title: Peripheral blood stem cell transplantation in children with beta-thalassemia major.
abstract: OBJECTIVE
  To share the preliminary data on stem cell transplantation in Pakistan.
  
  
  METHODS
  This is a single center retrospective analysis of the outcome of allogeneic peripheral blood stem cell (PBSC) transplantation in b-thalassemia major patients.
  
  
  METHODS
  Bismillah Taqee Blood Diseases Centre, Karachi; a tertiary care hematology center from September 1999 to September 2002.
  
  
  METHODS
  Twelve patients with b-thalassemia major received allogeneic PBSC transplantation from HLA identical sibling donors except in one case where mother was the donor. All patients received busulphan and cyclophosphamide conditioning therapy and cyclosporin A/methotrexate for graft versus host disease (GVHD) prophylaxis. Donors were primed with G-CSF for 4 days and stem cells were harvested using Haemonetics MCS + cell separator on 5th day. All patients received G-CSF starting from day + 4 until their neutrophil count rose to normal.
  
  
  RESULTS
  Engraftment was achieved in all patients except one who required a second dose of bone marrow graft on day +21. Median time to achieve absolute neutrophil count of > 0.5 x 10(9)/l was 9.0 days (range 8-31 days) and platelet count of > 20 x 10(9)/l was 14 days (12-35 days). Acute GVHD was seen in 3 patients, one patient had grade IV gut GVHD; another patient had grade III gut GVHD while third patient had grade II skin GVHD. Median hospital stay was 29 days. Six patients were well and transfusion independent 3 to 36 months posttransplant. One episode of primary graft failure required a second dose of bone marrow harvest. Another episode of graft rejection received two doses of donor lymphocytes infusion. There were 4 deaths due to grade IV gut GVHD because of uncontrolled systemic Candida infection and one due to hepatic veno-occlusive (VOD) disease.
  
  
  CONCLUSIONS
  Allogeneic peripheral blood stem cell transplantation can be safely and economically carried out in Pakistan. Although there had been 4 deaths during 36 months follow-up, with increasing understanding and experience the outcome is expected to improve.
authors.affiliation: Department of Haematology, Bismillah Taqee Blood Diseases Centre, Karachi.
authors.forename: T
authors.initials: T
authors.lastname: Farzana
authors.forename: T S
authors.initials: TS
authors.lastname: Shamshi
authors.forename: M
authors.initials: M
authors.lastname: Irfan
authors.forename: S H
authors.initials: SH
authors.lastname: Ansari
authors.forename: M I
authors.initials: MI
authors.lastname: Baig
authors.forename: N
authors.initials: N
authors.lastname: Shakoor
mesh_terms: D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease / Q000517:prevention & control; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D010154:Pakistan / Q000453:epidemiology; D036102:Peripheral Blood Stem Cell Transplantation; D012189:Retrospective Studies; D019172:Transplantation Conditioning; D017086:beta-Thalassemia / Q000453:epidemiology / Q000628:therapy*
chemical_list: D007166:Immunosuppressive Agents