Chronic Myeloid Leukemia: Part I—Real-World Treatment Patterns, Healthcare Resource Utilization, and Associated Costs in Later Lines of Therapy in the United States

Abstract

**Background:** Despite advances in tyrosine kinase inhibitor (TKI) therapy for chronic myeloid leukemia in chronic phase (CML-CP), a sizeable proportion of patients with CML-CP remains refractory or intolerant to these agents. **Objectives:** Treatment patterns, healthcare resource utilization (HRU), and costs were evaluated among patients with CML who received third or later lines of therapy (3L+), a clinical population that has not been previously well-studied, with unmet treatment needs as TKI therapy has repeatedly failed. **Methods:** Adult patients with CML who received 3L+ were identified in the IBM® MarketScan® Databases (January 1, 2001–June 30, 2019) and the SEER-Medicare–linked database (January 1, 2006–December 31, 2016). Treatment patterns were observed from CML diagnosis. HRU and direct healthcare costs (payer’s perspective, 2019 USD) were measured in a 3L+ setting. **Results:** Among 296 commercially insured patients with 3L+ (median age, 58.5 years; female, 49.7%), the median duration of first-line (1L), second-line (2L), and 3L therapy was 8.5, 4.2, and 8.3 months, respectively. The annual incidence rate during 3L+ was 3.4 for inpatient days, 30.8 for days with outpatient services, and 1.2 for emergency department visits. Mean per-patient-per-month (PPPM) total healthcare costs (pharmacy + medical costs) were \$18 784 in 3L+, \$15 206 in 3L, and \$19 546 in 4L, with inpatient costs driving most of the difference between 3L and 4L (mean [3L] = \$2528 PPPM, mean [4L] = \$6847 PPPM). Among 53 Medicare-insured patients with 3L+ (median age, 72.0 years; female, 39.6%), the median duration of 1L, 2L, and 3L therapy was 9.7, 5.0, and 7.0 months, respectively. During 3L+, the annual incidence rate was 10.3 for inpatient days, 61.9 for days with outpatient services, and 1.5 for emergency department visits. Mean PPPM total healthcare costs were \$14 311 in 3L+, \$15 100 in 3L, and \$16 062 in 4L. **Discussion:** Patients with CML receiving 3L+ rapidly cycled through multiple lines. Costs increased from 3L to 4L; in commercially insured patients, inpatient costs were responsible for most of the cost increase between 3L and 4L, underlying these patients’ continued need for care. **Conclusions:** These findings support the need for better treatment options in patients with CML undergoing later lines of therapy.