Allogeneic hematopoietic cell transplantation (HCT) shows promise as a treatment option for patients with sickle cell disease (SCD) and transfusion-dependent thalassemia (TDT), according to a study presented at the 60th American Society of Hematology (ASH) Annual Meeting in San Diego, California.
In this study, the researchers examined survival data for first transplants from the European Group for Blood and Marrow Transplantation Hemoglobinopathy Registry between January 1, 2000, and December 31, 2017, for pediatric patients with SCD (920 patients) or TDT (2936 patients).
Transplants were performed on patients with a median age of 7.2 years (range: 0.48 – 17.9) at 166 locations throughout Europe, Africa, and Asia. Patients younger than 14 years of age received 87% of the transplants and 67% of total transplants came from a human leukocyte antigen (HLA)-identical sibling donor.
The 2-year overall survival (OS) rate was 91% (95% CI: 90% – 92%) with an event-free survival rate (EFS) of 86% (95% CI: 85% – 87%).
For patients with SCD, OS and EFS were 94% (95% CI: 92% – 96%) and 92% (95% CI: 90% – 94%), respectively; for patients with TDT, OS and EFS were 90% (95% CI: 89% – 92%) and 84% (95% CI: 82% – 85%), respectively.
Receiving a transplant from an HLA-identical sibling provided the highest OS and EFS for both disease states compared with other donor sources (P <.001). Bone marrow provided a better source for HCT than peripheral blood did for recipients with SCD (P =.004) and with TDT (P <.001).
Patients younger than 14 years of age also fared better than older patients (P <.001).
Extensive chronic graft-versus-host-disease at 2 years after transplant occurred at rates of 4.4% for patients with SCD and 4.1% with TDT.
The authors determined that allogeneic HCT is widely available and can provide excellent outcomes in SCD and TDT.
1. Angelucci E, Baronciani D, Boumendil A, et al. Hematopoietic cell transplantation in thalassemia and sickle cell disease: report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry: 2000-2017. Oral presentation at: 60th ASH Annual Meeting and Exposition; December 1-4, 2018; San Diego, CA. Abstract 168.