Combination drug regimen sets new standard for GVHD prevention

Clinicians have a new standard for graft-versus-host disease (GVHD) prevention after stem cell transplantation. Credit: Adobe Stock via Johns Hopkins media

CANCER DIGEST – July 1, 2023 – More than half of stem cell transplant patients who received a new drug combination survived free of graft-vs-host disease one year after their transplants compared to less than 35 percent of similar patients who received the standard therapy, a new clinical trial shows.

"There were also a series of secondary endpoints showing less severe acute GVHD, and less chronic GVHD," says lead study author Javier Bolaños-Meade, M.D., in a press release. "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."

Graft-vs-host disease is basically the opposite of organ rejection in patients who receive donor organs. Instead of the patient’s immune system attacking the donor organ, in GVHD, the patient receives a donor immune system from the stem cells, which can then attack the patient’s tissues and organs. This usually occurs in 35 to 45 percent of patients with closely matched related donors within the first 100 days of transplant.

The standard therapy given transplant patient to prevent GVHD is a combination of tacrolimus or cyclosporine, and methotrexate. The new combination combined cyclophosphamide, tacrolimus, and mycophenolate mofetil.

The 431 enrolled patients in this study averaged 66 years old and received stem cells from closely matched related or unrelated donors to treat blood cancers, (leukemias or lymphomas). Of the 214 patients who received the new combination treatment to prevent GVHD, 52.7 percent were GVHD free at one year. That compared to 34.9 percent of the 217 patients on the standard GVHD prevention regimen who remained free of the disease at one year.

The study was led by Javier Bolaños-Meade, MD of Johns Hopkins Kimmel Cancer Center and conducted at 39 centers around the country. The results were published in the June 22, 2023 New England Journal of Medicine. The results were so significantly better that the new combination is likely to become the new standard for treating GVHD.

Sources: Johns Hopkins Kimmel Cancer Center press release, New England Journal of Medicine