The RELIVE consortium for relapsed or refractory pediatric hepatoblastoma and hepatocellular carcinoma: a scoping review of the problem and a proposed solution

Allison F O'Neill; Angela Trobaugh-Lotrario; James I Geller; Eiso Hiyama; Kenichiro Watanabe; Isabelle Aerts; Brice Fresneau; Fabienne Toutain; Michael J Sullivan; Howard M Katzenstein; Bruce Morland; Sophie Branchereau; József Zsiros; Rudolf Maibach; Marc Ansari

doi:10.1016/j.eclinm.2024.102446

The RELIVE consortium for relapsed or refractory pediatric hepatoblastoma and hepatocellular carcinoma: a scoping review of the problem and a proposed solution

EClinicalMedicine. 2024 Feb 15:69:102446. doi: 10.1016/j.eclinm.2024.102446. eCollection 2024 Mar.

Authors

Affiliations

¹ Department of Pediatric Oncology, Dana-Farber Cancer Institute and Harvard Medical School, Boston, MA, USA.
² Providence Sacred Heart Children's Hospital, Spokane, WA, USA.
³ Division of Oncology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH, USA.
⁴ Department of Pediatric Surgery, Hiroshima University Hospital, Hiroshima, Japan.
⁵ Shizuoka Children's Hospital, Shizuoka, Japan.
⁶ Institut Curie, PSL Research University, Oncology Center SIREDO, Paris, France.
⁷ Department of Children and Adolescent Oncology, Gustave Roussy, Paris-Saclay University, Villejuif, France.
⁸ Faculty of Medicine, Department of Pediatrics, Gynecology and Obstetrics, University Hospital of Geneva-Department of Women, Child, and Adolescent, Onco-hematology Unit and Cansearch Research Platform for Pediatric Oncology and Hematology, University of Geneva, Geneva, Switzerland.
⁹ The Royal Children's Hospital, Melbourne, Australia.
¹⁰ Nemours Children's Hospital, Wilmington, DE, USA.
¹¹ Birmingham Women's and Children's Hospital, Birmingham, UK.
¹² Bicetre Hospital APHP, ParisSaclay University, France.
¹³ Princess Maxima Center for Pediatric Oncology, Utrecht, the Netherlands.
¹⁴ ETOP IBCSG Partners Foundation, Bern, Switzerland.

Abstract

Liver tumors account for approximately 2% of all pediatric malignancies. Children with advanced stages of hepatoblastoma (HB) are cured only 50-70% of the time while children with advanced hepatocellular carcinoma (HCC) have a <20% 5-year overall survival. This scoping review was performed to highlight the paucity of rigorous, reliable data guiding the management of relapsed pediatric HB or HCC. When these patients are enrolled on prospective trials, the trials are often histology-agnostic, exclude patients less than a year of age, lack a liquid formulary of the drug under study, exclude recipients of a solid organ transplant, and enroll only 1-2 patients limiting the ability to deduce efficacious regimens for current use or future study. We highlight the creation of a global pediatric consortium intended to source retrospective relapse data from over 100 institutions spanning 4 continents. The data collected from this effort will inform future relapse trials.

Keywords: Carcinoma; Hepatoblastoma; Pediatric; Registry; Relapse; Review.

Publication types

Review