儿童急性早幼粒细胞白血病30例临床分析

Clinical Analysis of 30 Children with Acute Promyelocytic Leukemia

目的探讨应用儿童急性早幼粒细胞白血病(acute promyelocytic leukemia,APL)临床路径(2010年版)治疗APL患儿的疗效及预后,为APL患儿的治疗提供参考。方法回顾性分析2010年10月1日至2017年9月1日30例初诊为APL患儿的临床资料。根据患儿入院时外周血WBC、PLT计数分为低危组(WBC≤10×109 L^-1,PLT>40×109 L^-1, n =6)、中危组(WBC≤10×109 L^-1,PLT≤40×109 L^-1, n =12)、高危组(WBC> 10×109 L^-1, n =12),比较3组的发病情况、疗效及预后。结果 30例患儿发病初期多以皮肤黏膜出血、面色苍白和发热为临床表现。弥散性血管内凝血(DIC)发生率为50.0%(15/30),患儿危险度越高,其发生率越高(χ2 =6.460, P =0.034)。诱导治疗中,高危组2例因DIC致颅内出血死亡,另28例患儿诱导化疗后(5周)血液学完全缓解27例(96.4%)。维持治疗中有1例复发。随访中位时间为40个月(0.2~70.0个月),截至2017年9月1日28例患儿存活。高危组5年总生存率、无事件生存率与低、中危组比较,差异无统计学意义(χ2 =3.13、 P =0.209,χ2 =5.34、 P =0.679)。结论 APL的治愈率较高,但高危组早期病死率及复发率仍较高。对DIC及出凝血障碍进行早期干预,制定合理方案减少早期病死率、复发率仍是治疗APL的关键。

Objective To analyze the efficacy and prognosis of clinical pathway(2010) for primary acute promyelocytic leukemia(APL) in children,and to provide a reference for the treatment of APL. Methods A retrospective analysis was performed on the clinical data of 30 children initially diagnosed with APL between October 1,2010 and September 1,2017.According to WBC and PLT count on admission,these children were divided into low-risk group(WBC≤10× 109 L^-1,PLT>40×109 L^-1, n =6),medium-risk group(WBC≤10×109 L^-1,PLT≤40×109 L^-1, n =12) and high-risk group(WBC>10×109 L^-1, n =12).The disease condition,efficacy and prognosis were compared among the three groups. Results The clinical manifestations were mostly mucocutaneous hemorrhage,pallor and fever.The incidence of disseminated intravascular coagulation(DIC) was 50.0%(15/30).Furthermore,the incidence was positively associated with the risk(χ2 =6.460, P =0.034).In the induction treatment,2 children in high-risk group died due to intracranial hemorrhage caused by DIC.Among the other 28 cases,27(96.4%) achieved complete hematologic remission after induction chemotherapy for 5 weeks.One child had recurrence during maintenance treatment.Twenty-eight children survived as of September 1,2017,with a median follow-up period of 40 months(0.2-70.0 months).The 5-year overall survival rate and event-free survival rate in high-risk group were not significantly different from those in low-risk(χ2 =3.13, P =0.209) and medium-risk groups(χ2 =5.34, P =0.069). Conclusion APL has a higher cure rate.However,the early mortality and recurrence rate are still higher in high-risk group.Early intervention should be performed for DIC and coagulation disorders and reasonable plans should be developed to reduce the early mortality and recurrence rate in the treatment of APL.