Hematopoietic stem cell transplantation for hemoglobinopathies: Progress and prospects

 

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Abstract

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.

Publication History

Article published online:
07 March 2022

© 2008. Indian Society of Medical and Paediatric Oncology. This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial-License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/.)

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