Currently, there are no standards of care for patients with relapsed or refractory hepatoblastoma (rrHBL), as rrHBL has not been formally studied to any significant extent through the international and national cooperative group mechanisms. The rrHBL Registry aims to fill this gap. The rrHBL Registry is one of the core initiatives of the Liver Tumors Research Consortium (LTRC), a collaboration among physicians and other care providers co-led by Drs. James Geller (CCHMC) and Allison O’Neill (DFCI), dedicated to improving outcomes for children and adolescents with liver tumors. The specific aims of the rrHBL registry prioritize advancement of research and knowledge about rrHBL. Such efforts will enable better characterization of:
All patients of any age with a suspected diagnosis (per treating oncologist/surgeon) or confirmed diagnosis of rrHBL, and all patients with Hepatocellular Malignant Neoplasm – Not Otherwise Specified (HCN-NOS) who are <6 years of age at the time of initial diagnosis, are eligible to enroll on the rrHBL Registry. Patients with HCN-NOS who are ≥6 years of age at the time of initial diagnosis can enroll on the LTRC HCC Registry (https://joincountmein.org/).
The enrollment process involves little time/effort on the part of the patient/family, taking about 15 minutes to run through the consent form. For more information on the process, visit https://rrhblregistry.org/. Patients and/or parents/legal guardians interested in enrolling on the rrHBL Registry can do so by:
The launch of the registry is a culmination of years of planning and start-up work and is a realization of a vision proposed by Dr. Geller, other pediatric oncologists, and parent stakeholders. From this initial vision came a write-up of the study protocol and consent form, followed by the creation of disease-specific data entry forms and an associated electronic database to house data. A guide was created to standardize the entry of information into the database. Repositories were created for the storage of imaging and tumor/other biological samples. The abovementioned website establishes a new platform to provide patients, families, and care providers with information on the Registry, how to be involved, and a pathway for patients/families to share updates and photos and stories. A comprehensive Manual of Operations was developed operationalizing the processes for consent, enrollment, requests, abstraction, central review by specialists (pathology, radiology, etc), specimen collection and storage, legal agreements, secondary site collaboration, and general upkeep. In September of 2022, the study was launched. Patient enrollments have begun.
rrHBL patients, all of which is ultimately accessible by collaborators and researchers from anywhere in the world through a scientific review process. The registry and repository are located at and being run out of Cincinnati Children’s Hospital Medical Center (CCHMC), the primary site. James Geller, MD is the principal investigator at CCHMC functioning as the rrHBL Registry Director. Anthony Asher is the dedicated Clinical Research Coordinator for the rrHBL Registry, overseeing the day-to-day operations. As a co-lead of the LTRC, DFCI functions as a secondary site for the rrBHL registry. Primary and secondary site roles will be reversed for the upcoming Hepatocellular Carcinoma (HCC) Registry, directed by Dr. Allison O’Neill.
The LTRC: As mentioned, the rrHBL registry is one of two prospective registries that fall under the umbrella of the Liver Tumors Research Consortium (LTRC), formed via a legal agreement between CCHMC and Dana-Farber Cancer Institute (DFCI). This LTRC is supported by both sites and will feature a jointly managed website (in-progress). The agreement allows for registry cross-coverage where needed as well, bolstering collaboration between sites. Outside of the operation centers, the LTRC is comprised of and/or accessible by clinical and scientific research investigators who may propose data queries and/or utilize samples for tumor model development and drug testing. Any data or biological proposal is reviewed by a Scientific Advisory Committee (SAC) composed of researchers with multidisciplinary expertise in basic, translational and clinical cancer research as well as parent/patient stakeholders. The SAC advises the steering committee, composed of the principal investigators from CCHMC and DFCI, who will provide oversight for all LTRC operations. Any stakeholder is welcome to propose and/or suggest future LTRC directions, and/or apply to serve as representatives in the SAC.
For those interested in learning more about the rrHBL Registry or enrollment, please visit https://rrhblregistry.org/ or contact the study team at rrHBLRegistry@cchmc.org or 844-722-8774 (Option 1).