Hematologic Response to Vorinostat Treatment in Relapsed Myeloid Leukemia of Down Syndrome

Hematologic Response to Vorinostat Treatment in Relapsed Myeloid Leukemia of Down Syndrome
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唐氏综合症复发性粒细胞白血病伏立诺他治疗的血液学反应

DOI:
10.1002/pbc.26062
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发表时间:
2016
影响因子:
3.2
通讯作者:
Klusmann JH
Klusmann JH
中科院分区:
医学3区
文献类型:
--
作者:
Scheer C;Kratz C;Witt O;Creutzig U;Reinhardt D;Klusmann JH

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患有唐氏综合症的儿童患骨髓性白血病(ML-DS)的风险很高。尽管预后良好,但 ML-DS 儿童尤其会遭受严重的治疗相关毒性,而且复发性 ML-DS 的治愈率非常低。在此,我们报告了一名 ML-DS 儿童在第二次复发后接受组蛋白脱乙酰酶 (HDAC) 抑制剂伏立诺他(辛二酰苯胺异羟肟酸)治疗的临床病程。该孩子之前曾接受过常规化疗和干细胞移植,但表现出显着的临床和血液学反应。因此,HDAC抑制剂可能代表一类治疗ML-DS的有效药物。
Children with Down syndrome are at high risk to develop myeloid leukemia (ML‐DS). Despite their excellent prognosis, children with ML‐DS particularly suffer from severe therapy‐related toxicities and for relapsed ML‐DS the cure rates are very poor. Here we report the clinical course of one child with ML‐DS treated with the histone deacetylase (HDAC) inhibitor vorinostat (suberoylanilide hydroxamic acid) after second relapse. The child had previously received conventional chemotherapy and stem cell transplantation, yet showed a remarkable clinical and hematologic response. Thus, HDAC inhibitor may represent an effective class of drugs for the treatment of ML‐DS.
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