Table 1.
Workshop highlights*
| Understanding the incidence of HCC |
| • More detailed/accurate incidence data are needed. |
| ○ Accurate incidence data are lacking in some parts of the world (sub-Saharan Africa, parts of Asia, the Middle East). |
| ○ The biology of HCC may be different for different etiologies. Look at the effectiveness of surveillance by etiology. |
| ○ More information is needed on the role/impact of HBV genotype on incidence. |
| ○ HCC in younger individuals is being observed (persons under the age currently recommended for screening by AASLD). This may be associated with specific genotypes. |
| Detection of HCC: bringing patients into care |
| • For many other cancers, early diagnosis does not necessarily require screening; however, for HCC screening it is essential. The evidence of the value of HCC screening is strongest for viral etiologies but is much less robust for other etiologies (alcoholism, NAFLD). |
| • Most patients have unresectable HCC at the time of diagnosis (had not been in screening programs, were not aware of their risk status). |
| • There is poor compliance with current HCC screening guidelines among both patients and providers. Creative ways to improve compliance are needed. |
| ○ Incorporate prompts/reminders for providers into EHR systems; send reminders to patients every 6 months. |
| ○ Task shifting to enhance compliance. Train midlevel practitioners (eg, nurse practitioners, physician assistants) in HCC screening and surveillance. (Could the Patient-Centered Outcomes Research Institute [PCORI] study the impact of task shifting on compliance?) |
| • Even if current screening and surveillance guidelines were followed faithfully, it is estimated that 20% to 30% of cases will be missed. Current approaches are 70% to 80% sensitive, and 80% to 90% specific in detecting HCC. |
| ○ Need to identify HCC in low-incidence populations (those who fall outside of the current surveillance guidelines). |
| ○ How to address the high potential for false positives in screening lower-risk populations? |
| • Need better use of biomarkers and algorithms in HCC screening and risk stratification. |
| ○ Is there a need for different biomarkers/algorithms for different subpopulations (eg, those with low AFP; young age; female; genotype-associated, HBV-positive without recognized cirrhosis)? |
| • Collect biospecimens as part of surveillance (blood, serum, plasma, urine, and tissue) for use in future studies. |
| Medical management of HCC |
| • Reduction in HCC mortality will come from preventing viral hepatitis, finding and treating cases of chronic viral hepatitis, finding tumors early, and treating those early tumors. |
| • Need to emphasize the prevention of chronic viral hepatitis. |
| ○ Many clinics that treat underserved populations, many of which are at high risk for chronic viral hepatitis, do not follow the guidelines for HBV screening and treatment. |
| ○ Propose a PCORI study using the Hepatitis B Foundation HBV screening and management algorithm (49) in some of these clinics. |
| ○ Promote the timely administration of the birth dose of HBV vaccine; encourage funders of global vaccine initiatives to provide the birth dose in resource-constrained countries that are disproportionately impacted. |
| • The heterogeneous nature of HCC makes it unlikely that a single therapeutic agent will be universally effective. |
| • Develop the potential of biomarkers for management of HCC: |
| ○ Molecular-targeted therapies for subtypes of HCC. |
| ○ Need efficient, rapid, cost-effective systems to assess hypothetical molecular targets. (eg, new tissue explant methods). |
| ○ Predict prognosis/treatment outcome. |
| ○ Enrich preventive and therapeutic clinical trial populations. |
| • Spontaneous immune responses are frequently observed in patients with HCC. Study the potential of immune checkpoint disrupters, alone or in combination with other therapies. |
| • Employ case review by a multidisciplinary tumor board to determine treatment strategy. |
| Areas for further study/action |
| • Treatment of chronic viral hepatitis: |
| ○ What is the impact of therapeutic intervention for HBV on HCC risk? |
| ○ Does antiviral treatment (polymerase inhibitors) of those with low viral load but strong family history of HCC reduce their risk? |
| ○ Should patients who have had ablation for HCC and have very low viral loads receive antivirals treatment? |
| ○ Are biomarkers of HCC affected by treatment of chronic HBV infection? |
| • Link studies to proper biosample repositories. |
| • How to detect (and treat) the fastest growing tumors? |
| • Link validation type studies to outcome (not just a comparative marker result but actual outcome). |
| • Need large cohort studies of comparative effectiveness in treated populations. |
| ○ Need biomarkers and other intermediate outcomes measures. |
| ○ Build outcomes studies into screening and surveillance initiatives. |
| • Need data on the progression and regression of fibrosis (eg, regression in HCV patients who have been treated and cured or HBV patients who have been virally suppressed). If fibrosis regresses below a certain level, is surveillance still needed? |
| • Investigate further the reported association between statin use and reduced risk of HCC. |
| • Ask the National Cancer Institute to review HCC under the Recalcitrant Cancer Research Act of 2012. |
| • Survey current insurance coverage/reimbursement of HCC surveillances tests recommended by the guidelines (ie, ultrasound). |
* AASLD = American Association for the Study of Liver Diseases; AFP = alpha fetoprotein; EHR = electronic health record; HBV = hepatitis B virus; HCC = hepatocellular carcinoma; HCV = hepatitis C virus; NAFLD = nonalcoholic fatty liver disease; PCORI = Patient-Centered Outcomes Research Institute.