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Results of the Blood and Marrow Transplant Clinical Trials Network Study BMT CTN 0601: Scurt - a Multicenter Phase II Trial of Unrelated Donor Reduced Intensity Bone Marrow Transplantation (BMT) for Children with Severe Sickle Cell Disease

Year: 2016

Type: article

Abstract: Patients with severe sickle cell disease (SCD) experience organ damage, poor quality of life and premature mortality. BMT halts disease progression if donor erythropoiesis is established. BMT from matched sibling donors is effective, but most patients lack such donors. Myeloablative conditioning (MAC) can result in long term side effects such as infertility – a barrier to patient acceptance of BMT. The SCURT trial was a prospective, multicenter trial evaluating matched unrelated donor (MUD) BMT in children following reduced intensity conditioning (RIC).

Source: Transplantation and Cellular Therapy

DOI: 10.1016/j.bbmt.2015.11.407