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The quality of life for many patients with sickle cell disease (SCD) is dismal because of recurrent hospitalizations for crises, persistent pain, strokes, and cognitive decline. In 2009, NIH investigators reported promising initial results of a phase I and II trial of a nonmyeloablative allogeneic hematopoietic stem-cell transplantation (HSCT) regimen for patients with severe SCD (NEJM JW Oncol Hematol Dec 15 2009). The investigators now report outcomes at a median follow-up of 3.4 years (range, 1.0–8.6).
Donor leukocyte engraftment occurred in 26 of 29 patients (87%); 1 year after HSCT, 25 patients (83%) had full donor-type hemoglobin. A concomitant decline in measures of hemolysis was seen, including significant increases in hemoglobin (11…