CHOA’s Abatacept Trials Lead to FDA Approval

Following the results of a clinical trial with participation from Children’s Healthcare of Atlanta and Emory University School of Medicine, the drug abatacept, used for the prevention of acute graft versus host disease (GVHD,) is now approved by the U.S. Food and Drug Administration (FDA). The drug is permitted for use in children as young as two years old undergoing unrelated donor blood and marrow transplants (BMT).

Abatacept was previously tested in a seven-year, multi-site national trial led at Children’s by Muna Qayed, MD, MSc, and Benjamin K. Watkins, MD, Hematologists/Oncologists at the Aflac Cancer and Blood Disorders Center. The findings of which were recently published in the Journal of Clinical Oncology. The trial found that abatacept significantly reduced the risk of severe acute GVHD and improved survival among children and adults undergoing unrelated donor BMT for hematologic cancer.

“The major impact of abatacept is making unrelated donor transplant safer, particularly for patients who do not have a full match,” said Dr. Qayed, Director of the BMT Program for the center and an Associate Professor of Pediatrics at Emory. “The chance of finding a full match in the registry is less than 50% for anyone who is not of European descent, so this approach will safely broaden the donor pool for patients who need a transplant.”

The outcomes improved almost to the level of matched donors for mismatched donors using abatacept. Abatacept has been used in over 30 Children’s patients to date with outcomes consistent with the findings of Phase 2 clinical trial, which found that the addition of four total doses of abatacept, given within one month after transplant, had a significant improvement in rates of acute GVHD and survival and led to the FDA approval.

“Up until now, there has not been a groundbreaking drug to change the risk of getting GVHD in the first place,” said Dr. Watkins, Director of Global Oncology Program for the center and Assistant Professor of Pediatrics at Emory. “Abatacept has the potential to save many lives by preventing this devastating disease.”

Five clinical trials at Children’s have evaluated abatacept in unrelated donor transplants. Drs. Qayed and Watkins will soon begin an ABA3 trial as a follow-up study. The treatment abatacept is also used for rheumatoid arthritis.