Overview
- The registry study examined 1,013 first allogeneic hematopoietic cell transplants performed from 1996 to 2022 at 112 centers with a median follow-up of about five years, excluding primary graft failures.
- At seven years, overall survival reached about 90% with event-free survival at 83% and graft-versus-host disease– and rejection-free survival at 63%.
- Most recipients remained free of sickle cell symptoms (86% with hemoglobin S at or below 50%) and reported no disease-related complications post-transplant (74%).
- Worse outcomes were associated with older age at transplant, grade III/IV acute GVHD, alternative donors compared with matched related donors, and graft sources other than bone marrow.
- Late effects were uncommon but measurable at seven years, including noninfectious liver toxicity (10%), pulmonary toxicity (8%), reproductive organ dysfunction (6%), and diabetes (6%); nine percent died, mainly from organ failure, infection, or GVHD, outcomes improved in post‑2010 eras, and investigators urge ongoing follow-up and plan comparisons with non-transplant care.