Table of Contents
Leukemia Research and Treatment
Volume 2015, Article ID 982395, 12 pages
Research Article

Effectiveness and Cost-Effectiveness of Sequential Treatment of Patients with Chronic Myeloid Leukemia in the United States: A Decision Analysis

1Institute 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
2Area Health Technology Assessment, ONCOTYROL-Center for Personalized Cancer Medicine, Karl-Kapferer-Straße 5, 6020 Innsbruck, Austria
3Department of Pharmacotherapy, University of Utah, 30 South 2000, Salt Lake City, UT 84112, USA
4Huntsman Cancer Institute, University of Utah Hospitals & Clinics, 2000 Circle of Hope, Salt Lake City, UT 84112, USA
5Clinical Research Division, Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue N, Seattle, WA 98104, USA
6Program in Personalized Health, University of Utah, 15 North 2030 East, Room 2110, Salt Lake City, UT 84112, USA
7Center 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
8Institute for Technology Assessment, Department of Radiology, Massachusetts General Hospital, Harvard Medical School, 101 Merrimac Street, Boston, MA 02114, USA

Received 25 August 2015; Revised 5 November 2015; Accepted 11 November 2015

Academic Editor: Mario Tiribelli

Copyright © 2015 Ursula Rochau et al. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.


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 nilotinibdasatinibchemotherapy/SCT. In the economic evaluation, imatinibchemotherapy/SCT resulted in an incremental cost-utility ratio (ICUR) of $171,700/QALY compared to chemotherapy without TKI. Imatinibnilotinibchemotherapy/SCT yielded an ICUR of $253,500/QALY compared to imatinibchemotherapy/SCT. Nilotinibdasatinibchemotherapy/SCT yielded an ICUR of $445,100/QALY compared to imatinibnilotinibchemotherapy/SCT. All remaining strategies were excluded due to dominance of the clinically superior strategies. Based on our analysis and current treatment guidelines, imatinibnilotinibchemotherapy/SCT and nilotinibdasatinibchemotherapy/SCT can be considered cost-effective for patients with CML, depending on willingness-to-pay.