Real-World Analysis of the Therapeutic Management and Disease Burden in Chronic Myeloid Leukemia Patients with Later Lines in Italy

SIMPLE SUMMARY: Real world data represent a useful tool to obtain understanding into the management of cancer disease in routine daily practice. To date, little is known on management and burden of later lines for chronic myeloid leukemia (CML) treatment in Italy. Therefore, we conducted a real-world study to evaluate the characteristics, treatment pattern and drug utilization of patients with CML in 2 or ≥3 tyrosine kinase inhibitor (TKI) lines of therapy to estimate the impact of disease burden. Findings from our study underline an increasing complex management of CML patients while moving on later lines and suggest that the availability of more therapeutic options for CML patients might be an existing need. ABSTRACT: Real world data are becoming a crucial tool to understand how cancer is treated in routine daily practice. This real-world analysis aims to describe the characteristics of patients with CML in 2nd or ≥3rd tyrosine kinase inhibitors (TKI) lines of therapy, to evaluate their treatment sequence and utilization in settings of Italian clinical practice in Italy. A retrospective analysis was performed using an administrative databases covering around 15.3 million cases. All adult patients prescribed with TKI as 2nd or ≥3rd lines (L) of therapy for CML during January 2015–December 2018 were included. A total of 491 patients in 2nd and 144 in ≥3rd L was included. In both cohorts, hypertension was the most reported comorbidity, followed by metabolic and blood count alterations. In each calendar inclusion year, an increment of 97.6% was observed in the number of patients treated in ≥3rd L. In the 2nd L cohort, 18.7% had a switch to 3rd L, while 26.4% of ≥3rd L patients switched to a subsequent line. Around 40% in both lines discontinued their treatment after a median time of 5.5 (2nd L) and 4.3 (≥3rd L) years. The results provided insights into CML management clinical practice, indicating a heavy disease burden for patients in later lines that showed an increasing complex management, and suggest that a need for novel treatment strategies might exists.