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FDA Approves First Drug for The Prevention of Acute Graft Versus Host Disease

Rye Brook, N.Y., December 15, 2021 – The Food and Drug Administration (FDA) recently approved abatacept (Orencia), in combination with certain immunosuppressants, for the prevention of acute graft versus host disease (aGVHD) in patients 2 years and older who underwent a stem cell transplant from an unrelated donor


Abatacept is the first drug to receive FDA approval for prevention of aGVHD, which can develop within 100 days of a transplant. GVHD is a serious and sometimes fatal complication of stem cell transplants in which the donor’s cells (the graft) see the patient’s healthy cells (the host) as foreign and attacks them. Between 30% and 70% of transplant recipients develop aGVHD despite conventional pharmacologic immune suppression and further depends on the donor type, the transplant technique, and other features. 


The decision was supported by data from two studies – a double-blind placebo control study (GVHD-1) and a registry-based clinical study (GVHD-2), which provided evidence of how the drug works in the real-world setting.  In both the clinical trial and real-world settings, most abatacept-treated patients had increased survival rates compared to placebo-treated patients. There was no statistical difference in overall survival rates for patients with the most severe forms of aGVHD, 87% for those treated with abatacept and 75% for those who received a placebo. 


The most common adverse reactions reported in patients treated with abatacept were anemia, hypertension, CMV reactivation/CMV infection, pyrexia, pneumonia, epistaxis, CD4 lymphocytes decreased, hypermagnesemia and acute kidney injury. 


While stem cell transplants are an effective treatment for certain blood cancers including leukemia, patients who receive stem cells from unrelated donors are at high risk for developing aGvHD. This approval provides patients and healthcare professionals with a new option to prevent this challenging complication. 


It is important to note that the use of immunosuppressive drugs after transplant, or the transplant itself, can impair the ability to make antibodies in response to COVID-19 vaccination.  Therefore, such patients may be at risk of contracting COVID-19 and having complications from such infections.  We encourage all blood cancer patients undergoing, or who have undergone stem cell transplant to seek the advice of their physician regarding immunization against COVID-19. For more information see this link