Citations

Abstract Introduction: Bruton tyrosine kinase inhibitors (BTKis) have transformed the treatment landscape for CLL, offering significant improvement in patient outcomes. Despite the widespread adoption of BTKis and their proven clinical benefits, data on the treatment preferences and priorities of patients with CLL in Asia, particularly in Japan, are limited. Understanding patient preference is critical for optimizing clinical decision-making and tailoring treatment strategies for Japanese patients. A comprehensive quantitative analysis was conducted to evaluate patient preferences for covalent BTKi treatment attributes and trade-offs among these attributes. Methods: An online survey applying discrete choice experiment (DCE) methodology was administered between April 29 and June 30, 2025 to Japanese adults (≥18 years of year) diagnosed with CLL. The design of DCE attributes and levels was informed by literature review and clinical consultations. Treatment attributes included efficacy (i.e., progression-free survival [PFS]), safety (i.e., impacts of diarrhea, headache, atrial fibrillation, and hypertension on quality of life [QoL]), and convenience (i.e., formulation type including tablet or capsule, and dosing frequency including once daily or twice daily). A conditional logistic regression was applied to estimate preference weights, which were used to calculate the relative importance as well as willingness-to-tradeoff between BTKi treatment attributes. Results: The study included 50 patients (mean age: 62 years; 30% female; 38% employed full-time; 60% reported no comorbid conditions). More than half (60%) were diagnosed ≥5 years ago. Around one quarter (24%) were treatment naive, and 76% received ≥1 treatment (40% first-line, 26% second-line and 10% third-line or later). Most patients (82%) reported living with or near their caregiver. In terms of travel time to obtain CLL medication, 46% reported travel time of one hour, 40% traveled less than 30 minutes, and 14% traveled more than two hours. The majority of patients (66%) were using High-Cost Medical Expense Benefit, while an additional 10% were either currently applying or planning to apply. In general, patients preferred treatments with less impact of AEs on QoL, higher efficacy, and less dosing frequency (P<.05). The top three treatment attributes with the highest relative importance were the impact of atrial fibrillation (24.7%) and diarrhea (21.0%) on QoL, and PFS (15.8%), followed by impact of headache (15.7%) and hypertension (14.1%) on QoL, dosing frequency (7.8%), and formulation type (capsule or tablet) (0.9%). Patients were willing to trade off PFS for improved quality of life: accepting reductions of 3.1 years for less impact from atrial fibrillation, 2.7 years for diarrhea, 2.0 years for headache, and 1.8 years for hypertension (none or mild vs. significant impact). Additionally, they were willing to accept a reduction of 1.0 years of PFS to receive a treatment given once daily rather than twice daily.Conclusions: This is the first quantitative assessment of treatment preferences among Japanese patients with CLL using a DCE approach. Findings from this study provide valuable Japan-specific insights, highlighting that although efficacy is important, patients prioritize safety factors, such as the impact of atrial fibrillation and diarrhea on QoL. These findings support the integration of patient perspectives into treatment planning to enhance shared decision-making, personalize care, and promote better adherence to long-term therapies in CLL management.

Objective: This study aimed to evaluate the characteristics, clinical outcomes, and resource use of patients with unresectable hepatocellular carcinoma (uHCC) treated with first-line (1L) atezolizumab plus bevacizumab (A+B) at five United States (US) institutions: the Mayo Clinic, Houston Methodist, Moffitt Cancer Center, Mays Cancer Center, and University of Arizona. Methods: Treating oncologists extracted data from medical charts of patients with uHCC who were treated with A+B after 1 January 2019. Real-world progression-free survival (rwPFS) and overall survival (OS) were assessed using the Kaplan–Meier method for the overall cohort and for a “trial-like” subgroup with characteristics similar to those in the IMbrave150 trial (Eastern Cooperative Oncology Group Performance Status [ECOG PS] 0–1, Child–Pugh [CP] class A, albumin–bilirubin grade 1–2). Results: Of the 300 patients in the overall cohort (median age of 68 years; 12% ECOG PS ≥ 2; 73% CP A; 26% CP B; median follow-up of 8.7 months), the median rwPFS was 6.8 (95% confidence interval [CI]: 5.8, 8.4) months, and the median OS was 14.4 (95% CI: 12.3, 18.2) months. In the trial-like subgroup (n = 194), the median rwPFS was 8.8 (95% CI: 7.6, 12.1) months and the median OS was 19.5 (95% CI: 14.6, 24.7) months. A significantly lower proportion of patients with CP A compared with CP B (39.7% vs. 73.4%) experienced hospitalization within one year of A+B initiation, whereas hospitalizations due to treatment-related adverse events were similar. Conclusions: This study provides insights into the real-world effectiveness of 1L A+B in a diverse US patient cohort, with results from trial-like patients supporting the reproducible efficacy of A+B in clinical practice.

537 Background: A+B is the preferred first-line (1L) standard of care for uHCC, with emerging RW evidence supporting its use in a broad patient (pt) cohort. We evaluated pt characteristics, clinical and treatment outcomes in pts treated at five U.S. institutions: Mayo Clinic, Houston Methodist, Moffitt Cancer Center, Mays Cancer Center, and University of Arizona. Methods: This is a retrospective study of uHCC pts who initiated A+B after January 1, 2019. De-identified pt data were extracted by treating oncologists. Overall survival (OS) and real-world progression-free survival (rwPFS) were assessed using Kaplan-Meier methods for all pts and for a “trial-like” subgroup with characteristics similar to those in the IMbrave150 trial (ECOG performance status [PS] 0-1, Child-Pugh [CP] class A, albumin-bilirubin [ALBI] grade 1-2). HCC-related hospitalizations were evaluated within 1 year of A+B initiation. Results: A total of 300 eligible pts were treated with 1L A+B (median age 68 years, 79% male, 79% White, 82% BCLC C, 44% viral etiology,12% ECOG PS >1). Liver function was CP A in 73% (36% A5, 31% A6, 6% not reported), CP B in 26% pts (15% B7, 7% B8, 5% B9), and CP C in 2 (<1%) pts. Compared with CP A, more CP B pts had characteristics of compromised liver function (CP A vs B: cirrhosis: 61% vs 91%; ascites: 7% vs 46%; encephalopathy: 4% vs 17%; esophageal varices: 14% vs 32%), bile duct invasion (2% vs 11%), and ECOG PS>1 (8% vs 22%), all Ps<0.001. Over a median follow-up of 8.7 mos, 244 (81%) pts discontinued A+B, primarily due to disease progression. Toxicity-related treatment discontinuation and hospitalizations due to treatment-related adverse events (TRAE) were similar between the two groups (Table). Median OS (mOS) was 14.4 mos (95% CI: 12.3, 18.2) and median rwPFS was 6.8 mos (95% CI: 5.8, 8.4). In the trial-like subgroup (n=194), mOS was 19.5 mos (95%CI: 14.6, 24.7) and median rwPFS was 8.8 mos (95% CI: 7.6, 12.1). In the CP B subgroup, mOS was 5.6 mos (95% CI: 4.6, 11.3) and median rwPFS was 3.1 mos (95% CI: 2.4, 5.8). Conclusions: This study demonstrates the RW outcomes of 1L A+B in a diverse pt cohort. Results from the “trial-like” pts further confirm the reproducible efficacy of A+B in clinical practice. Although pts with CP-B had higher hospitalization rates due to disease progression or symptoms, consistent with their underlying liver complications, they had similar rates of toxicity-related treatment discontinuation and TRAE-related hospitalizations compared to CP-A. Further characterization of safety for A+B in CP B pts within a prospective, controlled trial is warranted. N (%) CP A (n=219) CP B (n=79) Treatment discontinuation* 167 (76) 75 (95) Atezolizumab-related toxicity 20 (12) 13 (17) Bevacizumab-related toxicity 26 (16) 16 (21) Hospitalization* 87 (40) 58 (73) Symptom-related 42 (48) 35 (60) Progression 8 (9) 11 (19) TRAE 7 (8) 6 (10) *P-value comparing CP A vs CP B <0.001.