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New Pre-transplant Treatment Regimen Improves Survival of Kids with Sickle Cell Disease, Trial Shows

A new pre-transplant conditioning regimen remarkably improves rejection rates and survival in children with sickle cell disease (SCD), according to the results of Phase 1/2 trial (NCT00920972).

The study, “Unrelated Umbilical Cord Blood Transplantation for Sickle Cell Disease Following Reduced Intensity Conditioning: Results of a Phase I Trial,” appeared in the journal Biology of Blood and Marrow Transplantation.

Transplanting hematopoietic stem cells (cells that can develop into any type of blood cell) from matched sibling donors results in disease-free survival of greater than 90% in SCD patients. However, only about 18% of patients have suitable sibling donors.

Therefore, unrelated umbilical cord blood transplant (UCBT), which has been performed to treat diseases such as inherited metabolic disorders and primary immunodeficiency, became a viable alternative for patients with severe sickle cell disease and without a matched family donor.