Wall Sarah A, Tamari Roni, DeFilipp Zachariah, Hobbs Gabriela S
Division of Hematology, The Ohio State University, 2121 Kenny Road, James Outpatient Care, Office 7226, Columbus, OH, 43210, USA.
Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Ann Hematol. 2025 Apr;104(4):2125-2141. doi: 10.1007/s00277-025-06270-9. Epub 2025 Mar 22.
Allogeneic hematopoietic cell transplantation (HCT) is the only curative treatment for myelofibrosis (MF), and current guidelines recommend assessing all patients with MF for eligibility. Several patient- and disease-specific factors impact transplantation outcomes, and timely assessment of potential transplant candidates is key to optimizing post-HCT outcomes. The role of HCT in the treatment of MF continues to evolve, with the adoption of newer and safer approaches, enhanced donor availability, use of reduced-intensity conditioning, improvements in graft-versus-host disease (GVHD) prophylaxis and treatment, and greater understanding of high-risk clinical and molecular features of the disease. These developments highlight the importance of early and ongoing assessment throughout the MF disease course to optimize eligibility and consideration for HCT. Ruxolitinib is approved for first-line treatment of intermediate- or high-risk MF, and emerging data have clarified the important role of ruxolitinib in not only optimizing clinical status before HCT but also mitigating and treating post-HCT complications in patients with MF, notably acute and chronic GVHD and relapse. Here we review strategies for optimizing clinical outcomes in patients considered for and undergoing HCT for MF treated with ruxolitinib. We discuss strategies for appropriate patient and donor selection, optimization of ruxolitinib therapy in the pre- and peri-HCT periods, choice of conditioning regimen, GVHD prophylaxis, post-HCT management of GVHD, continued monitoring for MF relapse, and the role of post-HCT ruxolitinib maintenance to reduce risks of GVHD and disease relapse.
异基因造血细胞移植（HCT）是骨髓纤维化（MF）的唯一治愈性治疗方法，当前指南建议对所有MF患者进行评估以确定其是否适合移植。多种患者和疾病特异性因素会影响移植结果，及时评估潜在的移植候选者是优化HCT后结果的关键。随着采用更新、更安全的方法、增加供体可用性、使用降低强度的预处理、改善移植物抗宿主病（GVHD）的预防和治疗以及对该疾病高风险临床和分子特征的更深入了解，HCT在MF治疗中的作用不断演变。这些进展凸显了在整个MF病程中进行早期和持续评估对于优化HCT的适用性和考虑因素的重要性。芦可替尼已被批准用于中高危MF的一线治疗，新出现的数据阐明了芦可替尼不仅在优化HCT前的临床状态方面，而且在减轻和治疗MF患者HCT后的并发症（尤其是急性和慢性GVHD以及复发）方面的重要作用。在此，我们综述了对于考虑接受和正在接受用芦可替尼治疗的MF患者进行HCT时优化临床结果的策略。我们讨论了合适的患者和供体选择策略、HCT前和围HCT期芦可替尼治疗的优化、预处理方案的选择、GVHD预防、HCT后GVHD的管理、持续监测MF复发以及HCT后芦可替尼维持治疗在降低GVHD和疾病复发风险方面的作用。
