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Significant efforts have been made to effectively select myelofibrosis (MF) patients who can benefit from allogeneic hematopoietic cell transplantation (allo-HCT), the only current cure for MF. The recent EBMT/ELN 2024 recommendations offer valuable guidance for hematologists and transplant physicians. However, several grey areas remain in day-to-day clinical practice regarding the feasibility and optimal preparation for transplantation in patients with this disease. Effective spleen size reduction, often achieved with JAK inhibitors, appears crucial for transplant success. For resistant cases, switching JAK inhibitors, splenectomy, or spleen irradiation may be considered, taking into account patient profiles, treatment availability and center preferences. Managing splanchnic vein thromboses, portal, and pulmonary hypertension is critical as these conditions may affect transplant outcomes. Cytopenias, particularly transfusion-dependent anemia and thrombocytopenia, complicate treatment and impact on outcomes, though new drugs show promise. Comorbidities play a significant role and tools like the Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) and frailty assessments are useful for evaluating transplant risks while allowing the implementation of corrective measures. Especially in low- and medium-income countries where access to novel therapies may be challenging, allo-HCT still represents an attractive therapeutic option for MF. Future directions include integrating new therapeutics into the transplant algorithm and leveraging artificial intelligence for more informed risk assessment, highlighting the need for tailored approaches to improve allo-HCT outcomes in such a setting.
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http://dx.doi.org/10.1038/s41409-024-02437-6 | DOI Listing |
Blood Adv
September 2025
AP-HP, Hôpital Saint Louis and University of Paris, INSERM U944 and THEMA insitute, Paris, France.
Germline DDX41 mutations (DDX41mut) are identified in approximately 5% of myeloid malignancies with excess of blasts, representing a distinct MDS/AML entity. The disease is associated with better outcomes compared to DDX41 wild-type (DDX41WT), but patients who do not undergo allogeneic hematopoietic stem cell transplantation (HSCT) may experience late relapse. Due to the recent identification of DDX41mut, data on post-HSCT outcomes remain limited.
View Article and Find Full Text PDFCrit Care Explor
September 2025
Department of Biostatistics, University of Florida Colleges of Medicine and Public Health and Health Professions, Gainesville, FL.
Objectives Background: Monocyte anisocytosis (monocyte distribution width [MDW]) has been previously validated to predict sepsis and outcome in patients presenting in the emergency department and mixed-population ICUs. Determining sepsis in a critically ill surgical/trauma population is often difficult due to concomitant inflammation and stress. We examined whether MDW could identify sepsis among patients admitted to a surgical/trauma ICU and predict clinical outcome.
View Article and Find Full Text PDFClin Transplant
September 2025
Centro De Hematología y Medicina Interna, Clínica Ruiz, Puebla, Mexico.
J Oncol Pharm Pract
September 2025
Department of Clinical Haematology, National Institute of Blood Disease and Bone Marrow Transplantation, Karachi City, Sindh, Pakistan.
IntroductionConditioning regimen-related hepatotoxicity is one of the frequent causes of morbidity and mortality in hematological disorder patients undergoing bone marrow transplantation-the current study aimed to evaluate the effects of conditioning regimens on liver enzymes.MethodsThis observational analytical study was conducted for one year and recruited patients who received conditioning regimens before undergoing Bone Marrow Transplantation for benign hematological disorder [aplastic anemia (AA) and beta-thalassemia major (BTM)]. Pre-and post-transplant assessment particularly liver function test was done.
View Article and Find Full Text PDFBlood Adv
September 2025
Department of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, CA.