EClinicalMedicine (Mar 2024)

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

  • 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

Journal volume & issue
Vol. 69
p. 102446

Abstract

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Summary: 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.

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