Saturday, 30 Sep 2023

Estimating the efficacy and cost of curative gene therapy for beta-thalassemia

Gene therapy offers the promise of a cure for beta-thalassemia and a new study has shown that it is associated with fewer complications and hospital admissions over 2 years than treatment by allogeneic hematopoietic stem cell transplantation (HSCT). The study, which analyzes and compares the effectiveness and cost of gene therapy versus (HSCT) in patients with major beta-thalassemia is published in Human Gene Therapy.

Séverine Coquerelle, URC Eco-Assistance Publique Hôpitaux de Paris, Université Paris Diderot (Sorbonne Paris Cité), and CRESS, INSERM UMR (Paris), France led a team of French researchers in publishing the article entitled “Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation.” Patients treated with HSCT had 3 times more frequent infectious complications. Gene therapy was shown to be about 2.8 times more costly, with nearly half the cost of gene therapy accounted for by preparation of the delivery vector.

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