Article
Sequential Treatment of Chronic Myeloid Leukemia Patients: A Decision-analytic Cost-effectiveness Study
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Authors
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Ursula Rochau
- Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology/ Area 4 Health Technology Assessment and Bioinformatics, ONCOTYROL - Center for Personalized Cancer Medicine, Hall i. T./ Innsbruck, Austria
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Gaby Sroczynski
- Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology/ Area 4 Health Technology Assessment and Bioinformatics, ONCOTYROL - Center for Personalized Cancer Medicine, Hall i. T./ Innsbruck, Austria
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Dominik Wolf
- Internal Medicine V, Hematology and Oncology, Medical University Innsbruck/ Internal Medicine III, University of Bonn, Innsbruck/ Bonn, Austria
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Stefan Schmidt
- Internal Medicine V, Hematology and Oncology, Medical University Innsbruck, Innsbruck, Austria
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Beate Jahn
- Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall i. T., Austria
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Annette Conrads-Frank
- Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology, Hall i. T., Austria
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David Stenehjem
- Department of Pharmacotherapy and Program in Personalized Health Care, University of Utah/ Huntsman Cancer Institute, University of Utah Hospitals & Clinics, Salt Lake City, United States
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Diana Brixner
- Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology/ Area 4 Health Technology Assessment and Bioinformatics, ONCOTYROL - Center for Personalized Cancer Medicine/ Department of Pharmacotherapy and Program in Personalized Health Care, University of Utah, Hall i. T./ Innsbruck/ Salt Lake City, Austria
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Jerald Radich
- Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, United States
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Guenther Gastl
- Internal Medicine V, Hematology and Oncology, Medical University Innsbruck, Innsbruck, Austria
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Uwe Siebert
- Institute of PH, MDM and HTA, Department of Public Health and Health Technology Assessment, UMIT - University for Health Sciences, Medical Informatics and Technology/ Area 4 HTA and Bioinformatics, ONCOTYROL - Center for Personalized Cancer Medicine/ Center for Health Decision Science, Department of Health Policy and Management, Harvard School of Public Health/ Institute for Technology Assessment and Department of Radiology, Massachusetts General Hospital, Harvard Medical School, Hall i. T./ Innsbruck/ Boston, Austria
| Published: | March 10, 2014 |
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Outline
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Purpose: The introduction of the tyrosine kinase inhibitor (TKI) imatinib dramatically extended the overall survival of chronic myeloid leukemia (CML) patients. Currently, there are several different TKIs approved and recommended for CML therapy. The aim of our study was to evaluate the long-term cost-effectiveness of different therapy regimens for CML within the Austrian health care context.
Methods: We performed a cost-effectiveness analysis using a state-transition Markov model. The model evaluates seven treatment strategies of differential sequencing of TKIs as well as chemotherapy or stem cell transplantation. For model parameters, we used published trial data, data from the Austrian CML registry, statistical and economic databases. We performed a cohort simulation over a lifelong time horizon, adopted a societal perspective with an annual 3% discount rate. Evaluated outcomes included life expectancy, quality-adjusted life years (QALYs), life-time costs, and discounted incremental cost-effectiveness/utility ratios (ICER/ICUR). We performed extensive univariate, and multivariate probabilistic sensitivity analyses (PSA) as well as a scenario analysis for generic drug pricing of imatinib.
Results: In the base-case efficiency frontier, nilotinib yielded an ICUR of 120,400 €/QALY (1) and an ICER of 107,000 €/LY compared to the baseline strategy imatinib without 2nd-line TKI. Imatinib followed by nilotinib after failure resulted in 129,500 €/QALY (2) and was dominated in terms of LY/€ compared to nilotinib without 2nd-line TKI. Nilotinib followed by dasatinib yielded an ICUR of 151,500 €/QALY (3) compared to imatinib followed by nilotinib after failure and an ICER of 192,400 €/LY compared to nilotinib without 2nd-line TKI. The remaining three strategies were excluded due to dominance. The PSA resulted in mean ICURs of 122,400 (1), 132,900 (2) and 153,300 €/QALYs (3). Deviations of the mean PSA results from the deterministic analysis range from 1.2% (3) to 2.6% (2).
Conclusions: Our analysis revealed high ICERs for the treatment of CML with TKIs. In Austria, the sequential application of TKIs is standard of care and withholding a 2nd-line TKI would not be acceptable. Therefore, based on our analysis we recommend imatinib followed by nilotinib as the most cost-effective treatment strategy. Mean results from PSA show only small deviation from the base-case analysis. When new path to cure treatment strategy data are available, results will need to be updated.
