First-Line Treatment of Chronic Lymphocytic Leukemia in Italy: Real-World Evidence on Utilization, Outcomes, and...

Chronic lymphocytic leukemia (CLL) is the most common adult leukemia in Western countries, mainly affecting older people. Targeted agents have reshaped first-line (1L) strategies, making real-world evidence important to complement clinical trials.

To estimate the incidence of Italian patients initiating first-line CLL therapy (2019-2022) and describe demographics/clinical profile, treatment patterns, adherence, outcomes (overall survival [OS], time to next treatment [TTNT]), and healthcare costs from the perspective of the Italian National Health System (NHS).

A retrospective observational study using administrative healthcare databases (~9 million residents) was conducted on CLL patients starting 1L therapy (index-date) for CLL. Baseline characteristics were assessed in the 12 months pre-index; follow-up was ≥12 months. Drug use, adherence (medication possession ratio), dose adjustments, OS, TTNT, and direct costs were analyzed with descriptive and multivariable methods.

A total of 1479 patients initiated 1L therapy: 63.9% chemotherapy (CHT), 23.2% ibrutinib, 3.2% acalabrutinib, and 9.7% other regimens. CHT remained common, especially among older and more comorbid patients. Ibrutinib showed lower mortality versus CHT (HR 0.663; p=0.002) and longer TTNT (median not reached). Dose adjustments were frequent; extended refill intervals did not appear to reduce drug survival. Mean annual cost per patient was €38,573, mainly driven by drug acquisition; ibrutinib users had lower hospitalization and outpatient costs than other 1L groups.

In Italian practice, ibrutinib was the main targeted 1L option and was associated with improved survival and delayed progression versus CHT. Despite higher drug costs, reduced hospital-based resource use suggests favourable overall clinical and economic impact.