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  • Title: Peripheral blood stem cell transplantation in children with beta-thalassemia major.
    Author: Farzana T, Shamshi TS, Irfan M, Ansari SH, Baig MI, Shakoor N.
    Journal: J Coll Physicians Surg Pak; 2003 Apr; 13(4):204-6. PubMed ID: 12718774.
    Abstract:
    OBJECTIVE: To share the preliminary data on stem cell transplantation in Pakistan. DESIGN: This is a single center retrospective analysis of the outcome of allogeneic peripheral blood stem cell (PBSC) transplantation in b-thalassemia major patients. PLACE AND DURATION OF STUDY: Bismillah Taqee Blood Diseases Centre, Karachi; a tertiary care hematology center from September 1999 to September 2002. PATIENTS AND 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. CONCLUSION: 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.
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