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A Real-World Observational Cohort of Patients with Hepatocellular Carcinoma: Design and Rationale for TARGET-HCC.

ABSTRACT: This study describes the design of the TARGET-hepatocellular carcinoma (HCC) cohort and descriptive characteristics of the patient population at diagnosis among those who were enrolled in the cohort across academic and community clinical centers. TARGET-HCC is a 5-year, longitudinal, observational cohort of patients with HCC receiving care in usual clinical practice. Redacted clinical information, obtained from medical records, captures the natural history and management of the disease, including the safety and efficacy of treatment interventions used in usual clinical practice. Patients can complete patient-reported outcome measures and provide biological specimens for future translational studies. The TARGET-HCC study includes adults with histologic, cytologic, or radiologic diagnosis of HCC from academic and community centers in both the United States and Europe. A total of 1,841 participants were enrolled between January 9, 2017, and July 23, 2019, at 67 sites in the United States and Europe. To date, the most common liver disease etiology in the cohort continues to be hepatitis C, although nearly half had a nonviral etiology, including alcohol-related liver disease or nonalcoholic steatohepatitis. Most included patients were diagnosed at an early stage (Barcelona Clinic Liver Cancer Stage [BCLC] 0/A), but only approximately one third underwent curative treatment. Systemic therapy has been used in 7.3% of enrolled patients, including 45.7% of those with BCLC stage C tumors. Conclusion: Overall, the TARGET-HCC cohort allows for the assessment of patient characteristics and investigation of new treatment paradigms and sequencing with existing agents as well as novel regimens for HCC.

SUBMITTER: Cabrera R

PROVIDER: S-EPMC7917285 | biostudies-literature |

REPOSITORIES: biostudies-literature

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Project description:Abstract Hepatocellular carcinoma (HCC) is the third most common cause of cancer-related mortality worldwide and accounts for 90% of all primary liver cancers. Chronic inflammation is the hallmark across most prevalent etiologies among which HBV is the leading cause worldwide (33%), followed by alcohol (30%), HCV (21%), other factors like non-alcoholic steatohepatitis linked to insulin resistance/metabolic syndrome, and obesity associated inflammation (16%). Deregulation of the tightly controlled immunological network leads to liver disease, including chronic infection, autoimmunity, and tumor development. While inflammation drives oncogenesis in the liver, HCC also recruits ICOS+ FOXP3+ Tregs and MDSCs and upregulates immune checkpoints to induce a state of immunosuppression in the tumor microenvironment. As such, research is focused on targeting and modulating the immune system to treat HCC. The Checkmate 040 and Keynote 224 studies established the role of immunotherapy in the treatment of patients with HCC. In Phase I and II trials, nivolumab and pembrolizumab demonstrated durable response rates of 15–20% and were subsequently approved as second-line agents after sorafenib. Due to the success of the IMbrave 150 and HIMALAYA trials, which examined the combination of atezolizumab/bevacizumab and tremelimumab/durvalumab, respectively, the FDA approved these regimens as first-time treatment options for patients with advanced HCC. The encouraging results of immunotherapy in the management of HCC has led researchers to evaluate if combination with locoregional therapies may result in a synergistic effect. Real-world studies represent an invaluable tool to assess and verify the applicability of clinical trials in the bedside setting with a more varied patient population. We herein review current real-life use of ICIs in the management of HCC and highlight some of the ongoing clinical trials that are expected to change current recommended first-line treatment in the near future.

Project description:Background: Naoshuantong capsules (NC) are commonly used for the treatment of ischemic stroke. Experimental research and small-sample clinical trials have demonstrated that NC is effective in improving neurological recovery. Yet, there is a substantial lack of high-quality evidence on the precision treatment population of NC and long-term safety when making real-world clinical decisions. The acquisition of prospective longitudinal data in the real-world setting is essential to fully characterize the effectiveness and safety profile of NC for patients with ischemic stroke. Methods: The Vital real-world Experience regarding Naoshuantong capsules for Unselected ischemic Stroke (VENUS) registry is a prospective, multicenter, observational study aiming to register 5,000 patients. Eligible adult patients diagnosed with ischemic stroke and newly treated with NC within 30 days of symptom onset will be consecutively registered from 84 participating sites across the Chinese mainland. Baseline data will be recorded at the patient registry, and all patients will be regularly followed up at 2, 4, 8, and 12 weeks after the initial patient registry, and 180 days after ischemic stroke onset. The primary outcome is the distribution of scores on the modified Rankin Scale at 12 weeks after initial patient registry. Adverse events will be recorded during the study for NC safety assessment. Results: A total of 4,185 patients with ischemic stroke were enrolled, among which 37.06% patients were female. The average age of all patients was 65.22 years. The proportion of patients whose course of ischemic stroke was less than 14 days accounted for 93.45%. Conclusion: The VENUS registry is designed to comprehensively document medical data regarding NC treatment for ischemic stroke in real-world settings. The findings of this study will provide valuable insights into the clinical management of patients with ischemic stroke and the subsequent outcomes of the use of NC when included in the best clinical practice. Study registration: This study was registered with the Chinese Clinical Trial Registry (URL: http://www.chictr.org.cn/index.aspx, Unique identifier: ChiCTR1900025053).

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A Real-World Observational Cohort of Patients with Hepatocellular Carcinoma: Design and Rationale for TARGET-HCC.

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