Health state utilities associated with treatment for transfusion-dependent β-thalassemia.

ABSTRACT:

Objectives

Transfusion-dependent β-thalassemia (TDT) is a genetic disease that affects production of red blood cells. Conventional treatment involves regular red blood cell transfusions and iron chelation, which has a substantial impact on quality of life. While potentially curative, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is associated with risk of complications, including graft-versus-host disease (GvHD). Gene addition therapy, a novel treatment approach, involves autologous transplantation of the patient's own genetically modified hematopoietic stem cells. The purpose of this study was to estimate utilities associated with treatment approaches for TDT.

Methods

General population respondents in England valued eight health state vignettes (developed w

SUBMITTER: Matza LS 

PROVIDER: S-EPMC7188724 | biostudies-literature | 2020 Apr

REPOSITORIES: biostudies-literature