COMPARATIVE SAFETY AND HEALTHCARE RESOURCE UTILIZATION ASSOCIATED WITH FIRST-LINE TREATMENTS FOR CHRONIC MYELOID LEUKEMIA

Treatment with tyrosine kinase inhibitors (TKIs) has dramatically improved the prognosis for patients with CML. No significant differences in overall survival have been observed between the three TKIs approved for first-line treatment of CML between 2010-2016, but these treatments vary in safety and price in ways that are not well-studied and may influence the initial treatment choice. The objectives of this dissertation were to investigate predictors of first-line treatment, comparative safety, and comparative healthcare expenditures among CML patients treated with imatinib, dasatinib, or nilotinib. MarketScan Commercial and Medicare Supplemental data was used to identify patients with CML treated with imatinib, dasatinib, or nilotinib between 2011 and 2016. Odds ratios (OR) for the effect of hypothesized predictors on treatment choice were estimated using multinomial logistic regression. The 1-year relative risk (RR) and cumulative incidence of hospitalizations and emergency department visits was compared across inverse probability of treatment weighted groups. Inflation-adjusted all-cause healthcare expenditures and patient out-of-pocket costs were compared among patients with ≥12 months’ follow-up using generalized linear models and quantile regression. In a study population of 1,417 imatinib-, 1,067 dasatinib-, and 647 nilotinib-treated patients, younger patients and patients with higher Klabunde-Charlson comorbidity indices (CCI) were significantly more likely to receive treatment with dasatinib and nilotinib, compared to imatinib (OR [95% confidence interval (CI)], age