title: HLA-matched family hematopoetic stem cell transplantation in children with beta thalassemia major: the experience of the Turkish Pediatric Bone Marrow Transplantation Group.
abstract: From January 1991 to June 2009, 245 children with beta thalassemia major who underwent their first allogeneic HSCT in Turkey and who were followed for a minimum of one yr post-transplantation were enrolled this study. The median age of the patients was 6.6 yr old (range, 1-22 yr). The distribution of Pesaro risk class I, II, and III categories was 41, 130, and 63 children, respectively. The median serum ferritin level was 2203 ng/mL. Eighty-eight patients received bone marrow (BM) stem cells; 137, peripheral blood (PB) stem cells; and 20, cord blood (CB) stem cells. The donors were HLA-matched siblings or parents. Median engraftment times were shorter in PBSCT patients compared with the BMT group (p < 0.001). Grade II-IV acute GvHD was observed in 33 children (13.5%), while cGvHD was observed in 28 patients (12.5%), eight of whom had the extensive form. Thalassemic reconstitution was observed in 43 (17%) of the transplant patients. Post-transplant aplasia occurred in three patients, and the TRM rate was 7.75%. Seventeen patients were lost after 100 days. The thalassemia-free survival and OS rates were 68% (95% CI, 61.8-74.2) and 85.0% (95% CI, 80.2-89.8), respectively. We believe that this study is important because it is the first multicenter national data for children with beta thalassemia major receiving HSCT.
authors.affiliation: Akdeniz University School of Medicine, Antalya, Turkey. yesilipek@akdeniz.edu.tr
authors.forename: M Akif
authors.initials: MA
authors.lastname: Yesilipek
authors.forename: Mehmet
authors.initials: M
authors.lastname: Ertem
authors.forename: Mualla
authors.initials: M
authors.lastname: Cetin
authors.forename: Haldun
authors.initials: H
authors.lastname: Öniz
authors.forename: Savas
authors.initials: S
authors.lastname: Kansoy
authors.forename: Atila
authors.initials: A
authors.lastname: Tanyeli
authors.forename: Sema
authors.initials: S
authors.lastname: Anak
authors.forename: Emin
authors.initials: E
authors.lastname: Kurekci
authors.forename: Volkan
authors.initials: V
authors.lastname: Hazar
mesh_terms: D000293:Adolescent; D000328:Adult; D001854:Bone Marrow Cells / Q000166:cytology; D016026:Bone Marrow Transplantation / Q000379:methods; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D006086:Graft vs Host Disease / Q000209:etiology / Q000628:therapy; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D013997:Time Factors; D016896:Treatment Outcome; D014421:Turkey; D055815:Young Adult; D017086:beta-Thalassemia / Q000276:immunology* / Q000628:therapy