Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation.

Authors:
Severine Coquerelle
Severine Coquerelle
Assistance Publique - Hopitaux de Paris
Mariem Ghardallou
Mariem Ghardallou
Assistance Publique - Hopitaux de Paris
Setti Rais
Setti Rais
Hospinomics
Pierre Taupin
Pierre Taupin
Université Paris-Descartes
France
Fabien Touzot
Fabien Touzot
Necker Hospital
France
Laure Boquet
Laure Boquet
Institut Imagine
Stephane Blanche
Stephane Blanche
Université Paris Descartes
France
Semir Benaouadi
Semir Benaouadi
Hopital universitaire Necker-Enfants malades

Hum Gene Ther 2019 May 3. Epub 2019 May 3.

1 URC Eco, Assistance Publique-Hôpitaux de Paris, Paris, France; Henri Mondor Teaching Hospital, Créteil, France.

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.

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http://dx.doi.org/10.1089/hum.2018.178DOI Listing
May 2019
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