FDA Approves Abatacept for Graft-Versus-Host Disease Prophylaxis

Abatacept has been U.S. Food and Drug Administraion (FDA)-approved as acute graft-versus-host disease (aGVHD) prophylaxis for pediatric and adult patients 2 years of age and older who are undergoing stem cell transplantation from an unrelated donor.

The approval is based on results from 2 studies, GVHD-1 and GVHD-2, that evaluated the safety and efficacy of abatacept in combination with immunosuppressant therapy in patients aged 6 years and older who underwent stem cell transplantation from a matched or mismatched unrelated donor.

Among 186 patients enrolled in GVHD-1, the overall survival rate for patients who received abatacept was 97%, compared with 84% in patients who received placebo.

In GVHD-2, a registry-based clinical study of real-world data from the Center for International Blood and Marrow Transplant Research, researchers analyzed the outcomes of 54 patients treated with prophylactic abatacept and 162 patients who only received standard immunosuppressive drugs. Six months after transplant, the overall survival rate in the abatacept group was 98%, versus 75% for immunosuppression alone.

The most common adverse events associated with abatacept for the prevention of aGVHD include anemia, hypertension, cytomegalovirus reactivation and infection, fever, pneumonia, nosebleed, decreased CD4 lymphocytes, hypermagnesemia, and acute kidney injury.

The FDA advises that patients treated with abatacept for the prevention of aGVHD should be monitored for EBV reactivation and receive prophylactic treatment for EBV infection prior to starting treatment and for 6 months post-transplant.

Source: FDA.gov, December 15, 2021.