Back to Search
Start Over
Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell Transplantation
- Source :
- Human Gene Therapy. 30:753-761
- Publication Year :
- 2019
- Publisher :
- Mary Ann Liebert Inc, 2019.
-
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.
- Subjects :
- medicine.medical_specialty
Adolescent
Total cost
Cost-Benefit Analysis
medicine.medical_treatment
Genetic enhancement
Genetic Vectors
Graft vs Host Disease
Hematopoietic stem cell transplantation
03 medical and health sciences
0302 clinical medicine
hemic and lymphatic diseases
Internal medicine
Genetics
medicine
Humans
Transplantation, Homologous
Vector (molecular biology)
Child
Molecular Biology
Retrospective Studies
030304 developmental biology
Clinical Trials as Topic
0303 health sciences
business.industry
beta-Thalassemia
Hematopoietic Stem Cell Transplantation
Infant
Beta thalassemia
Retrospective cohort study
Genetic Therapy
Health Care Costs
Prognosis
medicine.disease
Tissue Donors
Transplantation
Treatment Outcome
Curative treatment
Child, Preschool
030220 oncology & carcinogenesis
Molecular Medicine
business
Subjects
Details
- ISSN :
- 15577422 and 10430342
- Volume :
- 30
- Database :
- OpenAIRE
- Journal :
- Human Gene Therapy
- Accession number :
- edsair.doi.dedup.....c02cafb82d96f15b489eba945aad807e