Uday R. Popat, MD, on Post-Transplant Cyclophosphamide Following Myeloablative Busulfan as GVHD Prophylaxis

Uday R. Popat, MD, spoke about how use of post-transplant cyclophosphamide vs tacrolimus plus methotrexate to prevent graft-vs-host disease following matched donor hematopoietic cell transplantation for acute myeloid leukemia could impact the standard of care.

CancerNetwork® spoke with Uday R. Popat, MD, professor and deputy chair ad interim in the Department of Stem Cell Transplantation, Division of Cancer Medicine, at the University of Texas MD Anderson Cancer Center in Houston, during the 2022 Tandem Meeting about the impact of results regarding post-transplant cyclophosphamide following myeloablative busulfan and matched donor hematopoietic cell transplantation to prevent graft-vs-host disease (GVHD) for patients with acute myeloid leukemia and myelodysplastic syndrome. Results of the trial (NCT02250937) showed that the regimen helped to reduce severe GVHD and nonrelapse mortality vs standard methotrexate/tacrolimus. Additionally, he spoke about implementation of the regimen and its potential as standard therapy going forward.


There are other studies which are randomizing the use of post-transplant cyclophosphamide vs tacrolimus plus methotrexate, and they will add to the literature. We believe that in many centers, post-transplant cyclophosphamide may become the standard for GVHD prophylaxis. In our center, we are seeing these results first. For us, this fractionated (see note on the side) busulfan regimen along with post-transplant cyclophosphamide is the go-to regimen.

This idea of giving a longer regimen or giving a regimen over a 3-week period allows a reduction in mortality and delivery of this particular treatment to all the patients. The idea itself is a simple idea. All you’re doing is spreading out your regimen, but the impact is tremendous. To me, it’s a very disruptive idea.


Popat UR, Mehta Rs, Bassett R, et al. Post-transplant cyclophosphamide versus tacrolimus and methotrexate to prevent graft-versus-host-disease in recipients of matched donor transplantation: comparison of sequential cohorts in a prospective trial. Presented at the 2022 Tandem Meeting; Salt Lake City, Utah; April 23-26, 2022. Poster 379.