Morita Yasuyoshi
Division of Hematology and Rheumatology, Department of Internal Medicine, Kindai University Faculty of Medicine.
Rinsho Ketsueki. 2018;59(10):2050-2057. doi: 10.11406/rinketsu.59.2050.
Myelodysplastic syndromes (MDS) are clonal hematopoietic stem cell disorders characterized by cytopenia and leukemic transformation. Allogeneic hematopoietic stem cell transplantation is the only potential curative therapy for MDS; however, the first treatment option for transfusion-independent patients with low-risk MDS remains limited. Recently, lenalidomide has become available for patients with low-risk MDS and symptomatic anemia with del (5q) (5q-syndrome), darbepoetin (DA) for those with anemia without del (5q), and azacitidine for those with DA-resistant anemia or anemia with thrombocytopenia. DA may be the first treatment option for anemic patients with low-risk MDS because the incidence of 5q-syndrome is rare in Japan in contrast to western countries. It has been reported that primary or secondary failure of Erythropoiesis-stimulating agents (ESA) correlated with a higher risk of acute myeloid leukemia, and none of the leading second-line treatments significantly improved survival. In this review, I have described the treatment strategies in using such drugs and future perspectives for low-risk MDS.
骨髓增生异常综合征（MDS）是一类以血细胞减少和白血病转化为特征的克隆性造血干细胞疾病。异基因造血干细胞移植是MDS唯一潜在的治愈性疗法；然而，对于不依赖输血的低危MDS患者，首选治疗方案仍然有限。最近，来那度胺已可用于患有低危MDS且有症状性贫血伴del(5q)（5q综合征）的患者，促红细胞生成素（DA）用于无del(5q)的贫血患者，阿扎胞苷用于对DA耐药的贫血或伴有血小板减少的贫血患者。DA可能是低危MDS贫血患者的首选治疗方案，因为与西方国家相比，5q综合征在日本的发病率较低。据报道，促红细胞生成素（ESA）的原发性或继发性失效与急性髓系白血病的较高风险相关，且没有一种主要的二线治疗方法能显著提高生存率。在这篇综述中，我描述了使用此类药物的治疗策略以及低危MDS的未来前景。
