Patient report outcomes: Financial toxicity in cholangiocarcinoma.

Abstract

394 Background: While surgery is the only curative option for cholangiocarcinoma (CCA), it is amenable only in a minority of patients and has high recurrence rates. Numerous experimental and targeted therapies are under investigation; however, objective health-related quality of life (HRQoL) data for patients receiving these therapies are currently not available. Methods: Pts engaged in the Cholangiocarcinoma Foundation completed two validated HRQoL surveys: Functional Assessment of Cancer Therapy (FACT)-Hepatobiliary and COmprehensive Score for financial Toxicity (COST). Subscales (Physical Well-Being, PWB; Social Well-Being, SWB; Emotional Well-Being, EWB; Functional Well-Being, FWB; Hepatobiliary Cancer Subscale, HCS; Financial Toxicity Scale, FTS) and composite scores (FACT-Hep, composite for PWB, SWB, EWB, FWB, HCS) were calculated according to the FACIT-Manual, with higher values indicating improved HRQoL. Results: 208pts completed the surveys. 75% (n = 156) had intrahepatic CCA, 57% (n = 119) underwent resection, of which 48% (n = 56) had disease recurrence. 22% (n = 45) enrolled in a clinical trial and 80% (n = 167) underwent molecular profiling, of which 29% (n = 48) received targeted therapy. While pts enrolled in a clinical trial reported similar HRQoL compared to those not enrolled in a clinical trial, they also reported lower FTS (effect size 0.33, p = 0.05), suggesting higher financial burden. Pts who received targeted therapy reported equivalent HRQoL and lower FTS compared to those who did not (effect size 0.43, p = 0.01). Conclusions: Our data suggest that enrollment in a clinical trial does not affect a patient’s physical, emotional, social, or functional well-being. However, despite these treatments being paid for through trial enrollment, patients still report higher financial burden. This is also seen among patients who received targeted therapy. For patients with CCA, clinical trials and personalized therapy are mainly offered late in the advanced disease setting, at which point a significant financial toll has already been endured. Further, they are often only available at large academic centers, creating a physical barrier to access. These findings underscore the need to increase clinical trial availability and eliminate physical and financial barriers that threaten access and utilization of personalized and progressive therapies.[Table: see text]