A POOLED REAL-WORLD CASE ANALYSIS OF BIOMARKER-GUIDED TREATMENT PATTERNS AND CLINICAL BURDEN IN BING-NEEL SYNDROME
Author(s)
Tanvi R. Mhetre, Pharm D1, Srushti M. Jaiswal, Pharm D2, Pranav R. Mhetre, Pharm D3.
1Student, Dr. D.Y. Patil Dnyan Prasad University's School of Pharmacy and Research, Pune, Pimpri, India, 2Dr. D.Y. Patil Dnyan Prasad University's School of Pharmacy and Research, Pune, Pimpri , Pune, India, 3Dr. D.Y. Patil Dnyan Prasad University's School of Pharmacy and Research, Pune, Pune, India.
1Student, Dr. D.Y. Patil Dnyan Prasad University's School of Pharmacy and Research, Pune, Pimpri, India, 2Dr. D.Y. Patil Dnyan Prasad University's School of Pharmacy and Research, Pune, Pimpri , Pune, India, 3Dr. D.Y. Patil Dnyan Prasad University's School of Pharmacy and Research, Pune, Pune, India.
OBJECTIVES: Bing-Neel Syndrome (BNS) is a rare neurological complication of Waldenström Macroglobulinemia (WM), for which data-based treatment recommendations are still not available. In this study, association of biomarker domains, choice of treatment and clinical burden was assessed in BNS cases reported.
METHODS: A pooled case-level analysis for 124 published BNS cases were performed. The biomarkers were categorized into molecular, immunoglobulin, cerebrospinal fluid, immunophenotypic, histopathological, hematological. Association between domains of the biomarker and therapy was evaluated by Chi square or Fisher's exact test. To scan for biomarker-treatment relationships, a correlation-analysis was conducted. Clinical burden was quantified as the total number of biomarker domains that were affected, and measured the clinical burden relative to treatment utilization.
RESULTS: Immunoglobulin abnormalities (74.19%) and CSF abnormalities (61.29%) were the most common biomarker domains, and chemotherapy (82.26%) and monoclonal antibodies (70.97%) the most commonly used therapies. Significant associations were found between BTK inhibitor and molecular abnormalities (p=0.001), CSF immunophenotyping and BTK inhibitors (p=0.031), chemotherapy (p=0.047), steroid therapy (p<0.001). Hematological involvement was significantly associated with steroid use (p=0.028). However, moderate positive correlation was found between molecular domain and BTK inhibitor (r=0.31, p=0.001). The best correlation was between CSF immunophenotyping levels and steroid therapy (r=0.33,p<0.001). The clinical burden was 2.77±1.33 affected domains while 49.2% of patients had a moderate clinical burden. The chemotherapy (p=0.008) and use of steroid (p=0.002) were significantly associated with high clinical burden, suggesting that more complex cases might thus be being treated more intensively.
CONCLUSIONS: Molecular and CSF based biomarkers may be at the core of therapeutic decision making in BNS. More advanced treatments such as chemotherapy and steroids were more likely to be used for patients with increased clinical burden. The results contribute to real-world evidence for treatment selection based on biomarkers and may have implications for the standardization of diagnosis and treatment in this rare disease.
METHODS: A pooled case-level analysis for 124 published BNS cases were performed. The biomarkers were categorized into molecular, immunoglobulin, cerebrospinal fluid, immunophenotypic, histopathological, hematological. Association between domains of the biomarker and therapy was evaluated by Chi square or Fisher's exact test. To scan for biomarker-treatment relationships, a correlation-analysis was conducted. Clinical burden was quantified as the total number of biomarker domains that were affected, and measured the clinical burden relative to treatment utilization.
RESULTS: Immunoglobulin abnormalities (74.19%) and CSF abnormalities (61.29%) were the most common biomarker domains, and chemotherapy (82.26%) and monoclonal antibodies (70.97%) the most commonly used therapies. Significant associations were found between BTK inhibitor and molecular abnormalities (p=0.001), CSF immunophenotyping and BTK inhibitors (p=0.031), chemotherapy (p=0.047), steroid therapy (p<0.001). Hematological involvement was significantly associated with steroid use (p=0.028). However, moderate positive correlation was found between molecular domain and BTK inhibitor (r=0.31, p=0.001). The best correlation was between CSF immunophenotyping levels and steroid therapy (r=0.33,p<0.001). The clinical burden was 2.77±1.33 affected domains while 49.2% of patients had a moderate clinical burden. The chemotherapy (p=0.008) and use of steroid (p=0.002) were significantly associated with high clinical burden, suggesting that more complex cases might thus be being treated more intensively.
CONCLUSIONS: Molecular and CSF based biomarkers may be at the core of therapeutic decision making in BNS. More advanced treatments such as chemotherapy and steroids were more likely to be used for patients with increased clinical burden. The results contribute to real-world evidence for treatment selection based on biomarkers and may have implications for the standardization of diagnosis and treatment in this rare disease.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HSD116
Topic
Health Service Delivery & Process of Care, Study Approaches
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology, Personalized & Precision Medicine