Nonmyeloablative Matched Sibling Donor Hematopoietic Cell Transplantation in Children and Adolescents with Sickle Cell Disease

Hematopoietic cell transplantation (HCT) is the only established curative therapy for sickle cell disease (SCD). Rates of disease-free survival exceed 95% with a matched sibling donor (MSD) using myeloablative conditioning (MAC) [1]. However, busulfan-based conditioning regimens are associated with substantial morbidity and high rates of infertility, particularly in adolescent females; recipients older than 16 years of age have poorer outcomes [1,2]. Graft-versus-host disease (GVHD) remains a challenge and a major reason not all children and adolescents with an MSD are offered HCT [1,2].

Source: Biology of Blood and Marrow Transplantation - February 13, 2019 Category: Hematology Authors: Gregory M.T. Guilcher, Dania A. Monagel, Alberto Nettel-Aguirre, Tony H. Truong, Sunil J. Desai, Aisha Bruce, Ravi M. Shah, Michael T. Leaker, Victor A. Lewis Source Type: research