Auteurs : Séverine Coquerelle, Mariem Ghardallou, Setti Rais, Pierre Taupin, Fabien Touzot, Laure Boquet, Stéphane Blanche, Semir Benaouadi, Thomas Brice, Caroline Tuchmann-Durand, Jean Antoine Ribeil, Elisa Magrin, Etienne Lissillour, Lise Rochaix, Marina Cavazzana, and Isabelle Durand-Zaleski
Article publié dans : Human Gene Therapy, Volume 30, Issue 6, June 12, 2019
Date de parution : 2019
DOI : https://doi.org/10.1089/hum.2018.178
Abstract : Seventy-five percent of patients with beta thalassemia (β-thalassemia) do not have human leukocyte antigen–matched siblings and until recently had no access to a curative treatment. Gene therapy is a promising treatment that can be proposed to these patients. This study estimates its cost and efficacy. In a monocentric retrospective study and cost-efficacy analysis, this study compared the two-year outcomes and costs of patients with β-thalassemia treated by gene therapy and hematopoietic stem-cell transplantation (HSCT). Grade III and grade IV complications, hospitalizations, and length of stay were extracted from the hospital discharge data. Costs were estimated from hospital accounting information and national cost studies. A total of seven patients with β-thalassemia treated between 2009 and 2016 were included, of whom four received gene therapy. Patients treated by gene therapy were older and had fewer complications and hospital admissions. Infectious complications were three times more frequent for patients treated with HSCT than for gene therapy. Average costs were €608,086 for patients treated by gene therapy and €215,571 for HSCT. The total cost of the vector was 48% of the total cost of gene therapy. Gene therapy as a curative alternative for patients lacking human leukocyte antigen–matched donors was costlier but resulted in fewer complications than HSCT.