Azacitidine in the treatment of pediatric therapy-related myelodysplastic syndrome after allogeneic hematopoietic stem cell transplantation.
Azacitidine in the treatment of pediatric therapy-related myelodysplastic syndrome after allogeneic hematopoietic stem cell transplantation.
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阿扎胞苷治疗异基因造血干细胞移植后儿科治疗相关的骨髓增生异常综合征。
DOI：
10.1097/mph.0000000000000042
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发表时间：
2014
期刊：
影响因子：
--
通讯作者：
Tamai H.
中科院分区：
文献类型：
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作者：
Inoue A;Kawakami C;Takitani K;Tamai H.
我们在此报告一个复杂核型的小儿骨髓增生异常综合征（t-MDS）的病例，在异基因造血干细胞移植（HSCT）后，以阿扎胞苷（AZA）治疗AML 1-EVI 1融合转录物作为微小残留病。患者在HSCT后41天开始AZA治疗，但未达到完全缓解。AZA治疗9个周期后，AML 1-EVI 1融合基因转录物消失，AZA治疗期间无移植物抗宿主病表现。AZA对微小残留病的预防性治疗具有可接受的安全性，似乎是预防或显著延迟HSCT后高危骨髓增生异常综合征患儿血液学复发的有效策略。治疗相关骨髓增生异常综合征或急性髓细胞白血病（t-MDS/AML）是公认的癌症治疗并发症。导致t-MDS/AML的主要因素是暴露于烷基化剂、表鬼白毒素和放射治疗。t-MDS/AML患者的结局通常较差，因为与新发疾病患者相比，其临床病程进展更快，完全缓解（CR）率更低，CR持续时间更短。一、二
We herein present a case of pediatric therapy-related myelodysplastic syndrome (t-MDS) with complex karyotype who was treated with azacitidine (AZA) for AML1-EVI1 fusion transcript as minimal residual disease after allogeneic hematopoietic stem cell transplantation (HSCT). The patient was started on AZA 41 days after the HSCT without having achieved complete remission. After 9 cycles of AZA, the AML1-EVI1 fusion transcript disappeared, and there was no manifestation of graft versus host disease during AZA treatment. Preemptive AZA treatment for minimal residual disease has an acceptable safety profile and appears to be an effective strategy for preventing or substantially delaying hematological relapse in pediatric patients with high-risk myelodysplastic syndrome after HSCT.Therapy-related myelodysplastic syndrome or acute myeloid leukemia (t-MDS/AML) is a well-recognized complication of cancer treatment. The major factors contributing to t-MDS/AML are exposure to alkylating agents, epipodophyllotoxin, and radiation therapy. Patients with t-MDS/AML generally have an inferior outcome because of the more progressive clinical course compared with patients with de novo disease, with a lower complete remission (CR) rate and a shorter duration of CR. 1, 2