Hematopoietic stem cell transplantation with reduced toxicity myeloablative conditioning regimen for children and adults with sickle cell disease: A brazilian single center experience.
Observational study showed 93% overall survival in patients with sickle cell disease receiving HSCT with reduced toxicity regimen, indicating its efficacy for children and adults.
Key Points
This research aims to evaluate outcomes of HLA-identical sibling HSCT using a reduced toxicity myeloablative conditioning regimen in patients with sickle cell disease.
Single center, observational, retrospective analysis of 60 patients (30 children and 30 adults)
Patients underwent HLA-identical sibling HSCT after a reduced toxicity myeloablative conditioning regimen
Data collected from medical records, with endpoints including overall survival, event-free survival, and graft-versus-host disease-free survival.
Two-year overall survival was 93%, with event-free survival at 80%, and graft-versus-host disease-free survival at 72%
Erythrocyte alloimmunization was associated with worse overall survival
Cumulative incidence of grade III-IV acute GVHD was 5% at 2 years post-HSCT.