Effectiveness and Cost-Effectiveness of Sequential Treatment of Patients with Chronic Myeloid Leukemia in the United States: A Decision Analysis.
Ursula RochauMartina KluibenschaedlDavid StenehjemKuo Kuan-LingJerald RadichGary OderdaDiana BrixnerUwe SiebertPublished in: Leukemia research and treatment (2015)
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.
Keyphrases
- chronic myeloid leukemia
- locally advanced
- healthcare
- stem cell transplantation
- randomized controlled trial
- squamous cell carcinoma
- rectal cancer
- end stage renal disease
- chronic kidney disease
- low dose
- high dose
- electronic health record
- combination therapy
- big data
- cross sectional
- ejection fraction
- peritoneal dialysis
- artificial intelligence
- tyrosine kinase
- epidermal growth factor receptor