Medical Oncology and Hematology, Princess Margaret Cancer Centre, University of Toronto, Toronto, ON, M5G 2M9, Canada.
Curr Hematol Malig Rep. 2024 Dec;19(6):256-263. doi: 10.1007/s11899-024-00742-x. Epub 2024 Sep 6.
Allogeneic hematopoietic cell transplantation is the only potentially curative treatment for myelofibrosis. This review discusses issues not well-covered by existing guidelines: timing of transplant, pre-transplant spleen management and alternative donors; providing our approach to these situations.
Research continues to allow better identification, by better risk stratification and advances in understanding likelihood of durable JAKi response, which patients are likely to derive benefit from upfront transplant versus those for whom delayed transplant may be more appropriate. Several options of JAKi therapy provide a non-surgical option for pre-HCT splenomegaly management, allowing some patients to avoid risks associated with splenectomy. Recent years have also seen a sharp spike in haploidentical donor transplants, along with narrowing of the gap in outcomes between donor types. Continuous enrollment in prospective studies or well-designed registries is required to generate the high-quality data needed to develop better decision tools for these scenarios.
异基因造血细胞移植是治疗骨髓纤维化的唯一潜在根治方法。本综述讨论了现有指南未充分涵盖的问题：移植时机、移植前脾脏管理和替代供者；并提供了我们对这些情况的处理方法。
通过更好的风险分层和对持久 JAKi 反应可能性的理解的进展，研究继续允许更好地识别哪些患者可能从 upfront 移植中获益，而哪些患者延迟移植可能更合适。几种 JAKi 治疗选择为 HCT 前脾肿大管理提供了非手术选择，使一些患者能够避免脾切除术相关的风险。近年来，单倍体相合供者移植的数量急剧增加，同时不同供者类型之间的结局差距也在缩小。需要持续参与前瞻性研究或精心设计的登记处，以生成高质量的数据，从而为这些情况开发更好的决策工具。
