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- W2380329188 abstract "Objective To investigate the effect of hematopoietic stem cell transplantation(HSCT) for β-thalassemia major. Methods Fifteen β-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. Result 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 unengrafted. Two patients received unrelated cord blood HSCT were both unengrafted. 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. Conclusion Grade Ⅰ～Ⅱ β-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." @default.
- W2380329188 created "2016-06-24" @default.
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- W2380329188 date "2003-01-01" @default.
- W2380329188 modified "2023-09-24" @default.
- W2380329188 title "Allogeneic hematopoietic stem cell transplantation for β-thalassemia major" @default.
- W2380329188 hasPublicationYear "2003" @default.
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