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Indian Journal of Medical and Paediatric Oncology
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REVIEW ARTICLE
Year : 2008  |  Volume : 29  |  Issue : 1  |  Page : 39-48

Hematopoietic stem cell transplantation for hemoglobinopathies: Progress and prospects


Department of Comprehensive Hemoglobinopathies Program Division of Hematology/Oncology/BMT Children's Hospital of Pittsburgh 3705 Fifth Avenue, Pittsburgh, PA 15213

Correspondence Address:
Lakshmanan Krishnamurti
Department of Comprehensive Hemoglobinopathies Program Division of Hematology/Oncology/BMT Children's Hospital of Pittsburgh 3705 Fifth Avenue, Pittsburgh, PA 15213

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Source of Support: None, Conflict of Interest: None


DOI: 10.4103/0971-5851.51444

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Allogeneic bone marrow transplantation from an HLA-identical donor is currently the only means of curing thalassemia . Transplant outcome depends upon the presence of risk factors (hepatomegaly, portal fibrosis and poor quality of chelation). patients are defined to have class I - if no risk factor, class II with one or two and class III - if all three risk factors are present. For patients under 16 years of age, for class I, class II and III the probabilities of survival are approximately 95%, >80% and 60-70%, respectively. The risk of transplant related morbidity& mortality is low when transplant is done at an early age. Currently, busulfan, cyclophospahmide and antithymocyte globulin based combination is used for conditioning. More than 200 patients with sickle cell disease (SCD) have undergone allogeneic SCT with long term survival in >80% of patients. Results are better if donor is an HLA-identical sibling and if transplant is done early in the course of disease. Presently, experience with reduced intensity SCT and matched unrelated donor transplant is limited to recommend their routine use.


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