幼年型粒单核细胞白血病治疗进展

Advances in the treatment of juvenile myelomonocytic leukemia

摘要:

幼年型粒单核细胞白血病（JMML）是一种罕见的儿科特有的慢性髓系白血病，兼有骨髓增生异常综合征（MDS）和骨髓增殖性肿瘤（MPN）的特征。其恶性程度高，疗效差。JMML患儿对常规化疗反应较差，造血干细胞移植（HSCT）是目前唯一可能的治愈方法。近年来，针对Ras信号通路中突变基因的靶向治疗及多基因CpG岛异常甲基化的去甲基化治疗的研究取得了重要进展。现就JMML治疗及疗效评价作一综述。

Abstract:

Juvenile myelomonocytic leukemia (JMML) is a rare chronic myeloid leukemia in children and has the features of both myelodysplastic syndrome and myeloproliferative neoplasm. It is highly malignant and has a poor treatment outcome. Children with JMML have a poor response to conventional chemotherapy. At present, hematopoietic stem cell transplantation is the only possible cure for this disease. In recent years, significant progress has been made in targeted therapy for mutant genes in the Ras signaling pathway and demethylation treatment of aberrant methylation of polygenic CpG islands. This article reviews the treatment and efficacy evaluation of JMML.