Nilotinib versus dasatinib as second-line therapy in patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase who are resistant or intolerant to imatinib: a cost-effectiveness analysis based on real-world data

References

• NIH National Cancer Institute. Surveillance, epidemiology, and end results program (SEER) stat fact sheets: Chronic Myeloid Leukemia (CML). 2015, http://seer.cancer.gov/statfacts/html/cmyl.html. Accessed October 27, 2015
   Google Scholar
• Nowell PC, Hungerford DA. A minute chromosome in human chronic granulocytic leukemia. Science 1960;132:1488-501
   PubMed Web of Science ®Google Scholar
• Morel F, Ka C, Le Bris MJ, et al. Deletion of the 5′ abl region in Philadelphia chromosome-positive chronic myeloid leukemia. Leukemia 2003;17:473-4
   PubMed Web of Science ®Google Scholar
• Melo JV. The diversity of BCR-ABL fusion proteins and their relationship to leukemia phenotype. Blood 1996;88:2375-84
   PubMed Web of Science ®Google Scholar
• Baccarani M, Cortes J, Pane F, et al. Chronic myeloid leukemia: an update of concepts and management recommendations of European LeukemiaNet. J Clin Oncol 2009;27:6041-51
   PubMed Web of Science ®Google Scholar
• Mace ML, Dahl J, Jabbour EJ. Which tyrosine-kinase inhibitor to use first in chronic phase chronic myelogenous leukemia? Expert Opin Pharmacother 2015;16:999-1007
   PubMed Web of Science ®Google Scholar
• O’Brien SG, Guilhot F, Larson RA, et al. Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med 2003;348:994-1004
   PubMed Web of Science ®Google Scholar
• Radich JP, Deininger M, Abboud CN, et al. NCCN clinical practice guidelines in oncology: Chronic myelogenous leukemia, Version 1; 2016. https://www.nccn.org/professionals/physician_gls/pdf/aml.pdf. Accessed March 15, 2016
   Google Scholar
• Jabbour E, Kantarjian H, Cortes J. Use of second- and third-generation tyrosine kinase inhibitors in the treatment of chronic myeloid leukemia: an evolving treatment paradigm. Clin Lymphoma Myeloma Leuk 2015;15:323-34
   PubMed Web of Science ®Google Scholar
• Rogers G, Hoyle M, Thompson Coon J, et al. Dasatinib and nilotinib for imatinib-resistant or -intolerant chronic myeloid leukaemia: a systematic review and economic evaluation. Health Technol Assess 2012;16:1-410
   Web of Science ®Google Scholar
• Loveman E, Cooper K, Bryant J, et al. Dasatinib, high-dose imatinib and nilotinib for the treatment of imatinib-resistant chronic myeloid leukaemia: a systematic review and economic evaluation. Health Technol Assess 2012;16:iii-xiii, 1–137
   PubMed Web of Science ®Google Scholar
• Whalen J, Ozer-Stillman I, Ambavane A, et al. Cost-effectiveness analysis (CEA) of sequential treatment with tyrosine kinase inhibitors (TKIs) for chronic myelogenous leukemia (CML) (Poster). 56th ASH Annual Meeting and Exposition; December 6–9, 2014; San Francisco, CA. https://ash.confex.com/ash/2014/webprogram/Paper74905.html. Accessed July 14, 2016
   Google Scholar
• Bristol-Myers Squibb Pharmaceuticals Ltd. Dasatinib (Sprycel) for the treatment of adults with chronic, accelerated or blast phase CML with resistance or intolerance to prior therapy including imatinib. Multiple Technology Appraisal Submission to the National Institute for Health & Clinical Excellence; 2009
   Google Scholar
• Kantarjian H, Giles F, Wunderle L, et al. Nilotinib in imatinib-resistant CML and Philadelphia chromosome-positive ALL. N Engl J Med 2006;354:2542-51
   PubMed Web of Science ®Google Scholar
• Shah NP, Guilhot F, Cortes JE, et al. Long-term outcome with dasatinib after imatinib failure in chronic-phase chronic myeloid leukemia: follow-up of a phase 3 study. Blood 2014;123:2317-24
   PubMed Web of Science ®Google Scholar
• Griffin JD, Guerin A, Chen L, et al. Comparing nilotinib with dasatinib as second-line therapies in patients with chronic myelogenous leukemia resistant or intolerant to imatinib—a retrospective chart review analysis. Curr Med Res Opin 2013;29:623-31
   PubMed Web of Science ®Google Scholar
• Minacori R, Bonastre J, Lueza B, et al. How to model survival in cost-effectiveness analysis? Differences between Markov and partitioned survival analysis models. ISPOR 18th Annual European Congress; November 11, 2015; Milan, Italy. http://www.ispor.org/research_pdfs/51/pdffiles/PRM123.pdf. Accessed March 1, 2016
   Google Scholar
• Pan F. Evaluation of treatment pathways in oncology: Modeling approaches. ISPOR; May 18–22, 2013; New Orleans, LA. http://www.ispor.org/meetings/neworleans0513/releasedpresentations/W27-Pan.pdf. Accessed March 14, 2014
   Google Scholar
• Briggs A, Sculpher M. An introduction to Markov modelling for economic evaluation. Pharmacoeconomics 1998;13:397-409
   PubMed Web of Science ®Google Scholar
• Federal Drug Administration (FDA). Highlights of prescribing information. SPRYCEL (dasatinib) tablet for oral use. FDA; August 2015, http://www.accessdata.fda.gov/drugsatfda_docs/label/2009/021986s004lbl.pdf
   Google Scholar
• Federal Drug Administration (FDA). Highlights of prescribing information. TASIGNA (nilotinib) capsules for oral use. FDA; January 2015, http://www.accessdata.fda.gov/drugsatfda_docs/label/2007/022068lbl.pdf
   Google Scholar
• Akaike H, Nakagawa T. Statistical analysis and control of dynamic systems. Boston: Kluwer Academic Publishers; 1988
   Google Scholar
• Konishi S, Kitagawa G. Information criteria and statistical modeling. New York: Springer; 2008
   Google Scholar
• Saefken B, Kneib T, van Waveren C-S, et al. A unifying approach to the estimation of the conditional Akaike information in generalized linear mixed models. Electronic Journal of Statistics. 2014;8:201-25
   Google Scholar
• Schwarz G. Estimating the dimension of a model. Ann Statist 1978;6:461-4
   Web of Science ®Google Scholar
• Fouskakis D, Ntzoufras I, Draper D. Bayesian variable selection using cost-adjusted BIC, with application to cost-effective measurement of quality of health care. Ann App Stat 2009;3:663-90
   Web of Science ®Google Scholar
• Arias E. United States life tables, 2010. Natl Vital Stat Rep 2014;63:1-63
   PubMedGoogle Scholar
• Szabo SM, Levy AR, Davis C, et al. A multinational study of health state preference values associated with chronic myelogenous leukemia. Value Health 2010;13:103-11
   PubMed Web of Science ®Google Scholar
• United States Bureau of Labor Statistics. Consumer Price Index (CPI); United States Bureau of Labor Statistics; 2014, http://www.bls.gov/cpi/
   Google Scholar
• Truven Health Analytics MicroMedex Solutions. Red Book Online Search. 2015. http://www.redbook.com/redbook. Accessed June 6, 2015
   Google Scholar
• Li N, Yang C, Fan L, et al., editors. Abstract 2090: Nilotinib vs dasatinib as second-line therapy in patients with Philadelphia-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) who are resistant or intolerant to imatinib: a cost-effectiveness analysis (CEA) based on real-world data. American Society of Hematology (ASH); December 5–8, 2015; Orlando, FL
   Google Scholar
• Federal Drug Administration (FDA). Highlights of prescribing information. BOSULIF (bosutinib) tablet for oral use. November 2014, http://www.accessdata.fda.gov/drugsatfda_docs/label/2012/203341lbl.pdf
   Google Scholar
• Federal Drug Administration (FDA). Highlights of prescribing information. ICLUSIG (ponatinib) tablets for oral use. September 2014, http://www.accessdata.fda.gov/drugsatfda_docs/label/2012/203469lbl.pdf
   Google Scholar
• Federal Drug Administration (FDA). Highlights of prescribing information. SYNRIBO (omacetaxine mepesuccinate) for injection, for subcutaneous use. April 2014, http://www.accessdata.fda.gov/drugsatfda_docs/label/2012/203585lbl.pdf
   Google Scholar
• Maziarz RT, Guerin A, Gauthier G, et al., editors. Abstract 872: Five-year direct cost of pediatric patients with acute lymphoblastic leukemia (ALL) undergoing allogeneic stem cell transplantation (HSCT): an analysis from US payers’ perspective. American Society of Hematology (ASH); December 5–8, 2015; Orlando, FL.
   Google Scholar
• CMS Physician Fee Schedule [Internet]. Centers for Medicare & Medicaid Services (CMS). 2015. https://www.cms.gov/apps/physician-fee-schedule/overview.aspx. Accessed June 5, 2015
   Google Scholar
• Goldberg SL, Chen L, Guerin A, et al. Association between molecular monitoring and long-term outcomes in chronic myelogenous leukemia patients treated with first line imatinib. Curr Med Res Opin 2013;29:1075-82
   PubMed Web of Science ®Google Scholar
• Reed SD, Anstrom KJ, Ludmer JA, et al. Cost-effectiveness of imatinib versus interferon-alpha plus low-dose cytarabine for patients with newly diagnosed chronic-phase chronic myeloid leukemia. Cancer 2004;101:2574-83
   PubMed Web of Science ®Google Scholar
• Yabroff KR, Lamont EB, Mariotto A, et al. Cost of care for elderly cancer patients in the United States. J Natl Cancer Inst 2008;100:630-41
   PubMed Web of Science ®Google Scholar
• Kantarjian HM, Giles FJ, Bhalla KN, et al. Nilotinib is effective in patients with chronic myeloid leukemia in chronic phase after imatinib resistance or intolerance: 24-month follow-up results. Blood 2011;117:1141-5
   PubMed Web of Science ®Google Scholar
• Liou SY, Stephens JM, Carpiuc KT, et al. Economic burden of haematological adverse effects in cancer patients: a systematic review. Clin Drug Investig 2007;27:381-96
   PubMed Web of Science ®Google Scholar
• Healthcare Cost and Utilization Project’s (HCUP) HCUPnet [Internet]. Agency for Healthcare Research and Quality, Rockville, MD; 2012. http://hcupnet.ahrq.gov/. Accessed July 10, 2015
   Google Scholar
• Hoyle M, Rogers G, Moxham T, et al. Cost-effectiveness of dasatinib and nilotinib for imatinib-resistant or -intolerant chronic phase chronic myeloid leukemia. Value Health 2011;14:1057-67
   PubMed Web of Science ®Google Scholar
• Kulpeng W, Sompitak S, Jootar S, et al. Cost-utility analysis of dasatinib and nilotinib in patients with chronic myeloid leukemia refractory to first-line treatment with imatinib in Thailand. Clin Ther 2014;36:534-43
   PubMed Web of Science ®Google Scholar
• Rochau U, Kluibenschaedl M, Stenehjem D, et al. Effectiveness and cost-effectiveness of sequential treatment of patients with chronic myeloid leukemia in the United States: a decision analysis. Leuk Res Treat 2015;2015:1-12
   Google Scholar
• Padula WV, Larson RA, Dusetzina SB, et al. Cost-effectiveness of tyrosine kinase inhibitor treatment strategies for chronic myeloid leukemia in chronic phase after generic entry of imatinib in the United States. J Natl Cancer Inst 2016;108, https://www.ncbi.nlm.nih.gov/pubmed/26944912
   Google Scholar
• National Health Expenditures 2014 Highlights. Centers for Medicare & Medicaid Services (CMS); 2015. https://www.cms.gov/research-statistics-data-and-systems/statistics-trends-and-reports/nationalhealthexpenddata/downloads/highlights.pdf. Accessed February 5, 2016
   Google Scholar
• Tangka FK, Trogdon JG, Richardson LC, et al. Cancer treatment cost in the United States: has the burden shifted over time? Cancer 2010;116:3477-84
   PubMed Web of Science ®Google Scholar
• Schnipper LE, Davidson NE, Wollins DS, et al. American Society of Clinical Oncology statement: a conceptual framework to assess the value of cancer treatment options. J Clin Oncol 2015;33:2563-77
   PubMed Web of Science ®Google Scholar
• Bryan S, Sofaer S, Siegelberg T, et al. Has the time come for cost-effectiveness analysis in US health care? Health Econ Policy Law 2009;4:425-43
   PubMed Web of Science ®Google Scholar