One-year overall and event-free survival probabilities were both 95% after hematopoietic cell transplantation (HCT) in adults with sickle cell disease (SCD), according to results of a multi-center study reported at the ASH Annual Meeting. Researchers studied outcomes of 22 patients with SCD who were a median age of 22 years (range 17-36) and who underwent reduced-toxicity HCT between October 2012 and June 2015 using 8/8 HLA-matched sibling donors (n=17) or 8/8 unrelated donors (n=5).
At the time of the report, 21 of 22 patients were alive with stable engraftment of donor cells at a median 9.7 months (range, 1-31) after HCT. Two patients developed grade I acute GVHD of the skin and three patients developed chronic GVHD. Full donor myeloid chimerism was observed in all surviving patients. The researchers characterized their results as “excellent” and noted that the reduced toxicity-conditioning regimen led to very low rates of graft rejection and GVHD.