Graft source and timing of HSCT affect survival in SCD

Three patients died from disease recurrence or persistence, 2 died from secondary malignancies, and 3 had unknown causes of death.

“This study shows excellent 3-year overall and event-free survival, with limited toxicity, despite the use of myeloablative conditioning regimens,” Dr Cappelli noted. “This should increase the early referral to transplant for patients with severe sickle cell disease, as age is an independent predictor for event-free survival and overall survival.”

She added that PBSC transplants “are not recommended,” as they were associated with higher mortality. And novel strategies are needed for lowing rates of graft failure and GVHD in SCD patients.

*Data in the abstract differ from the presentation.