Clinical therapeutics
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Clinical therapeutics · Feb 2020
Systematic Literature Review of the Burden of Disease and Treatment for Transfusion-dependent β-Thalassemia.
β-Thalassemia is an inherited blood disorder characterized by reduced or no production of adult hemoglobin. Systematic identification of the burden of β-thalassemia with contemporary treatments is lacking in published literature. Thus, a gap exists in understanding the baseline burden on which to assess future treatments. Therefore, a systematic literature review (SLR) was performed to assess management and outcomes in patients with transfusion-dependent β-thalassemia (TDT) who received long-term transfusion regimens. ⋯ Consistent with improvements in transfusion practices and iron monitoring and management, outcomes in patients with TDT have improved. However, iron overload and disease-associated complications remain a challenge in this population. This review supports the burden of disease affecting patients with β-thalassemia and provides a baseline health status against which to assess future improvements in care.