OBJECTIVES: Management of chronic myeloid leukemia (CML) is associated with extensive economic burden. With improvements in economic level and clinical research, findings and conclusions with regards to this crucial issue are evolving. The objective of this systematic literature review (SLR) was to conduct a comprehensive assessment of economic evaluations in CML.

METHODS: Literature search of English language publications from 2010-2020 was conducted in Embase^®, MEDLINE^®,NHS EED and relevant conference proceedings to identify economic evaluations of CML that fulfilled pre-defined inclusion criteria. Relevant health technology assessment reports (HTAs) were also included. Data on model specifications, patient characteristics, cost and main outcomes were extracted from included studies.

RESULTS: The SLR retrieved 1492 records; 44 fulfilled eligibility criteria. Majority of studies evaluated first-line treatment with tyrosine kinase inhibitors (TKIs). Treatments evaluated included imatinib, nilotinib, ponatinib and bosutinib. Most studies used a Markov model and time horizon varied from ≤5 years to life-time. Most studies and HTAs reported cycle length of 1 month. As first-line therapy, imatinib was cost-effective versus nilotinib and dasatinib in six studies; nilotinib was cost-effective versus imatinib and dasatinib in five studies. As second-line therapy, nilotinib and dasatinib were cost-effective versus high-dose imatinib. Bosutinib and dasatinib were cost-effective under patient access schemes. As third-line treatment, ponatinib yielded discounted quality adjusted life years (QALYs) and improved outcomes versus second-generation TKIs.

CONCLUSIONS: Imatinib is cost-effective in newly-diagnosed patients with CML as first-line therapy, while nilotinib and dasatinib are cost-effective as second-line agent in patients who are resistant/intolerant to imatinib. Limited evidence exists regarding the economic evaluations in the third-line setting. More studies assessing the long-term efficacy and cost-effectiveness of novel treatments in CML are necessary.