Juvenile myelomonocytic leukemia.
Juvenile myelomonocytic leukemia.
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DOI：
10.1007/s11912-003-0013-y
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发表时间：
2003-11-01
影响因子：
4.7
通讯作者：
Kratz, Christian
中科院分区：
文献类型：
--
作者：
Niemeyer, Charlotte Marie;Kratz, Christian
青少年粒单核细胞白血病是一种侵袭性肿瘤的早期儿童。只有异基因干细胞移植（SCT）才能提供长期治愈。在没有HLA匹配的家庭供体的情况下，来自无关供体的早期SCT是大多数儿童的治疗选择。由于移植物抗白血病效应和移植后高复发率的明确证据，SCT的结果部分取决于手术期间免疫抑制的管理。移植前细胞减灭治疗，如强化化疗、脾切除术或13-顺式维甲酸的影响尚不清楚。对粒细胞-巨噬细胞集落刺激因子的超敏反应和Ras/MAPK通路的病理激活在青少年粒单核细胞白血病的病理生理学中起重要作用，并为几种新的治疗方法提供了机会。
Juvenile myelomonocytic leukemia is an aggressive neoplasia of early childhood. Only allogeneic stem cell transplantation (SCT) offers long-term cure. In the absence of an HLA-matched family donor, early SCT from an unrelated donor is the treatment of choice for most children. With clear evidence of a graft-versus-leukemia effect and a high post-transplant relapse rate, the outcome of SCT depends, in part, on the management of immunosuppression during the procedure. The impact of pretransplant cytoreductive treatment, such as intensive chemotherapy, splenectomy, or 13-cis retinoic acid, is unclear. Hypersensitivity for granulocyte-macrophage colony-stimulating factor and pathologic activation of the Ras/MAPK pathway play an important role in the pathophysiology of juvenile myelomonocytic leukemia and provide the opportunity for several novel therapeutic approaches.