Cost-Effectiveness of Chimeric Antigen Receptor T-Cell Therapy in Pediatric Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia
| Autor: | Deborah Schiff, Reith R Sarkar, James D. Murphy, Nicholas J. Gloude |
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| Rok vydání: | 2018 |
| Předmět: |
Male
Oncology Cancer Research medicine.medical_specialty Cost effectiveness Cost-Benefit Analysis medicine.medical_treatment Receptors Antigen T-Cell Immunotherapy Adoptive Pediatrics Disease-Free Survival 03 medical and health sciences 0302 clinical medicine Quality of life Refractory Internal medicine medicine Humans Child health care economics and organizations B-Lymphocytes business.industry Complete remission Immunotherapy Precursor Cell Lymphoblastic Leukemia-Lymphoma Chimeric antigen receptor Quality-adjusted life year 030220 oncology & carcinogenesis Female Chimeric Antigen Receptor T-Cell Therapy Quality-Adjusted Life Years business |
| Zdroj: | JNCI: Journal of the National Cancer Institute. 111:719-726 |
| ISSN: | 1460-2105 0027-8874 |
| DOI: | 10.1093/jnci/djy193 |
| Popis: | BackgroundChimeric antigen receptor T-cell (CAR-T) therapy is a promising new class of cancer therapy but has a high up-front cost. We evaluated the cost-effectiveness of CAR-T therapy among pediatric patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL).MethodsWe built a microsimulation model for pediatric patients with relapsed/refractory B-ALL receiving either CAR-T therapy or standard of care. Outcomes included costs, quality of life (health utility), complications, and survival. We measured cost-effectiveness with the incremental cost-effectiveness ratio (ICER), with ICERs under $100 000 per quality-adjusted life-year (QALY) considered cost effective. One-way and probabilistic sensitivity analyses were used to test model uncertainty.ResultsCompared to standard of care, CAR-T therapy increased overall cost by $528 200 and improved effectiveness by 8.18 QALYs, resulting in an ICER of $64 600/QALY. The model was sensitive to assumptions about long-term CAR-T survival, the complete remission rate of CAR-T patients, and the health utility of long-term survivors. The base model assumed a 76.0% one-year survival with CAR-T, although if this decreased to 57.8%, then CAR-T was no longer cost effective. If the complete remission rate of CAR-T recipients decreased from 81% to 56.2%, or if the health utility of disease-free survivors decreased from 0.94 to 0.66, then CAR-T was no longer cost effective. Probabilistic sensitivity analysis found that CAR-T was cost effective in 94.8% of iterations at a willingness to pay of $100 000/QALY.ConclusionCAR-T therapy may represent a cost-effective option for pediatric relapsed/refractory B-ALL, although longer follow-up of CAR-T survivors is required to confirm validity of these findings. |
| Databáze: | OpenAIRE |
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