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Cord Blood Forum: Annotated Bibliography- Thalessemia-Clinical Aspects

Source: www.cordbloodforum.org
Topic: Thalassemia
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Sort Desciption: In this concise editorial and commentary, the author points out that it is gratifying that, with modern supportive therapy, 68% of patients with beta-thalassemia are alive at the age of 35. ...

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In this concise editorial and commentary, the author points out that it is gratifying that, with modern supportive therapy, 68% of patients with beta-thalassemia are alive at the age of 35. The most frequent causes of death are still heart failure and/or cardiac arrhythmia, which are mostly caused by myocardial iron overload. The second cause is intercurrent infections, and thrombotic events are emerging as an important complication and relatively frequent cause of death. Many patients still have cardiac disease and delayed pubertal development, and develop hypogonadism, hypothyroidism, hypoparathyroidism, and diabetes mellitus. Data on the survival of patients and on quality of life are very useful in the genetic counseling of couples at risk of having betathalassemic offspring when planning a pregnancy, and in the discussion, with the parents of an affected child, on the available options for treatment of a newly diagnosed case. The pros and cons of traditional treatment should be evaluated vis-a-vis the results of bone marrow or cord blood stem cell transplantation. In this counseling, it should be pointed out that the only treatment that may lead to a definitive cure in thalassemia major is stem cell transplantation which, even in the best conditions, is still associated with a mortality of 5% when performed from an HLA identical family donor. (Comment: The author indicates that, with modern supportive etherapy, 32% of patients will die by the age of 35. The question that immediately comes to mind is, If 100 children with beta-thalassemia major were to be transplanted at an early age, would 32 be dead at the age of 35? Transplants of ...

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