| Literature DB >> 31416718 |
Valeria Maria Pinto1, Maurizio Poggi2, Rodolfo Russo3, Andrea Giusti4, Gian Luca Forni5.
Abstract
Thalassemia is among the most common monogenic diseases worldwide. Stem cell transplantation can be curative but is reserved for young patients, as probably gene therapy will be in the future. Adult thalassemia patients are treated with transfusion therapy and iron chelation, and improvements in the safety of transfusion protocols, use of iron chelation, monitoring of iron overload, and management of comorbidities have substantially prolonged survival, increasing the proportion of adult patients in the thalassemic population. However, older patients are more likely to develop multiple disease-related morbidities, including osteoporosis, endocrine disorders, liver disease, renal dysfunction, and cancer. Thus, the main objective of this article is to describe new challenges posed by the increasing life expectancy of patients with thalassemia, focusing on data from Italy where there is a well-documented history of thalassemia management. It is hoped that the mortality and morbidity benefits already seen in patients with thalassemia will continue to improve with ongoing advances in the quality of treatment.Entities:
Keywords: Anemia; Hemoglobinopathy; Iron chelation; Iron overload; Thalassemia; Transfusion
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Year: 2019 PMID: 31416718 DOI: 10.1016/j.blre.2019.100594
Source DB: PubMed Journal: Blood Rev ISSN: 0268-960X Impact factor: 8.250