title: [Allogeneic hematopoietic stem cell transplantation for beta-thalassemia major].
abstract: OBJECTIVE
  To investigate the effect of hematopoietic stem cell transplantation (HSCT) for beta-thalassemia major.
  
  
  METHODS
  Fifteen beta-thalassemia major patients with a median age of 3. 5 years (range 1 - 10 years) received allogeneic HSCT. According to the Pesaro's classification for thalassemia, 12 patients were grade I - II, and 3 grade III. The bone marrow transplantation (BMT) plus peripheral blood stem cell (PBSC) transplant mobilized by granulocyte colony-stimulating factor (G-CSF) was used when donor is low body-weight.
  
  
  RESULTS
  Of the fifteen patients, nine were disease-free survival (DFS) at a median follow-up of 2.5 years (range 6 - 54 months). Of eight grade I - II patients received HLA identical sibling BMT, seven were DFS, and of two received HLA mismatched marrow from their mother, one DFS, another not engrafted. Two patients received unrelated cord blood HSCT were both not engrafted. Two patients received PBSC transplantation alone were not engrafted, but one of them soon received BMT from the same donor and was DFS. The incidences of grade I - II and grade III acute graft-versus-host disease (aGVHD) were 20% (3/15) and 6.7% (1/15), respectively. Interstitial pneumonia occurred in 4/15 (26.7%) patients. There were no long-term complications in the survivors.
  
  
  CONCLUSIONS
  Grade I - II beta-thalassemia major patients received HLA identical sibling BMT had higher DFS. It was propitious for engraftment to use BMT plus PBSC, but with a higher incidence of acute and chronic GVHD.
authors.affiliation: Department of Pediatrics, Nanfang Hospital, Guangzhou 510515, China.
authors.forename: Chun-fu
authors.initials: CF
authors.lastname: Li
authors.forename: Yue-lin
authors.initials: YL
authors.lastname: He
authors.forename: Yu-ming
authors.initials: YM
authors.lastname: Zhang
authors.forename: Ming
authors.initials: M
authors.lastname: Yang
authors.forename: Xue-dong
authors.initials: XD
authors.lastname: Wu
authors.forename: Jing
authors.initials: J
authors.lastname: Bai
mesh_terms: D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D019650:Hematopoietic Stem Cell Mobilization / Q000379:methods; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D008297:Male; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000628:therapy*