单中心MAE方案诱导治疗儿童急性髓系白血病的合并症及疗效分析

Mitoxantrone-cytarabine-etoposide induction therapy in children with acute myeloid leukemia: a single-center study of complications and clinical outcomes

摘要:

目的 回顾性分析米托蒽醌、阿糖胞苷联合依托泊苷（MAE）方案诱导治疗初诊儿童急性髓系白血病（AML）的合并症发生情况及疗效。方法 170例AML患儿采用MAE方案诱导治疗，分析诱导治疗后的合并症及缓解率。结果 170例中男女比例为1.33：1，年龄7.4（1~15）岁，诊断时WBC 29.52（0.77~351）×10⁹/L，其中M0 2例、M2 24例、M4 2例、M5 48例、M6 3例、M7 7例，AML伴有t（8；21）（q22；q22）69例，AML伴有inv（16）（p13.1q22）或t（16；16）（p13.1；q22）15例。最常见的合并症为感染，占92.9%，其中无明确感染灶的粒缺伴发热占13.9%（22/158），有明确感染病灶（包括血流感染）的占86.1%（136/158）；其他合并症包括非感染性腹泻、出血、药物性肝炎等。治疗相关死亡10例，其中严重感染8例、多脏器功能衰竭1例、呼吸衰竭1例。156例进行了缓解率评估，完全缓解率85.3%、部分缓解率4.5%、未缓解率10.3%。结论 MAE方案治疗儿童AML的1疗程诱导缓解率较好，合并症及治疗相关死亡原因以感染为主。

Abstract:

Objective To investigate the complications and clinical outcome of children with acute myeloid leukemia (AML) undergoing mitoxantrone-cytarabine-etoposide (MAE) induction therapy. Methods A total of 170 children with AML were given MAE induction therapy, and the complications and remission rate were analyzed after treatment. Results The male/female ratio was 1.33:1 and the mean age was 7.4 years (range 1-15 years). Leukocyte count at diagnosis was 29.52×10⁹/L[range (0.77-351)×10⁹/L]. Of all children, 2 had M0-AML, 24 had M2-AML, 2 had M4-AML, 48 had M5-AML, 3 had M6-AML, 7 had M7-AML, 69 had AML with t(8;21)(q22;q22), and 15 had AML with inv(16)(p13.1q22) or t(16;16)(p13.1;q22). The most common complication was infection (158/170, 92.9%). Among these 158 patients, 22 (13.9%) had agranulocytosis with pyrexia (with no definite focus of infection), and 136 (86.1%) had definite focus of infection (including bloodstream infection). Other complications included non-infectious diarrhea, bleeding, and drug-induced hepatitis. Treatment-related mortality was observed in 10 children, among whom 8 had severe infection, 1 had multiple organ failure, and 1 had respiratory failure. Remission rate was evaluated for 156 children and the results showed a complete remission rate of 85.3%, a partial remission rate of 4.5%, and a nonremission rate of 10.3%. Conclusions Induction therapy with the MAE regimen helps to achieve a good remission rate in children with AML after one course of treatment. Infection is the main complication and a major cause of treatmentrelated mortality.