地西他滨联合改良CAG及外周造血干细胞移植治疗儿童难治急性髓系白血病1例报告

Decitabine combined improved CAG chemotherapy and peripheral hematopoietic stem celltransplantation in treatment for child with high-risk and refractory acute myeloid leukemia:A case report

目的报道1例高危难治急性髓系白血病(acute myeloid leukemia,AML)患儿采用地西他滨+改良CAG方案(阿柔比星+阿糖胞苷+聚乙二醇化重组人粒细胞集落刺激因子)化疗及单倍体相合外周造血干细胞移植后,获完全缓解。方法对解放军总医院第一医学中心儿科2019年2月收治的1例难治高危AML患儿的临床资料及治疗方案进行回顾,并复习相关文献。结果患儿男,9岁,新疆乌鲁木齐人,以发热、下肢皮疹伴肿痛、两系减低起病,确诊为急性髓系白血病M4b型,伴DEK-CAN融合基因阳性。先后行CAG、2次DAE(柔红霉素+阿糖胞苷+依托泊苷)诱导化疗,骨髓未获缓解。尝试行地西他滨+改良CAG方案,获完全缓解,化疗过程中出现Ⅳ度骨髓抑制,粒细胞缺乏期无发热,未见胃肠道、心、肝、肾损害等不良反应。后行亲缘单倍体相合外周造血干细胞移植,目前无病生存1年余,仍在随访中。结论地西他滨对儿童AML的治疗具有潜在价值,联合改良CAG方案作为儿童难治AML诱导化疗的备选方案,可能提高临床缓解率,值得进一步研究。

Objective To report a child who suffered high-risk and refractory acute myeloid leukemia(AML)achieved complete remission by treating with decitabine combined improved CAG chemotherapy and haploid-identical peripheral hematopoietic stem cell transplantation.Methods Clinical data about a high-risk and refractory AML child in the First Medical Center of Chinese PLA General Hospital in February 2019 were analyzed and related literatures were reviewed.Results A 9-year-old boy from Urumchi in Xinjiang Uyghur Autonomous Region,whose main clinical symptoms included fever,skin rash of lower limbs accompanied with swelling and pain,lower hemoglobin and thrombocytopenia,was diagnosed as acute myeloid leukemia(M4b)with DEK-CAN fusion gene and classified into the high-risk group.The bone marrow did not achieve remission after CAG and two cycles of CAE chemotherapy.Decitabine plus improved CAG regimen was tried and complete remission was obtained.During chemotherapy,gradeⅣbone marrow suppression was observed,and there were no fever,no damage of heart,liver,kidney and other adverse reactions in the period of granulocytopenia.Then,the haploid-identical peripheral hematopoietic stem cell transplantation was performed.At present,the child had been disease-free for more than one year and was still under follow-up.Conclusion Decitabine has potential value in the treatment of childhood AML.As an alternative,decitabine plus improved CAG chemotherapy may improve the clinical remission rate for refractory AML in children,which is a promising therapeutic method and worthy of deeply study.