title: Allogeneic peripheral blood stem cell transplantation in children with homozygous beta-thalassemia and severe beta-thalassemia/hemoglobin E disease.
abstract: To determine the outcome of children with homozygous beta-thalassemia (beta/beta) and severe beta-thalassemia/hemoglobin E disease (beta/E) who underwent allogeneic peripheral blood stem cell transplantation (PBSCT). The authors conducted a cohort study of allogeneic PBSCT in beta/beta and beta/E patients who had 6/6 or 5/6 HLA-matched sibling donors. All patients received a conditioning regimen including busulfan and cyclophosphamide, except one who received busul-fan and cyclophosphamide plus antithymocyte globulin. Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine A and methotrexate for eight patients and cyclosporine and mycophenolate mofetil for one patient. Donors received G-CSF for 4 days before leukapheresis collections. There were five beta/beta and four beta/E patients in this study. The median age was 9 years (range 1.5-10 years). The median CD34+ cell count was 7.4 x 10(6) cells/kg recipient body weight. All patients achieved neutrophil and platelet engraftment with a median time of 15 days and 21 days respectively. Acute GVHD grade 2 to 4 appeared in four patients (grade 2, n = 3; grade 4, n = 1). Three patients developed chronic GVHD (limited, n = 2; extensive, n = 1). All patients were alive with a median follow-up time of 23 months (range 7-52 months). Neither graft failure nor graft rejection was observed. Allogeneic PBSCT is feasible for children with beta/beta and beta/E, although the incidence of GVHD was apparently high compared with bone marrow transplant study in Thais.
authors.affiliation: Department of Pediatrics, Faculty of Medicine at Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
authors.forename: Samart
authors.initials: S
authors.lastname: Pakakasama
authors.forename: Suradej
authors.initials: S
authors.lastname: Hongeng
authors.forename: Wattana
authors.initials: W
authors.lastname: Chaisiripoomkere
authors.forename: Ampaiwan
authors.initials: A
authors.lastname: Chuansumrit
authors.forename: Nongnuch
authors.initials: N
authors.lastname: Sirachainun
authors.forename: Saengsuree
authors.initials: S
authors.lastname: Jootar
mesh_terms: D001803:Blood Transfusion; D002648:Child; D015331:Cohort Studies; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control; D006446:Hemoglobin E; D006650:Histocompatibility Testing; D006720:Homozygote; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007937:Leukapheresis; D007958:Leukocyte Count; D008099:Liver / Q000473:pathology; D008297:Male; D010976:Platelet Count; D013156:Splenectomy; D033581:Stem Cell Transplantation; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000235:genetics / Q000628:therapy*
chemical_list: D007166:Immunosuppressive Agents; D006446:Hemoglobin E