POST-2020 REAL-WORLD EVIDENCE ON PHARMACOTHERAPIES FOR CHRONIC LYMPHOCYTIC LEUKEMIA: A SCOPING REVIEW
Author(s)
Xueye Yan, Ph.D.;
University of Illinois Chicago, Doctoral student, Chicago, IL, USA
University of Illinois Chicago, Doctoral student, Chicago, IL, USA
OBJECTIVES: The introduction of Bruton’s tyrosine kinase inhibitors (BTKis) and B-cell lymphoma 2 inhibitors (BCL2is) has transformed the treatment of chronic lymphocytic leukemia (CLL), demonstrating significant efficacy and safety profiles in both clinical and real-world evidence (RWE) studies. BCL2is, Venetoclax-based therapy, might attract more patients with fixed treatment duration compared to lifelong treatment of BTKis. This scoping review aims to update previous reviews to synthesize the effectiveness and toxicity of CLL pharmacotherapies, especially BTKis and BCL2is by including RWE studies after 2020.
METHODS: We searched RWE studies on CLL pharmacotherapies published since 2020 on PubMed. Studies with survival outcomes and adverse events were included. Data of real-world data sources, patient demographics and clinical information, and effectiveness and safety outcomes were extracted. Descriptive statistics were used to summarize the key characteristics of CLL treatments and methodologies of included literature.
RESULTS: A total of 131 RWE studies were included. The study population consisted of individuals from North America (e.g., the USA, Canada), Europe (e.g., Germany, Italy, Poland), and Asia (e.g., Japan, China). Among the included literature, 92 (70.6%) studies focused on BTKis (mainly ibrutinib), and 46 (35.3%) studies focused on BCL2is. Additionally, 92 (70.6%) studies addressed at least one effectiveness outcome, including time to next treatment, overall survival, and progression-free survival. We have 85 (64.7%) studies that reported the safety profile of CLL pharmacotherapies. The most frequent adverse events were neutropenia, infection, and cardiac disorders. Methodologically, 69 (52.9%) studies used patient electronic medical records, 27 (20.6%) used claims data, and 15 (11.8%) used registry data.
CONCLUSIONS: RWE studies demonstrated the effectiveness and intolerance of CLL therapies of BTKis and BCL2is. Nevertheless, a relatively small number of studies investigated BCL2is compared to BTKis. More RWE studies are needed to better understand the effects of lifelong vs. fixed-time duration treatments.
METHODS: We searched RWE studies on CLL pharmacotherapies published since 2020 on PubMed. Studies with survival outcomes and adverse events were included. Data of real-world data sources, patient demographics and clinical information, and effectiveness and safety outcomes were extracted. Descriptive statistics were used to summarize the key characteristics of CLL treatments and methodologies of included literature.
RESULTS: A total of 131 RWE studies were included. The study population consisted of individuals from North America (e.g., the USA, Canada), Europe (e.g., Germany, Italy, Poland), and Asia (e.g., Japan, China). Among the included literature, 92 (70.6%) studies focused on BTKis (mainly ibrutinib), and 46 (35.3%) studies focused on BCL2is. Additionally, 92 (70.6%) studies addressed at least one effectiveness outcome, including time to next treatment, overall survival, and progression-free survival. We have 85 (64.7%) studies that reported the safety profile of CLL pharmacotherapies. The most frequent adverse events were neutropenia, infection, and cardiac disorders. Methodologically, 69 (52.9%) studies used patient electronic medical records, 27 (20.6%) used claims data, and 15 (11.8%) used registry data.
CONCLUSIONS: RWE studies demonstrated the effectiveness and intolerance of CLL therapies of BTKis and BCL2is. Nevertheless, a relatively small number of studies investigated BCL2is compared to BTKis. More RWE studies are needed to better understand the effects of lifelong vs. fixed-time duration treatments.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EPH163
Topic
Epidemiology & Public Health
Topic Subcategory
Safety & Pharmacoepidemiology
Disease
SDC: Oncology