维甲酸及蒽环类药物联合治疗急性早幼粒细胞性白血病37例

Treatment with combined all-trans retinoic acid and anthracycline of 37 children with acute promyelocytic leukemia

目的 分析儿童急性早幼粒细胞性白血病（APL）的临床特点及维甲酸及蒽环类药物联合治疗方案安全性及疗效分析.方法 回顾性分析首都医科大学附属北京儿童医院血液病中心2005年1月至2009年2月收治的37例APL患儿的临床特点及生存情况.结果 APL低危患者占30％（ 11/37）,中危患者占43％ （16/37）,高危患者占27％ （10/37）.60％ （22/37）患者发生不同程度的弥漫性血管内凝血,根据危险度不同弥漫性血管内凝血的发生率也逐渐增加（P =0.029）,分别是低危组27％（ 3/11）,中危组69％ （11/16）,高危组80％ （8/10）.3例患者死亡（8％）.2例患者发生维甲酸综合征（6％）.完全缓解率为83.7％,各危险组缓解时间无差异（P=0.612）.使用去甲氧柔红霉素及柔红霉素两组的缓解时间无差异（P =0.628）.Kaplan-Meier曲线生存率分析表明,本组患儿2年无事件生存率为81％,低、中、高危险度组2年无事件生存率分别100％、81％、60％.结论 全反式维甲酸结合蒽环类药物为主的化疗方案完全缓解率高,生存率高,毒副作用小.弥漫性血管内凝血是儿童APL诱导缓解治疗的主要合并症.采用个体化治疗方案,对于提高缓解率及生存率有重要意义.

Objective To study the clinical features of childhood acute promyelocytic leukemia （APL） and to analyze the survival and prognostic factors and efficacy and safety of combined treatment with all-trans retinoic acid （ATRA） and anthracycline.Method The clinical features of 37 children with newly diagnosed APL hospitalized in our center during January 2005 to February 2009 were retrospectively analyzed.Result Thirty percent of patients were at low risk,43% patients were at intermediate risk,27%patients were at high risk.Sixty percent of patients had DIC.Retinoic acid syndrome （RAS） was present in 2 patients （6%）.Death during induction occurred in 3 patients （8%）.Complete remission （CR） was achieved in 83.7% of patients.The patients in high risk group had higher risk than those in intermediate and low risk group （ P =0.029）.The time to achieve CR was not significantly different （ P =0.612）.Idarubicin had no advantage compared with daunorubicin in time to achieve CR（P =0.628）.Survival rates were calculated using Kaplan-Meier statistical method,and 2 years event-free survival （EFS） rate was 81%,the 2-year EFS rate was 100% for low-risk group,81% for intermediate-risk group,and 60% for high-risk group.Conclusion Using combined chemotherapy with ATRA and anthracyclines had the following advantages：high CR rate,high long-time survival rate and low side effect.DIC remained the main complication among patients receiving induction treatment. Initial WBC count and platelet count are important prognostic factors which might be useful in prognostication and treatment planning.