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Published on: November 26, 2018
Long-Term Clinical Outcomes, Adherence, Persistence, and Adverse Event Management in Patients With Chronic
Santoleri Fiorenzo1, Pennese Elsa1, Sanna Alessandro2
1General Hospital Pescara, Pescara, Italy.
Insights
Long-term ibrutinib treatment for chronic lymphocytic leukemia (CLL) demonstrates high adherence and favorable outcomes in real-world practice. Proactive management of adverse events is key to sustained treatment and maximizing patient benefit.
Area of Science:
- Oncology
- Pharmacology
- Hematology
Background:
- Long-term Bruton's tyrosine kinase inhibitor (BTKi) therapy significantly improves chronic lymphocytic leukemia (CLL) outcomes.
- Limited real-world data exists on BTKi treatment dynamics, including adherence, persistence, and adverse event management.
- This study evaluates long-term ibrutinib treatment patterns and clinical outcomes in routine CLL care.
Purpose of the Study:
- To assess long-term treatment adherence, persistence, and clinical outcomes of ibrutinib in CLL patients.
- To investigate the impact of adverse event management on treatment duration and efficacy.
- To identify factors associated with treatment adherence and outcomes in a real-world setting.
Main Methods:
- Multicenter, retrospective observational study of 276 adult CLL patients treated with ibrutinib.
- Data linkage between clinical records and pharmacy dispensing registries.
- Evaluation of progression-free survival (PFS), overall survival (OS), time to treatment discontinuation (TTD), adherence, and adverse event management using Kaplan-Meier and regression models.
Main Results:
- High adherence (0.75-0.90) was observed across treatment lines and follow-up.
- At 8 years, first-line ibrutinib showed PFS (86.4%), OS (64.5%), and TTD (61.4%) rates.
- Higher comorbidity burden (CIRS > 6) correlated with optimal adherence and reduced discontinuation risk.
Conclusions:
- Long-term ibrutinib therapy is linked to positive clinical outcomes, sustained adherence, and prolonged persistence in CLL.
- Effective adverse event management and personalized treatment strategies are crucial for maximizing long-term benefits.
- Real-world data supports ibrutinib's role in managing CLL effectively.
Background:
Long-term treatment with Bruton's tyrosine kinase inhibitors has substantially improved outcomes in chronic lymphocytic leukemia (CLL). However, real-world evidence simultaneously evaluating clinical outcomes, treatment adherence, persistence, and adverse event management remains limited. This study assessed long-term treatment dynamics and clinical outcomes in patients with CLL receiving ibrutinib in routine clinical practice.
Methods:
We conducted a multicenter, retrospective, observational study involving four Italian centers. Adult patients with CLL treated with ibrutinib between January 2016 and June 2024 were included. Clinical records were linked with hospital pharmacy dispensing registries to evaluate progression-free survival (PFS), overall survival (OS), time to treatment discontinuation (TTD), adherence, persistence, and adverse event management. Adherence was calculated using dispensing data adjusted according to the prescribed daily dose (PDD). Kaplan-Meier analyses were performed for survival outcomes, while multivariable logistic and Cox regression models were used to identify factors associated with adherence and clinical outcomes.
Results:
A total of 276 patients were included (47% first-line, 36% second-line, and 17% third-line or later therapy). Mean age was 70 ± 10 years and 63% were male. Adherence remained consistently high across treatment lines and throughout follow-up, with mean adherence values generally ranging between 0.75 and 0.90. At 8 years, first-line patients achieved PFS, OS, and TTD rates of 86.4%, 64.5%, and 61.4%, respectively. Corresponding estimates were 72.7%, 49.5%, and 23.1% in second-line patients and 95.8%, 49.4%, and 53.5% among patients receiving third-line or later therapy. Adverse drug reactions occurred in 53% of patients; 74% resolved following clinical management. Higher comorbidity burden (CIRS > 6) was independently associated with optimal adherence (OR 2.12, 95% CI 1.12-4.00; p = 0.016) and lower risk of treatment discontinuation (HR 0.36, 95% CI 0.16-0.80; p = 0.013).
Conclusions:
Long-term treatment with ibrutinib was associated with favorable clinical outcomes, sustained adherence, and prolonged treatment persistence in routine clinical practice. Proactive adverse event management and individualized treatment optimization may contribute substantially to maintaining long-term treatment exposure and maximizing clinical benefit.
