Summary:Fifteen patients with beta-thalassemia received an allogeneic peripheral blood stem cell transplant. Median age was 3.5 years (1-15 years). Six were class I, four class II and five class III according to the Pesaro criteria. All of the donors were HLA-phenotypically identical (13 siblings and two parents). Nine patients were given BU ؉ CY and six BU ؉ CY plus ATG as conditioning. All patients received MTX (؉1, ؉3, ؉6) and CsA (9-12 months) post transplant for GVHD prophylaxis. The median neutrophil and platelet engraftment times were day 12 and day 16, respectively. cGVHD was observed in three patients. Two patients died. Thirteen patients are well, and transfusion-independent 2-30 months after PSCT. No recurrences of thalassemia have been seen. Overall and event-free survival were 86.6%. In conclusion, we suggest that PSCT can be considered a safe and effective treatment for children with betathalassemia. Bone Marrow Transplantation (2001) 28, 1037-1040. Keywords: peripheral blood stem cell transplantation; beta-thalassemia; children Stem cell transplantation is considered the only curative treatment approach for the hemoglobinopathies. Successful cure of -thalassemia by bone marrow transplantation (BMT) was first reported by Thomas and associates in 1982. 1 Subsequently, a number of centers have explored this approach as a curative therapy for -thalassemia. The most extensive experience with BMT in -thalassemia was reported by Lucarelli and co-workers 2 in Italy who studied more than 800 transplanted patients. Recently, peripheral blood stem cells (PBSC) have been increasingly used in transplantation. Some authors have suggested that peripheral blood stem cell transplantation (PSCT) has some advantages as compared with bone marrow stem cell transplantation. geneic transplantation appears to be safe for both pediatric donors and patients, leading to rapid hematopoietic engraftment with a similar incidence of acute graft-versushost disease (AGVHD) as is seen after BMT.We report the PSCT experience of our department concerning 15 patients with beta-thalassemia.
Materials and methodsFifteen patients with beta-thalassemia received an allogeneic peripheral blood stem cell transplant at Akdeniz University School of Medicine Department of Paediatric Haematology-Oncology. Patient characteristics are shown in Table 1. All of the patients were transfusion-dependent before stem cell transplantation. Fourteen patients had homozygous beta-thalassemia and one had HbS-betathalassemia. Female to male ratio was 9/6 and median age of the patients was 3.5 years (range 1-15 years). Liver biopsy was performed on all patients who were older than 3 years. Six of them were class I, four class II and five were class III according to the Pesaro criteria. 6 All but two patients were HbS antibody-positive. Only one patient (No. 4) was HCV antibody-positive. Thirteen of the donors were fully HLA-matched siblings and two were parents
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