title: Allogeneic hematopoietic stem cell transplantation in children with sickle cell disease.
abstract: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) represents the only curative treatment for sickle cell disease (SCD), being successful in around 85-90% of patients. Mortality and long-term morbidity (including infertility, gonadal failure, and chronic graft-vs.-host disease) associated with conventional approaches curtail the number of patients who undergo allo-HSCT. Recently, it has been demonstrated that cord blood is as effective as and possibly safer than bone marrow in pediatric patients with SCD. Likewise, transplant strategies based on the use of reduced-intensity regimens and the induction of mixed chimerism have been explored to decrease allo-HSCT short- and long-term complications.
authors.affiliation: Dipartimento di Ematologia ed Oncologia Pediatrica, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy. franco.locatelli@opbg.net
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.forename: Daria
authors.initials: D
authors.lastname: Pagliara
mesh_terms: D000755:Anemia, Sickle Cell / Q000628:therapy*; D002648:Child; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous