Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation Se ´ verine Coquerelle, 1–3, * Mariem Ghardallou, 1 Setti Rais, 4,5 Pierre Taupin, 6 Fabien Touzot, 6,7 Laure Boquet, 5 Ste ´ phane Blanche, 6 Semir Benaouadi, 6 Thomas Brice, 6 Caroline Tuchmann-Durand, 5 Jean Antoine Ribeil, 6,{ Elisa Magrin, 5 Etienne Lissillour, 6 Lise Rochaix, 4,8 Marina Cavazzana, 5,9,10 and Isabelle Durand-Zaleski 1,3,11 1 URC Eco, Assistance Publique—Ho ˆ pitaux de Paris, Paris, France; 2 Universit ´ e Paris Diderot, Sorbonne Paris Cit ´ e, France; 3 CRESS, INSERM UMR 1153, Paris, France; 4 Hospinnomics (Paris School of Economics, Assistance Publique—Ho ˆ pitaux de Paris), Paris, France; 5 Institut Imagine, Paris, France; 6 Ho ˆ pital Necker—Enfants Malades, Assistance Publique—Ho ˆ pitaux de Paris, Paris, France; 7 De ´partement de Pe ´diatrie - Centre de Recherche du CHU Sainte-Justine, Montre ´al, Canada; 8 Universite ´ Paris I Sorbonne, Paris, France; 9 D´ epartement de Bioth ´ erapie, Centre d’Investigation Clinique Int ´ egr ´ e en Bioth ´ erapies, Ho ˆ pital Necker—Enfants Malades, Assistance Publique—Ho ˆ pitaux de Paris, Paris, France; 10 Universit ´ e Paris Descartes, Sorbonne Paris Cit ´ e, Institut Imagine, Paris, France; and 11 AP-HP Department of Public Health, Henri Mondor Teaching Hospital, Cr ´ eteil, France. { Current address: bluebird bio Ò , Paris, France. Seventy-five percent of patients with beta thalassemia (b-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 b-thalassemia treated by gene therapy and hematopoietic stem-cell trans- plantation (HSCT). Grade III and grade IV complications, hospitalizations, and length of stay were ex- tracted from the hospital discharge data. Costs were estimated from hospital accounting information and national cost studies. A total of seven patients with b-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 pa- tients treated with HSCT than for gene therapy. Average costs were e608,086 for patients treated by gene therapy and e215,571 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. Keywords: beta thalassemia, gene therapy, HSCT, cost-efficacy analysis BACKGROUND THE MANAGEMENT OF BETA THALASSEMIA (b-thalass- emia) requires lifelong red blood cell transfusion, iron chelation, splenectomy, or allogeneic hematopoietic stem-cell transplantation (HSCT), 1 all of which im- pose a significant patient burden and high costs to healthcare systems. 2,3 HSCT was until recently the only existing curative treatment and requires a hu- man leukocyte antigen (HLA) identical donor. 4,5 The concept of gene therapy for b-thalassemia has moved from bench to bedside and is a promising treatment approach for patients with no matching donor available. 6–9 While long-term studies are underway in multiple centers in Europe and the United States, proof-of-principle of efficacy and safety has already been obtained in patients with b-thalassemia. 10–12 In addition to safety and effectiveness, the cost of gene therapy versus other options needs to be con- sidered. 13,14 Both gene therapy and HSCT are cu- rative options for b-thalassemia. From a health policy perspective, comparing gene therapy to an- other curative option such as HSCT makes sense, as *Correspondence: Se´verine Coquerelle, URC-Eco, Assistance Publique—Ho ˆpitaux de Paris (AP-HP), Ho ˆtel Dieu, 1 Place du Parvis Notre Dame, 75004 Paris, France. E-mail: severine.coquerelle@urc-eco.fr HUMAN GENE THERAPY, VOLUME 30 NUMBER 6 DOI: 10.1089/hum.2018.178 j 753 ª 2019 by Mary Ann Liebert, Inc. Downloaded by 54.162.69.248 from www.liebertpub.com at 06/30/20. For personal use only.