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Aim: Many pivotal trials in advanced hepatocellular carcinoma (HCC) require participants to have Child-Pugh A disease. However, many patients in real-world practice are Child-Pugh B or C. This study examined treatment patterns and clinical outcomes in patients with advanced HCC treated with first-line systemic therapy. Materials & methods: In this retrospective study, patients with HCC treated with first-line systemic therapy (2010–2017) were identified from US Oncology Network records. Outcomes included overall survival and progression-free survival, by Child-Pugh Class and prior liver-directed therapy. Results: Of 352 patients, 78.7% were Child-Pugh A or B, 96.6% received first-line sorafenib, and 33.8% received first-line-prior liver-directed therapy. Survival outcomes were similar for Child-Pugh A or B, and longer after first-line prior liver-directed therapy. Conclusion: First-line systemic therapy is beneficial in patients with Child-Pugh A or B, and after first-line prior liver-directed therapy. These findings may help position systemic therapy in the community setting.

Aim: To assess real-world management of patients diagnosed with hepatocellular carcinoma (HCC) within an integrated delivery network. Materials & methods: A retrospective cohort analysis of adults newly diagnosed with HCC from January 2014 to March 2019. Overall survival and treatment journey were assessed over the entire available follow-up period per patient. Results: Of the 462 patients, 85% had ≥1 treatment. The 24-month overall survival rate (95% CI) from first treatment was 77% (72–82%). Majority of Child-Pugh class A (71%) and B (60%) patients received locoregional therapy first. Half (53.6%) of the patients with liver transplantation first were Child-Pugh class C patients. Sorafenib was the predominant systemic therapy. Conclusion: This integrated delivery network data analysis offers a comprehensive insight into the real-world management of HCC.

Abstract Background Hepatocellular carcinoma (HCC) is an aggressive form of liver cancer with increasing incidence and mortality worldwide. For metastatic disease, systemic treatment is recommended. In addition to tumor characteristics, adverse events (AEs) may influence regimen choice. Aim To analyze healthcare burden among patients with advanced HCC, by treatment type and AEs observed. Methods Included were adult commercial and Medicare Advantage enrollees with ≥2 non‐diagnostic claims coded for HCC (the first setting the index date); ≥1 claim for systemic treatment of advanced/metastatic HCC; and continuous enrollment for a 6‐month pre‐index baseline period to ≥1 month post‐index (follow‐up). Patients were excluded by lack of systemic treatment; incomplete demographic information; pregnancy, liver transplant, other cancers during baseline or clinical trial participation. We describe patient characteristics, common AEs, overall survival, and healthcare burden in 2017 USD up to 12 months after initiation of tyrosine kinase inhibitor (TKI) monotherapy; immune checkpoint inhibitor (ICI) monotherapy; or FOLFOX combination therapy. Results The analytic sample consisted of 322 patients (median age 65.8 years, 76% male) who had 12 months' (unless death occurred prior) available follow‐up, with median follow‐up of 9 months. Among these, 241 (75%) had TKI monotherapy, 23 (7%) had ICI monotherapy, and 58 had FOLFOX (18%) first‐line treatment. Overall, patients had a high burden of AEs (mean 3.2), with the most prevalent being pain (75%), infection (39%), ascites (34%), and bleeding (29%). After adjusting for covariates, infection ($50 374), fever ($47 443), and diarrhea ($29 912) imposed the highest incremental annual costs versus patients without the AE. Up to 90% of costs were attributable to inpatient admissions, with 56% to 60% involving intensive care. Median 1‐year survival was 32%. Conclusions This real‐world study demonstrated AE burden in alignment with previous clinical studies. Regardless of regimen used, AEs are associated with substantial healthcare costs due to inpatient care.

Aim: To identify and evaluate the similarity of all trials assessing recommended treatments for advanced hepatocellular carcinoma. Materials & methods: Single arm and randomized trials from any phase and published any time up to February 2021 were systematically searched. Results: From 5677 records reviewed, 50 trials were included in the review, and 24 for assessed for similarity. In the first-line (1L) setting, several trials assessing sorafenib were noted for enrolling patients with more severe disease and/or performance status than other 1L trials; trials within the second-line (2L) setting were generally similar. Median survival was

Aim: To examine the locoregional therapy (LRT) patterns and the healthcare economic burden of patients with hepatocellular carcinoma (HCC) in the USA. Patients & methods: Patients with newly diagnosed HCC were identified from the MarketScan® databases (1 July 2015–31 May 2018). The LRTs received and all-cause and HCC-related healthcare costs were measured. Results: Among 2101 patients with HCC, most received embolization therapy as their first LRT treatment (57.8%, n = 1215); 17.1% (n = 360) received ablative therapy and 8.7% (n = 182) radiation therapy; 16.4% (n = 344) received multiple LRTs. After patients received their first LRT treatment, total all-cause healthcare costs averaged $20,316 per patient per month; 70.7% ($14,359) were HCC related. Conclusion: Among newly diagnosed HCC patients treated with LRT in the USA, the economic burden is high.

Aim: To describe the epidemiologic, humanistic and economic burdens of hepatocellular carcinoma (HCC) in the USA. Materials & methods: Studies describing the epidemiology and economic burden from national cohorts, any economic models, or any humanistic burden studies published 2008–2018 were systematically searched. Results: HCC incidence was 9.5 per 100,000 person-years in most recent data, but was ∼100-times higher among patients with hepatitis/cirrhosis. Approximately a third of patients were diagnosed with advanced disease. Patients with HCC experienced poor quality of life. Direct costs were substantial and varied based on underlying demographics, disease stage and treatment received. Between 25–77% of patients did not receive surgical, locoregional or systemic treatment. Conclusion: Better treatments are needed to extend survival and improve quality of life for patients with HCC.

Abstract The purpose of this study was to estimate the risk of developing skeletal‐related events (SREs) based on site of metastasis at diagnosis and identify other predictors of developing SREs among metastatic prostate cancer patients. We conducted a retrospective cohort study using linked SEER (Surveillance, Epidemiology, and End Results) and Medicare data and identified men over the age of 65 with incident metastatic prostate cancer diagnosed during 2005–2009. SREs included radiation (RAD), pathological fractures (PF), bone surgery (BS), and spinal cord compression (SCC). The association between site of metastasis at diagnosis and SRE was examined using a Cox proportional hazards model that accounts for death as a competing risk. Among 4404 men (median age: 79 years) with incident metastatic prostate cancer, 44% experienced SREs at a median of 9.6 months post diagnosis. Compared to bone metastasis only, our model showed that patients were significantly less likely to develop SREs if they had LN‐only metastasis at diagnosis (Sub‐Hazard Ratio [SHR] 0.56; 95% Confidence Interval [CI]: 0.43–0.72) or unknown site of metastasis (SHR: 0.79; CI: 0.64–0.97). Other predictors of reduced SRE risk were age 80+ years (SHR: 0.83; CI: 0.75–0.91), non‐Hispanic Black (SHR: 0.77; CI: 0.65–0.90), or being diagnosed in year 2009 (SHR: 0.85; CI: 0.72–0.99). Patients were significantly more likely to develop SREs if they received androgen deprivation therapy (SHR: 1.73; CI: 1.48–2.02) or had Gleason score 8–10 disease (SHR: 0.79; CI: 0.64–0.97). Compared to patients who present with bone metastasis only at diagnosis, patients presenting with other metastatic sites have similar risk of developing SREs, with the exception of those presenting with lymph node only metastasis who have a significantly reduced risk of SREs.