title: Transplantation of a child with sickle cell anemia with an unrelated cord blood unit after reduced intensity conditioning.
abstract: Sickle cell disease can be corrected by hematopoietic cell transplantation but success is limited by low availability of matched related/unrelated donors and comorbidities leading to the increased transplant-related morbidity/mortality. There is a need for expanded donor pools and reduced intensity regimens. We describe a case of a second unrelated cord blood transplant after a novel preparative regimen in a child with sickle cell disease related stroke and liver fibrosis. Hydroxyurea, rituximab, and alemtuzumab were followed by thiotepa and low dose total body irradiation before unrelated cord blood transplant. Rapid full donor chimerism and improved performance status was achieved and sustained over 2 years after transplant.
authors.affiliation: Department of Pediatrics, Pediatric Blood and Marrow Transplant Program, Duke University Medical Center, Durham, NC 27705, USA.
authors.forename: Melissa
authors.initials: M
authors.lastname: Mazur
authors.forename: Joanne
authors.initials: J
authors.lastname: Kurtzberg
authors.forename: Edward
authors.initials: E
authors.lastname: Halperin
authors.forename: Gilbert
authors.initials: G
authors.lastname: Ciocci
authors.forename: Paul
authors.initials: P
authors.lastname: Szabolcs
mesh_terms: D000755:Anemia, Sickle Cell / Q000628:therapy*; D002648:Child; D036101:Cord Blood Stem Cell Transplantation; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D019653:Myeloablative Agonists / Q000008:administration & dosage*; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous
chemical_list: D019653:Myeloablative Agonists