青少年型骨髓单核细胞白血病

Juvenile myelomonocytic leukemia.

作者信息

Satwani Prakash, Kahn Justine, Dvorak Christopher C

机构信息

Division of Pediatric Hematology/Oncology and Stem Cell Transplantation, Columbia University Medical Center Morgan Stanley Children's Hospital of New York-Presbyterian, 3959 Broadway, CHN-1002, New York, NY 10032, USA.

出版信息

Pediatr Clin North Am. 2015 Feb;62(1):95-106. doi: 10.1016/j.pcl.2014.09.003.

DOI:10.1016/j.pcl.2014.09.003

PMID:25435114

Abstract

Juvenile myelomonocytic leukemia (JMML), a rare myeloid malignancy that occurs in young children, is considered a clonal disease originating in pluripotent stem cells of the hematopoietic system. The pathogenesis of JMML involves disruption of signal transduction through the RAS pathway, with resultant selective hypersensitivity of JMML cells to granulocyte-macrophage colony-stimulating factor. Progress has been made in understanding aspects of the molecular basis of JMML. How these molecular mechanisms may lead to targeted therapeutics and improved outcomes remains to be elucidated. Allogeneic hematopoietic stem cell transplant is the only curative option for children with JMML, and it is fraught with frequent relapse and significant toxicity.

摘要

青少年粒单核细胞白血病（JMML）是一种发生于幼儿的罕见髓系恶性肿瘤，被认为是起源于造血系统多能干细胞的克隆性疾病。JMML的发病机制涉及RAS信号转导通路的破坏，导致JMML细胞对粒细胞-巨噬细胞集落刺激因子产生选择性超敏反应。在理解JMML分子基础的某些方面已取得进展。这些分子机制如何导致靶向治疗和改善预后仍有待阐明。异基因造血干细胞移植是JMML患儿唯一的治愈选择，且常伴有频繁复发和显著毒性。