A paradigm shift on beta-thalassaemia treatment: How will we manage this old disease with new therapies?
Beta-thalassaemia causes defective haemoglobin synthesis leading to ineffective erythropoiesis, chronic haemolytic anaemia, and subsequent clinical complications. Blood transfusion and iron chelation allow long-term disease control, and hematopoietic stem cell transplantation offers a potential cure for some patients. Nonetheless, there are still many challenges in the management of beta-thalassaemia. The main treatment option for most patients is supportive care; furthermore, the long-term efficacy and safety of current therapeutic strategies are limited and adherence is suboptimal.
Source: Blood Reviews - Category: Hematology Authors: Maria Domenica Cappellini, John B. Porter, Vip Viprakasit, Ali T. Taher Tags: Review Source Type: research
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