title: Effectiveness and Cost-Effectiveness of Sequential Treatment of Patients with Chronic Myeloid Leukemia in the United States: A Decision Analysis.
abstract: Currently several tyrosine kinase inhibitors (TKIs) are approved for treatment of chronic myeloid leukemia (CML). Our goal was to identify the optimal sequential treatment strategy in terms of effectiveness and cost-effectiveness for CML patients within the US health care context. We evaluated 18 treatment strategies regarding survival, quality-adjusted survival, and costs. For model parameters, the literature data, expert surveys, registry data, and economic databases were used. Evaluated strategies included imatinib, dasatinib, nilotinib, bosutinib, ponatinib, stem-cell transplantation (SCT), and chemotherapy. We developed a Markov state-transition model, which was analyzed as a cohort simulation over a lifelong time horizon with a third-party payer perspective and discount rate of 3%. Remaining life expectancies ranged from 5.4 years (3.9 quality-adjusted life years (QALYs)) for chemotherapy treatment without TKI to 14.4 years (11.1 QALYs) for nilotinib→dasatinib→chemotherapy/SCT. In the economic evaluation, imatinib→chemotherapy/SCT resulted in an incremental cost-utility ratio (ICUR) of $171,700/QALY compared to chemotherapy without TKI. Imatinib→nilotinib→chemotherapy/SCT yielded an ICUR of $253,500/QALY compared to imatinib→chemotherapy/SCT. Nilotinib→dasatinib→chemotherapy/SCT yielded an ICUR of $445,100/QALY compared to imatinib→nilotinib→chemotherapy/SCT. All remaining strategies were excluded due to dominance of the clinically superior strategies. Based on our analysis and current treatment guidelines, imatinib→nilotinib→chemotherapy/SCT and nilotinib→dasatinib→chemotherapy/SCT can be considered cost-effective for patients with CML, depending on willingness-to-pay.
authors.affiliation: Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, UMIT-University for Health Sciences, Medical Informatics and Technology, Eduard Wallnoefer Center 1, 6060 Hall in Tirol, Austria; Area Health Technology Assessment, ONCOTYROL-Center for Personalized Cancer Medicine, Karl-Kapferer-Straße 5, 6020 Innsbruck, Austria.
authors.forename: Ursula
authors.initials: U
authors.lastname: Rochau
authors.affiliation: Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, UMIT-University for Health Sciences, Medical Informatics and Technology, Eduard Wallnoefer Center 1, 6060 Hall in Tirol, Austria.
authors.forename: Martina
authors.initials: M
authors.lastname: Kluibenschaedl
authors.affiliation: Department of Pharmacotherapy, University of Utah, 30 South 2000, Salt Lake City, UT 84112, USA; Huntsman Cancer Institute, University of Utah Hospitals & Clinics, 2000 Circle of Hope, Salt Lake City, UT 84112, USA.
authors.forename: David
authors.initials: D
authors.lastname: Stenehjem
authors.affiliation: Department of Pharmacotherapy, University of Utah, 30 South 2000, Salt Lake City, UT 84112, USA.
authors.forename: Kuo
authors.initials: K
authors.lastname: Kuan-Ling
authors.affiliation: Clinical Research Division, Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue N, Seattle, WA 98104, USA.
authors.forename: Jerald
authors.initials: J
authors.lastname: Radich
authors.affiliation: Department of Pharmacotherapy, University of Utah, 30 South 2000, Salt Lake City, UT 84112, USA.
authors.forename: Gary
authors.initials: G
authors.lastname: Oderda
authors.affiliation: Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, UMIT-University for Health Sciences, Medical Informatics and Technology, Eduard Wallnoefer Center 1, 6060 Hall in Tirol, Austria; Area Health Technology Assessment, ONCOTYROL-Center for Personalized Cancer Medicine, Karl-Kapferer-Straße 5, 6020 Innsbruck, Austria; Department of Pharmacotherapy, University of Utah, 30 South 2000, Salt Lake City, UT 84112, USA; Program in Personalized Health, University of Utah, 15 North 2030 East, Room 2110, Salt Lake City, UT 84112, USA.
authors.forename: Diana
authors.initials: D
authors.lastname: Brixner
authors.affiliation: Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, UMIT-University for Health Sciences, Medical Informatics and Technology, Eduard Wallnoefer Center 1, 6060 Hall in Tirol, Austria; Area Health Technology Assessment, ONCOTYROL-Center for Personalized Cancer Medicine, Karl-Kapferer-Straße 5, 6020 Innsbruck, Austria; Center for Health Decision Science, Department of Health Policy and Management, Harvard T.H. Chan School of Public Health, 718 Huntington Avenue, Boston, MA 02215, USA; Institute for Technology Assessment, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, 101 Merrimac Street, Boston, MA 02114, USA.
authors.forename: Uwe
authors.initials: U
authors.lastname: Siebert