterparts. CR rate is similar to other study series but long-term survival rate is lower as compared to those reported by oversea clinical trials. Patient age and initial WBC count are important prognostic factors which might be useful in prognostication and treatment planning.