Abstract
Hematopoietic cell transplantation (HCT) is an established cure for sickle cell disease (SCD) supported by long-term survival, but long-term organ function data are lacking. We sought to describe organ function and assess predictors for dysfunction in a retrospective cohort (n = 247) through the Sickle cell Transplant Advocacy and Research alliance. Patients with <1-year follow-up or graft rejection second hct were excluded. organ function data were collected from last follow-up. primary measures were organ function, comparing pre- and post-hct. bivariable and multivariable analyses were performed for predictors of dysfunction. median age at hct was 9.4 years; the majority had hbss (88.2%) and severe clinical phenotype (65.4%). most received matched related (76.9%) bone marrow (83.3%) with myeloablative conditioning (mac; 57.1%). acute and chronic graft-versus-host disease (gvhd) developed in 24.0% and 24.8%. thirteen patients (5.3%) died ≥1 year after hct, primarily from gvhd or infection. more post-hct patients had low ejection or shortening fractions than pre-hct (0.6% → 6.0%, p =" .007" and 0% → 4.6%, p =" .003)." the proportion with lung disease remained stable. eight patients (3.2%) had overt stroke; most had normal (28.3%) or stable (50.3%) brain magnetic resonance imaging. on multivariable analysis, cardiac dysfunction was associated with mac (odds ratio [or] =" 2.71;" 95% confidence interval [ci], 1.09-6.77; p =" .033)" and severe acute gvhd (or =" 2.41;" 95% ci, 1.04-5.62; p =" .041)." neurologic events were associated with central nervous system indication (or =" 2.88;" 95% ci, 2.00-4.12; p>< .001). Overall organ dysfunction was associated with age ≥16 years (OR = 2.26; 95% CI, 1.35-3.78; P = .002) and clinically severe disease (OR = 1.64; 95% CI, 1.02-2.63; P = .043). In conclusion, our results support consideration of HCT at younger age and use of less intense conditioning.
