부산대학교 도서관

The management of thalassaemia with a severe phenotype includes blood transfusion, iron chelation, bone marrow transplantation, prenatal diagnosis and national programmes to co-ordinate these in countries with a high prevalence. If blood transfusion and iron chelation therapy are not administered regularly, as was the case historically and as is still the case in many poorer regions, progressive deterioration occurs, viz. impaired growth and development, hepatosplenomegaly, bony abnormalities, cardiac failure, increased susceptibility to infections and premature mortality. Remarkable progress has been made in the past few decades, which has led to much-improved survival rates. Transfusion therapy has evolved to a hyper-transfusion regimen designed to maintain a physiological haemoglobin level and achieve a post-transfusion haemoglobin of 14 g/dL, which, as a matter of course, necessitated intensification of iron chelation. The development of effective oral iron chelators has led to improved compliance. Exploration of novel therapeutic approaches continues, with several agents under study. The prospect of gene therapy is particularly exciting as it has potential to provide cure on a large scale. Currently, regular blood transfusion and iron chelation therapy remain the cornerstone of management of thalassaemia major. https://doi.org/10.7196/SAMJ.2021.v111i9.15898
Classification, pathophysiology and diagnosis of thalassaemia are discussed in part 1 [1] of this 2-part CME series. The current article (part 2) discusses management aspects relating to the three clinical [...]