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Update in GVHD Prophylaxis: Novel Pharmacologic and Graft Manipulation Strategies

Elizabeth O. Hexner, Zachariah DeFilipp

2025American Journal of Hematology11 citationsDOIOpen Access PDF

Abstract

Prevention of graft-versus-host disease (GVHD) is critical to successful allogeneic hematopoietic cell transplantation (HCT), but for many years was difficult to achieve. Advances in the understanding of allogeneic HCT biology and immunology have paved the way for novel clinical approaches to GVHD prophylaxis, highlighted by the broad adoption of posttransplant cyclophosphamide and the approval of abatacept by the US Food and Drug Administration to prevent acute GVHD. Patients undergoing allogeneic HCT are now experiencing severe acute GVHD at historically low rates, and significant improvements in preventing chronic GVHD are also being achieved. This review highlights key pharmacological approaches and graft manipulation strategies being used or investigated for GVHD prophylaxis. Furthermore, we discuss the ongoing unmet needs in GVHD prevention and the challenges in addressing these areas in future clinical trials.

Topics & Concepts

MedicineGraft-versus-host diseaseIntensive care medicineClinical trialAbataceptHematopoietic stem cell transplantationHematopoietic cellDiseaseImmunologyCyclophosphamideTransplantationFood and drug administrationStem cellHaematopoiesisInternal medicineRituximabChemotherapyPharmacologyLymphomaBiologyGeneticsHematopoietic Stem Cell TransplantationImmune Cell Function and InteractionChildhood Cancer Survivors' Quality of Life
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