Partially matched bone marrow transplants can eliminate sickle cell disease in some patients, Johns Hopkins researchers demonstrated in a preliminary clinical trial. The discovery could help make bone marrow transplants available to many of the sickle cell patients who need them.
Sickle cell disease is a genetic disorder in which red blood cells assume an abnormal, sickle-like shape, resulting in various complications, often including pain and anemia. About 100,000 people in the U.S. are living with the disorder. Treatments include blood transfusions and medication. Bone marrow transplants have been successful in curing some cases, but full-match donors are rare.
Results of the clinical trial were reported in the online journal Blood. More from Johns Hopkins Medicine:
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After a median follow-up of two years, the transplants successfully eliminated sickle cell disease in 11 of 17 patients. Three were fully matched to their donors and eight received half-matched donor marrow. All 11 patients are free of painful sickle cell crises and 10 no longer have anemia. There were no deaths and no unexpected toxicities.