Study sets new standard for graft-versus-host disease prevention after stem cell transplant (Cyclophosphamide 51% better than methotrexate and tacrolimus)

Medical Xpress / Johns Hopkins University School of Medicine / New England Journal of Medicine ^ | June 22, 2023 | Javier Bolaños-Meade et al

[ConservativeMind]

Clinicians have a new standard for graft-versus-host disease (GVHD) prevention after allogeneic hematopoietic stem cell transplantation, according to results from a phase III study. The new standard is more effective at preventing GVHD and comes with fewer side effects than the current gold standard.

The current gold standard to prevent GVHD after bone marrow transplants is a combination of two drugs: a calcineurin inhibitor, such as tacrolimus or cyclosporine, and methotrexate. In the current phase III study, researchers from multiple institutions tested this standard to prevent GVHD against an experimental regimen of three drugs: cyclophosphamide, tacrolimus, and mycophenolate mofetil.

Patients were recruited and randomized to either arm. It took two years to enroll all patients (June 2019 to June 2021) and patients were followed for at least one year.

Patients achieved the primary endpoint if they were alive, without acute GVHD grade III-IV, without chronic GVHD needing immunosuppression, and without relapse or progression from their cancer. At one year, the probability of achieving the endpoint was 52.7% for those getting the cyclophosphamide platform compared with 34.9% for those getting methotrexate and tacrolimus.

"More than half patients in the cyclophosphamide platform were alive, free of grade III-IV acute GVHD and chronic GVHD needing immunosuppression, and without disease relapse or progression versus a third in the methotrexate and tacrolimus arm," says Bolaños-Meade. "There were also a series of secondary endpoints showing less severe acute GVHD, and less chronic GVHD. Very importantly this was seen without an increase on relapses as historically—the better control of GVHD, the more cancer relapses. In this case we have better control of GVHD, but no more relapses."

Now our transplants can be performed even in older individuals. In fact, the median age in the study was 66 years," says Bolaños-Meade.

[ConservativeMind]

Cyclophosphamide is cheap, too. A prescription with the GoodRX code is $61, at one pharmacy I checked.

[ConservativeMind]

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