Background: Oral tyrosine kinase inhibitors (TKIs) have been the standard of care for chronic myeloid leukemia (CML) since 2001. However, few studies have evaluated changes in the treatment landscape of CML over time. This study assessed the long-term treatment patterns of oral anticancer therapies among patients with CML. Methods: This retrospective cohort study included patients newly diagnosed with CML between January 1, 2000, and December 31, 2016, from 10 integrated healthcare systems. The proportion of patients treated with 5 FDA-approved oral TKI agents—bosutinib, dasatinib, imatinib, nilotinib, and ponatinib—in the 12 months after diagnosis were measured, overall and by year, between 2000 and 2017. We assessed the use of each oral agent through the fourth-line setting. Multivariable logistic regression estimated the odds of receiving any oral agent, adjusting for sociodemographic and clinical characteristics. Results: Among 853 patients with CML, 81% received an oral agent between 2000 and 2017. Use of non-oral therapies decreased from 100% in 2000 to 5% in 2005, coinciding with imatinib uptake from 65% in 2001 to 98% in 2005. Approximately 28% of patients switched to a second-line agent, 9% switched to a third-line agent, and 2% switched to a fourth-line agent. Adjusted analysis showed that age at diagnosis, year of diagnosis, and comorbidity burden were statistically significantly associated with odds of receiving an oral agent. Conclusions: A dramatic shift was seen in CML treatments away from traditional, nonoral chemotherapy toward use of novel oral TKIs between 2000 and 2017. As the costs of oral anticancer agents reach new highs, studies assessing the long-term health and financial outcomes among patients with CML are warranted.
Submitted December 18, 2018; accepted for publication March 29, 2019.
Author contributions: Study concept: Banegas, Rivera, Pawloski, Tabano, Epstein, Ritzwoller. Data analysis: Banegas, Rivera, Pawloski, O’Keeffe-Rosetti, Carroll, Ritzwoller. Data interpretation: All authors. Manuscript writing: All authors.
Disclosures: Dr. Banegas has disclosed that he has received grant/research support from AstraZeneca for work that is outside the scope of this study. Dr. Tabano has disclosed that he is employed by Bristol-Myers Squibb. The remaining authors have not received any financial consideration from any person or organization to support the preparation, analysis, results, or discussion of this article.
Funding: This work was supported by an NCI grant (U24CA171524). The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.