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骨髓增生异常综合征和急性髓性白血病作为获得性再生障碍性贫血患儿的晚期克隆性并发症。

Myelodysplastic syndrome and acute myelogenous leukemia as a late clonal complication in children with acquired aplastic anemia.

作者信息

Ohara A, Kojima S, Hamajima N, Tsuchida M, Imashuku S, Ohta S, Sasaki H, Okamura J, Sugita K, Kigasawa H, Kiriyama Y, Akatsuka J, Tsukimoto I

机构信息

Aplastic Anemia Committee of the Japanese Society of Pediatric Hematology.

出版信息

Blood. 1997 Aug 1;90(3):1009-13.

PMID:9242530
Abstract

The improved outcome of acquired aplastic anemia (AA) has revealed later complications, such as myelodysplastic syndrome (MDS) and acute myelogenous leukemia (AML). We retrospectively analyzed 167 children with severe acquired AA. Eleven of 50 children treated with cyclosporin (CSA) and recombinant human granulocyte colony-stimulating factor (rhG-CSF) developed MDS/AML; 8 of these were within 36 months of the diagnosis of AA, much earlier than previous reports. Six of the 11 children received rhG-CSF exceeding 10 microg/kg/d, and 9 received rhG-CSF therapy for over 1 year. Ten children showed monosomy 7 at diagnosis of MDS. All of the 11 children were administered both CSA and rhG-CSF. There was no development of MDS/AML among 41 children treated with either CSA or rhG-CSF or among 48 children who underwent bone marrow transplantation. A well-controlled clinical trial is warranted to determine whether therapeutic modalities affect the development of MDS/AML in children with severe acquired AA.

摘要

获得性再生障碍性贫血(AA)治疗效果的改善揭示了一些后期并发症,如骨髓增生异常综合征(MDS)和急性髓系白血病(AML)。我们回顾性分析了167例重度获得性AA患儿。50例接受环孢素(CSA)和重组人粒细胞集落刺激因子(rhG-CSF)治疗的患儿中有11例发生了MDS/AML;其中8例在AA诊断后的36个月内发病,比之前的报道要早得多。11例患儿中有6例接受的rhG-CSF剂量超过10μg/kg/d,9例接受rhG-CSF治疗超过1年。10例患儿在MDS诊断时显示7号染色体单体。11例患儿均接受了CSA和rhG-CSF治疗。在41例接受CSA或rhG-CSF治疗的患儿以及48例接受骨髓移植的患儿中均未发生MDS/AML。有必要进行一项严格控制的临床试验,以确定治疗方式是否会影响重度获得性AA患儿发生MDS/AML。

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