Real-World Evidence and Outcomes of Bruton Tyrosine Kinase Inhibitors in Chronic Lymphocytic Leukemia: A Systematic Review
Author(s)
Vassiki Sanogo, BA, MSc, PhD1, Karam Diaby, BSc, MSc, PhD2.
1HEOR | Data Scientist, Integrity Analytics, Lakeland, FL, USA, 2Otsuka, Princeton, NJ, USA.
1HEOR | Data Scientist, Integrity Analytics, Lakeland, FL, USA, 2Otsuka, Princeton, NJ, USA.
OBJECTIVES: Chronic lymphocytic leukemia (CLL) is a prevalent hematological malignancy, with treatment strategies evolving, particularly with the introduction of Bruton tyrosine kinase inhibitors (BTKis) like Ibrutinib, Acalabrutinib, and Zanubrutinib. This study aimed to review real-world evidence (RWE) applications in CLL research.
METHODS: A systematic literature review (SLR) was conducted following PRISMA guidelines. The search databases include, PubMed, Ovid, and ASCO, as well as the National Cancer Institute (NCI) and American Cancer Society (ACS) websites. We used keywords, Boolean operators, truncation, and wildcard operators, spanning January 1, 2015, to March 28, 2025, to identify RWE studies, targeting best practices, methods, CLL prevalence, treatment outcomes, and market shares of BTKi therapies.
RESULTS: Of 2040 studies, this SLR analyzed 15 articles. The SLR highlighted common analytical methods in CLL studies, including descriptive statistics, propensity score matching, difference-in-difference, Kaplan-Meier survival analysis, and Cox regression models. Also, the SLR noted an estimated CLL prevalence of 1 in 200 and 23690 new cases and 4460 deaths in 2025, with a higher occurrence in older males. The SLR revealed that advancements in CLL treatment focus on BTKis, with Ibrutinib, Acalabrutinib, and Zanubrutinib as key first-line options. Second-generation BTKis are gaining traction, while emerging therapies like Pirtobrutinib and BTK degraders expand the landscape. CLL management also includes chemoimmunotherapy, immunotherapy, bone marrow transplantation, and supportive care. BTKis block BTK signaling, with irreversible options like Ibrutinib, Acalabrutinib, and Zanubrutinib showing efficacy, while Pirtobrutinib offers a reversible alternative. Zanubrutinib surpasses Ibrutinib in progression-free survival, though Ibrutinib maintains a strong market position. Real-world data remains essential for refining treatment strategies, and based on databases such as Flatiron, Medicare claims, and Optum.
CONCLUSIONS: This SLR highlights key analytical methods and evolving CLL treatment landscape, emphasizing BTKis, real-world data, and policy considerations. Optimized therapy selection, affordability, and research collaboration are essential for improving patient outcomes and healthcare strategies.
METHODS: A systematic literature review (SLR) was conducted following PRISMA guidelines. The search databases include, PubMed, Ovid, and ASCO, as well as the National Cancer Institute (NCI) and American Cancer Society (ACS) websites. We used keywords, Boolean operators, truncation, and wildcard operators, spanning January 1, 2015, to March 28, 2025, to identify RWE studies, targeting best practices, methods, CLL prevalence, treatment outcomes, and market shares of BTKi therapies.
RESULTS: Of 2040 studies, this SLR analyzed 15 articles. The SLR highlighted common analytical methods in CLL studies, including descriptive statistics, propensity score matching, difference-in-difference, Kaplan-Meier survival analysis, and Cox regression models. Also, the SLR noted an estimated CLL prevalence of 1 in 200 and 23690 new cases and 4460 deaths in 2025, with a higher occurrence in older males. The SLR revealed that advancements in CLL treatment focus on BTKis, with Ibrutinib, Acalabrutinib, and Zanubrutinib as key first-line options. Second-generation BTKis are gaining traction, while emerging therapies like Pirtobrutinib and BTK degraders expand the landscape. CLL management also includes chemoimmunotherapy, immunotherapy, bone marrow transplantation, and supportive care. BTKis block BTK signaling, with irreversible options like Ibrutinib, Acalabrutinib, and Zanubrutinib showing efficacy, while Pirtobrutinib offers a reversible alternative. Zanubrutinib surpasses Ibrutinib in progression-free survival, though Ibrutinib maintains a strong market position. Real-world data remains essential for refining treatment strategies, and based on databases such as Flatiron, Medicare claims, and Optum.
CONCLUSIONS: This SLR highlights key analytical methods and evolving CLL treatment landscape, emphasizing BTKis, real-world data, and policy considerations. Optimized therapy selection, affordability, and research collaboration are essential for improving patient outcomes and healthcare strategies.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
SA81
Topic
Study Approaches
Topic Subcategory
Literature Review & Synthesis
Disease
Oncology