Beyond transfusion therapy: new therapies in thalassemia including drugs, alternate donor transplant, and gene therapy.

ABSTRACT: Transfusion combined with chelation therapy for severe β thalassemia syndromes (transfusion-dependent thalassemia [TDT]) has been successful in extending life expectancy, decreasing comorbidities and improving quality of life. However, this puts lifelong demands not only on the patients but also on the health care systems that are tasked with delivering long-term treatment and comprehensive support. Prevention programs and curative approaches are therefore an important part of overall strategy. Curative treatments alter the dynamic of a patient's health care costs, from financial commitment over 50 years, into a potential "one-off" investment. Since the 1980s, this has usually been available only to the 30% or so of young children with matched sibling donors. By improving the safety of mat

SUBMITTER: Porter J 

PROVIDER: S-EPMC6245990 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature