Project Summary

Background: Hepatoblastoma is the most common liver cancer in children, but with only 150-200 cases/year in the United States. The international community has developed a clinical trial through cooperative groups to assess the effectiveness of front-line therapy. This Pediatric Hepatic International Liver Tumors Trial (PHITT) includes the Children’s Oncology Group (COG trial AHEP1531; Chairs: James Geller, MD; Greg Tiao, MD), the Society of International Oncology Pediatric European Liver Tumors Group (SIOPEL trial PHITT; Chair Bruce Morland, MBChB) and the Japanese Children’s Cancer Group (JCCG – JPLT4; Chair Eiso Hiyama, MD). The collaborative, spanning dozens of countries, will enroll approximately 1,000 children with hepatoblastoma over five years. The trial does not address outcomes research beyond survival and limited toxicity endpoints, does not address progression on therapy or relapse, and does not include stakeholder engagement

Proposed Solution to the Problem: On February 28 and 29, building on developing stakeholder/clinician relationships, a 1.5-day meeting will take place in Cincinnati, Ohio with specific objectives aiming to fill the above-mentioned gap.

  • Consolidate an engaged collaborative (hepatoblastoma community) integrating clinical researchers and patient/parent stakeholders to advance an agenda focusing on hepatoblastoma patient-reported outcomes beyond cure; advance knowledge and awareness on the management of controversial issues related to hepatoblastoma care including treatment of relapsed/refractory disease; and standardization of care for such affected patients for whom front-line therapy is not ideal or inadequate. 
  • Identify key priorities for focus of hepatoblastoma community related to patient-reported outcomes and clinical effectiveness standards and advancements. 
  • Develop organization for a sustainable effort going forward to bring action to priority items.
Activities will include a premeeting questionnaire, presentations by stakeholders and clinicians, and several facilitated subgroup breakout sessions/discussions. Facilitation will be used to integrate subgroup feedback and discussions to focus the group toward conceptualizing priorities in terms of patient-reported outcomes research that can translate into clinical effectiveness advancements. Longer term, the hepatoblastoma community will advance an organized action plan such that, within a year of the meeting, a structure to advance the relevant research and data management is in place, funding options are in process, and clear pathways for dissemination of results will be defined.
  • Establishment of a defined hepatoblastoma engagement community composed of researchers and stakeholders
  • A list of key priorities (research agenda)
  • Structured organization
  • Pathways for results dissemination
Engagement Plan: The patients are children with hepatoblastoma, and the stakeholders are patients and their parents. Every aspect of meeting planning, meeting conduct, and meeting follow-up involves stakeholders, including approximately 12 providing conference presentations.
Collaborators include CCHMC/COG liver tumor researchers, SIOPEL leaders, > 1 dozen stakeholders, experts in epidemiology, data management, and survivorship research.

Project Information

James Geller, MD, FAAP
Cincinnati Children’s Hospital Medical Center

Key Dates

February 2021


Project Status
State State The state where the project originates, or where the primary institution or organization is located. View Glossary
Last updated: April 8, 2024