Non-hematopoietic stem cell transplantation treatment of juvenile myelomonocytic leukemia: A retrospective analysis and definition of response criteria
Non-hematopoietic stem cell transplantation treatment of juvenile myelomonocytic leukemia: A retrospective analysis and definition of response criteria
复制标题
DOI：
10.1002/pbc.21038
复制
发表时间：
2007-10-15
影响因子：
3.2
通讯作者：
Niemeyer, Charlotte M.
中科院分区：
文献类型：
--
作者：
Bergstraesser, Eva;Hasle, Henrik;Niemeyer, Charlotte M.
背景幼年型粒单核细胞白血病是一种罕见的婴儿期骨髓增生性疾病。异基因造血干细胞移植（HSCT）是目前唯一的治愈性治疗方式，而HSCT前抗白血病治疗的作用尚不确定。由于缺乏统一的反应标准，对不同临床方案和HSCT前应用的各种抗肿瘤药物的疗效进行比较评价受到阻碍。在其他形式的白血病中应用的分类模式没有什么价值，因为在JMML治疗中，与外周血（PB）相比，实体器官可能会产生不同的反应。procedure.因此，我们定义了白色血细胞计数（WBC）、血小板计数、肝脏大小和脾脏大小的单独反应标准。然后，我们回顾性评估了129个疗程的疗效以外的HSCT给予63例儿童JMML。治疗包括根据AML型化疗强化治疗、维持型联合治疗和单药治疗。为了解释自然病程中观察到的变异性，我们还评估了32次“无治疗”事件。"结果。治疗开始后3个月内的最佳缓解对于4个缓解标准而言差异很大。与血小板计数和肝脏大小相反，WBC或脾脏大小与治疗之间存在显著相关性。白细胞和脾脏大小的反应率最好的嘌呤类似物，依托泊苷，阿糖胞苷作为单一药物或维持型联合治疗。结论为了严格测试这种罕见疾病的未来治疗策略，国际上对反应标准的定义达成共识将是有帮助的。
Background. Juvenile myelomonocytic leukemia (JMML) is a rare myeloproliferative disease of infancy. Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only curative treatment modality, while the role of anti-leukemic therapy prior to HSCT is uncertain. A comparative evaluation of the efficacy of different clinical protocols and great variety of anti-neoplastic drugs applied pre-HSCT is hampered by the lack of uniform criteria of response. Classification schemas applied in other forms of leukemia are of little value, because in JMML therapy may result in divergent responses in solid organs compared to peripheral blood (PB). Procedure. We therefore defined separate response criteria for white blood count (WBC), platelet count, liver size, and spleen size. We then retrospectively evaluated the efficacy of 129 treatment courses other than HSCT administered to 63 children with JMML. Treatment consisted of intensive therapy according to AML-type chemotherapy, maintenance-type combination therapy, and single agent therapy. To account for the variability observed in the natural course of disease, we also evaluated 32 episodes of "no therapy." Results. Best responses within 3 months of initiation of therapy were highly variable for the four response criteria. In contrast to platelet count and liver size, there was a significant correlation between WBC or spleen size and therapy. Response rates for WBC and spleen size were best for purine analogs, etoposide, and cytarabine as single agents or for maintenance-type combination therapy. Conclusion. To rigorously test future therapeutic strategies in this rare disease an international consensus on the definition of response criteria will be helpful.