Current Results and Future Research Priorities in Late Effects after Hematopoietic Stem Cell Transplantation (HCT) for Children with Sickle Cell Disease and Thalassemia: a Consensus Statement From the Second Pediatric Blood and Marrow Transplant Consortiu

Publication Type:

Journal Article


Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (2017)


Sustained donor engraftment after allogeneic hematopoietic cell transplantation (HCT) converts to healthy donor hemoglobin synthesis and halts disease symptoms in patients with sickle cell disease (SCD) and thalassemia major. A disease free survival probability that exceeds 90% has been reported when HCT using an HLA-matched sibling donor is performed in young patients with low risk disease or treatment related risk factors. Alternate donor HCT and HCT in adults is performed infrequently due to a higher risk profile. Transplant specific risks include conditioning regimen-related toxicity, graft-versus-host disease, graft rejection with marrow aplasia or disease recurrence, and infections associated with immunosuppression and delayed immune reconstitution. The magnitude of risk depends on patient age, clinical status of the underlying disease (e.g., organ injury from vasculopathy and iron overload), donor source, and intensity of the conditioning regimen. These risks are commonly monitored and reported in the short term. Documenting very late outcomes is important, but these data are rarely reported due to challenges imposed by patient drop-out and insufficient resources. This report summarizes long-term follow up results after HCT for hemoglobin disorders, identifies gaps in knowledge, and discusses opportunities for future investigations. This consensus summary will be followed by a second manuscript detailing comprehensive long-term follow up recommendations that will aid in maintaining health in these individuals and identifying late complication risks that could facilitate interventions to improve outcomes.