Abstract
TDT is a rare genetic disease with impaired production of healthy red blood cells due to insufficient adult hemoglobin (HbA) production. The resulting ineffective erythropoiesis, and iron overload from chronic transfusions, makes this a complex disease. Cost-utility analyses in TDT to-date use health-state utility values that have not been compared to disease-specific quality of life (QoL) measures. This study aimed to contextualize TDT utility-value estimates with disease-relevant data.
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