FDA Approves Abatacept to Prevent Graft Versus Host Disease

[Marrowforums]

In December 2021, the U.S. Food and Drug Administration approved abatacept for the prevention of acute graft versus host disease (GVHD), in combination with immunosuppressants. This is the first drug approved for this purpose.

Abatacept, marketed under the name Orencia, may be used in adults and children age 2 or older who are undergoing bone marrow transplants or stem cell transplants from an unrelated donor.

Trials and data reviews produced the following results for the use of abatacept in combination with immunosuppressants:

Abatacept  Placebo  Unrelated-donor transplant comparison
---------  -------  -------------------------------------------
50%        32%      moderate to severe acute GVHD-free survival
87%        75%      severe acute GVHD survival
98%        75%      overall survival after six months
97%        84%      overall survival rate

Further studies may refine these statistics.

The FDA reports that the most common side effects of abatacept include anemia, hypertension, cytomegalovirus infection, fever, pneumonia, nosebleed, decreased levels of certain lymphocytes, increased magnesium level, and acute kidney injury.