Diagnostic journey and life impact of cholangiocarcinoma: results from surveys of patient and caregiver experiences.

Aim: To understand cholangiocarcinoma symptoms, diagnosis and treatment experience from the patient and caregiver perspective, including cholangiocarcinoma's impact on daily life, quality of life (QoL) and mental health. Methods: Patients and caregivers participated in two online surveys (in partnership with the Cholangiocarcinoma Foundation). Results: The patient survey data (n = 707) show a substantial impact of cholangiocarcinoma on QoL and mental health, with 34% of patients reporting symptoms consistent with moderately severe/severe depression. The caregiver survey data (n = 60) show that although caregivers experience satisfaction in their role of caring for a loved one, managing the demands of caregiving exacts a physical, mental and emotional toll. Conclusion: These surveys highlight the need for better palliative and supportive care interventions.

What is this article about? It shows results from two surveys, one for people with cholangiocarcinoma (CCA, pronounced ko·lan·jee·o·car·sin·no·muh), and one for caregivers (people who take care of family or friends with CCA without payment). CCA is a rare and aggressive cancer. The caregivers we surveyed were not necessarily taking care of the people with CCA who we surveyed. We did the surveys to find out how CCA changed the lives of people in these two groups. What were the results? We surveyed 707 people with CCA. Patients reported in the survey that having CCA impacted their daily lives in lots of ways. Most needed help with daily chores like housekeeping and shopping. Both tiredness and anxiety were reported by about two in three people with CCA. More than one in three had said they had symptoms indicating potential depression, which means patients should have their mental health evaluated. CCA also reduced their sexual desire and intimacy with their partner. We surveyed 60 caregivers who reported both good and bad experiences taking care of a person with CCA. The good experiences included knowing that their loved one was well cared for and learning to deal with difficult situations. Many caregivers also felt closer to their loved one with CCA. The bad experiences included exhaustion and emotional and mental stress. Caregivers felt challenged by trying to understand CCA and the treatment options available. What do the results of the study mean? Patients with CCA and their caregivers need more help and support.

The Cholangiocarcinoma in the Young (CITY) Study: Tumor Biology, Treatment Patterns, and Survival Outcomes in Adolescent Young Adults With Cholangiocarcinoma.

PURPOSE: Increased awareness of the distinct tumor biology for adolescents and young adults (AYAs) with cancer has led to improvement in outcomes for this population. However, in cholangiocarcinoma (CCA), a paucity of data exist on the AYA population. To our knowledge, we present the largest study to date on AYA disease biology, treatment patterns, and survival outcomes in CCA. METHODS: A multi-institutional cohort of patients with CCA diagnosed with intrahepatic cholangiocarcinoma (ICC) or extrahepatic cholangiocarcinoma (ECC) was used for analysis. Retrospective chart review was conducted on patients who were 50 years old and younger (young; n = 124) and older than 50 years (older; n = 723). RESULTS: Among 1,039 patients screened, 847 patients met eligibility (72% ICC, 28% ECC). Young patients had a larger median tumor size at resection compared with older patients (4.2 v 3.6 cm; P = .048), more commonly had N1 disease (65% v 43%; P = .040), and were more likely to receive adjuvant therapy (odds ratio, 4.0; 95% CI, 1.64 to 9.74). Tumors of young patients were more likely to harbor an FGFR2 fusion, BRAF mutation, or ATM mutation (P < .05 for each). Young patients were more likely to receive palliative systemic therapy (96% v 69%; P < .001), targeted therapy (23% v 8%; P < .001), and treatment on a clinical trial (31% v 19%; P = .004). Among patients who presented with advanced disease, young patients had a higher median overall survival compared with their older counterparts (17.7 v 13.5 months; 95% CI, 12.6 to 22.6 v 11.4 to 14.8; P = .049). CONCLUSION: Young patients with CCA had more advanced disease at resection, more commonly received both adjuvant and palliative therapies, and demonstrated improved survival compared with older patients. Given the low clinical trial enrollment and poor outcomes among some AYA cancer populations, data to the contrary in CCA are highly encouraging.

Molecular profiling and treatment pattern differences between intrahepatic and extrahepatic cholangiocarcinoma.

BACKGROUND: Treatment patterns for intrahepatic cholangiocarcinoma (ICC) and extrahepatic cholangiocarcinoma (ECC) differ, but limited studies exist comparing them. This study examines differences in molecular profiling rates and treatment patterns in these populations, focusing on use of adjuvant, liver-directed, targeted, and investigational therapies. METHODS: This multicenter collaboration included patients with ICC or ECC treated at 1 of 8 participating institutions. Retrospective data were collected on risk factors, pathology, treatments, and survival. Comparative statistical tests were 2-sided. RESULTS: Among 1039 patients screened, 847 patients met eligibility (ICC = 611, ECC = 236). Patients with ECC were more likely than those with ICC to present with early stage disease (53.8% vs 28.0%), undergo surgical resection (55.1% vs 29.8%), and receive adjuvant chemoradiation (36.5% vs 4.2%) (all P < .00001). However, they were less likely to undergo molecular profiling (50.3% vs 64.3%) or receive liver-directed therapy (17.9% vs 35.7%), targeted therapy (4.7% vs 18.9%), and clinical trial therapy (10.6% vs 24.8%) (all P < .001). In patients with recurrent ECC after surgery, the molecular profiling rate was 64.5%. Patients with advanced ECC had a shorter median overall survival than those with advanced ICC (11.8 vs 15.1 months; P < .001). CONCLUSIONS: Patients with advanced ECC have low rates of molecular profiling, possibly in part because of insufficient tissue. They also have low rates of targeted therapy use and clinical trial enrollment. While these rates are higher in advanced ICC, the prognosis for both subtypes of cholangiocarcinoma remains poor, and a pressing need exists for new effective targeted therapies and broader access to clinical trials.

Patient reported outcomes: Financial toxicity is a barrier to clinical trials and personalized therapy in cholangiocarcinoma.

PURPOSE: Numerous experimental and targeted therapies are under investigation for patients with cholangiocarcinoma (CCA). Objective health-related quality of life (HRQoL) data for patients receiving these therapies are limited. METHODS: Patients engaged in the Cholangiocarcinoma Foundation completed two validated HRQoL surveys: Functional Assessment of Cancer Therapy (FACT)-Hepatobiliary and COmprehensive Score for financial Toxicity (COST). RESULTS: Two hundred eight patients were included. Seventy-five percent had intrahepatic CCA and 57% underwent resection, of which 48% had disease recurrence. Twenty-two percent enrolled in a clinical trial and 80% underwent molecular profiling, of which 29% received targeted therapy. While patients enrolled in a clinical trial or received targeted therapy reported similar HRQoL compared to those who did not, they reported higher financial toxicity (p = 0.05 and p = 0.01, respectively). CONCLUSION: Enrollment in a clinical trial or receipt of targeted therapy do not affect a patient's physical, emotional, social, or functional well-being. However, patients report higher financial burden. These therapies are mainly offered in the advanced setting after significant financial strain has been endured and are often only available at large academic centers, creating a physical barrier to access. These findings underscore the need to increase availability and eliminate physical and financial barriers that threaten access and utilization of personalized and progressive therapies.

American Society of Clinical Oncology Road to Recovery Report: Learning From the COVID-19 Experience to Improve Clinical Research and Cancer Care.

This report presents the American Society of Clinical Oncology's (ASCO's) evaluation of the adaptations in care delivery, research operations, and regulatory oversight made in response to the coronavirus pandemic and presents recommendations for moving forward as the pandemic recedes. ASCO organized its recommendations for clinical research around five goals to ensure lessons learned from the COVID-19 experience are used to craft a more equitable, accessible, and efficient clinical research system that protects patient safety, ensures scientific integrity, and maintains data quality. The specific goals are: (1) ensure that clinical research is accessible, affordable, and equitable; (2) design more pragmatic and efficient clinical trials; (3) minimize administrative and regulatory burdens on research sites; (4) recruit, retain, and support a well-trained clinical research workforce; and (5) promote appropriate oversight and review of clinical trial conduct and results. Similarly, ASCO also organized its recommendations regarding cancer care delivery around five goals: (1) promote and protect equitable access to high-quality cancer care; (2) support safe delivery of high-quality cancer care; (3) advance policies to ensure oncology providers have sufficient resources to provide high-quality patient care; (4) recognize and address threats to clinician, provider, and patient well-being; and (5) improve patient access to high-quality cancer care via telemedicine. ASCO will work at all levels to advance the recommendations made in this report.