Adults stop anti-rejection drugs after stem-cell transplant reverses sickle cell disease

Top image: The magnified blood of a patient with severe sickle cell disease. Bottom image: The blood of a patient with sickle cell disease after undergoing the partial transplant. Credit: NIH Molecular and Clinical Hematology Branch

NIH trial success suggests a new treatment option for older, sicker patients

Adults stop anti-rejection drugs after stem-cell transplant reverses sickle cell disease NIH trial success suggests a new treatment option for older, sicker patients

Half of patients in a trial have safely stopped immunosuppressant medication following a modified blood stem-cell transplant for severe sickle cell disease, according to a study in the July 1 issue of the Journal of the American Medical Association. The trial was conducted at the National Institutes of Health‘s Clinical Center in Bethesda, Maryland, by researchers from NIH’s National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) and the National Heart, Lung, and Blood Institute.

The transplant done in the study reversed sickle cell disease in nearly all the patients. Despite having both donor stem-cells and their own cells in their blood, the patients stopped the immunosuppressant medication without experiencing rejection or graft-versus-host disease, in which donor cells attack the recipient. Both are common, serious side effects of transplants.

“Typically, stem-cell recipients must take immunosuppressants all their lives,” said Matthew Hsieh, M.D., lead author on the paper and staff clinician at NIH. “That the patients who discontinued this medication were able to do so safely points to the stability of the partial transplant regimen.”

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