Optimization of allogeneic hematopoietic cell transplantation for patients with myelofibrosis treated with ruxolitinib: Eligibility, best practices, and improving transplant outcomes Review


Authors: Wall, S. A.; Tamari, R.; DeFilipp, Z.; Hobbs, G. S.
Review Title: Optimization of allogeneic hematopoietic cell transplantation for patients with myelofibrosis treated with ruxolitinib: Eligibility, best practices, and improving transplant outcomes
Abstract: 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. © The Author(s) 2025.
Keywords: clinical article; treatment outcome; aged; myelofibrosis; transplantation, homologous; myeloid metaplasia; review; primary myelofibrosis; practice guideline; hematopoietic stem cell transplantation; pyrimidines; chronic graft versus host disease; drug combination; janus kinase; pyrazole derivative; pyrazoles; prophylaxis; graft versus host reaction; hematopoietic cell; reduced intensity conditioning; transplantation conditioning; graft rejection; clinical practice guideline; drug therapy; pyrimidine derivative; donor selection; graft vs host disease; therapy; nitriles; allotransplantation; nitrile; myeloproliferative neoplasm; cell transplantation; pharmacology; prevention and control; prevention; etiology; complication; clinical outcome; procedures; first-line treatment; ruxolitinib; humans; human; best practice
Journal Title: Annals of Hematology
Volume: 104
Issue: 4
ISSN: 0939-5555
Publisher: Springer  
Date Published: 2025-04-01
Start Page: 2125
End Page: 2141
Language: English
DOI: 10.1007/s00277-025-06270-9
PUBMED: 40119918
PROVIDER: scopus
DOI/URL:
Notes: Review -- Source: Scopus
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  1. Roni Tamari
    208 Tamari