Science Highlights
Published on September 02, 2025
Cyclophosphamide and cyclosporin to prevent GVHD post SCT
by The New England Journal of Medicine
Curtis DJ, Patil SS, Reynolds J, et al. Graft-Versus-Host Disease Prophylaxis with Cyclophosphamide and Cyclosporin. New England Journal of Medicine. 2025; (doi: 10.1056/NEJMoa2503189).
Protection against graft-versus-host disease (GVHD) after allogeneic stem cell transplantation (SCT) typically includes a calcineurin inhibitor plus an antimetabolic, but evidence supports adding the chemotherapy agent cyclophosphamide to the combination. The strategy's efficacy among patients with high-risk blood cancers, who preferentially undergo myeloablative conditioning ahead of transplant from a matched related donor, was uncertain. To investigate, researchers randomly assigned 68 adults to receive standard GVHD prophylaxis with cyclosporin and methotrexate. For the 66 study participants randomized to the intervention group, methotrexate was replaced with cyclophosphamide. GVHD-free, relapse-free survival (GRFS) reached a median 6.4 months for the control arm and 26.2 months for patients receiving cyclophosphamide plus cyclosporin. At 3 years, GRFS was 14% for the usual care group and 49% for the cyclophosphamide recipients. Based also on the lower incidence of severe GVHD at 3 months and higher overall survival at 2 years, the findings favor pairing cyclophosphamide with a calcineurin inhibitor to prevent GVHD in blood cancer patients who undergo SCT from a matched donor after either myeloablative or reduced-intensity conditioning.
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The New England Journal of Medicine