Prioritization of novel agents for patients with rhabdomyosarcoma: A report from the children’s oncology group (cog) new agents for rhabdomyosarcoma task force

Holly L. Pacenta, Wendy Allen-Rhoades, David Langenau, Peter J. Houghton, Charles Keller, Christine M. Heske, Michael D. Deel, Corinne M. Linardic, Jack F. Shern, Elizabeth Stewart, Brian Turpin, Douglas J. Harrison, Javed Khan, Leo Mascarenhas, Stephen X. Skapek, William H. Meyer, Douglas S. Hawkins, Eleanor Y. Chen, James F. Amatruda, Pooja HingoraniTheodore W. Laetsch

Research output: Contribution to journalReview articlepeer-review

Abstract

Rhabdomyosarcoma is the most common soft tissue sarcoma diagnosed in children and adolescents. Patients that are diagnosed with advanced or relapsed disease have exceptionally poor outcomes. The Children’s Oncology Group (COG) convened a rhabdomyosarcoma new agent task force in 2020 to systematically evaluate novel agents for inclusion in phase 2 or phase 3 clinical trials for patients diagnosed with rhabdomyosarcoma, following a similar effort for Ewing sarcoma. The task force was comprised of clinicians and basic scientists who collectively identified new agents for evaluation and prioritization in clinical trial testing. Here, we report the work of the task force including the framework upon which the decisions were rendered and review the top classes of agents that were discussed. Representative agents include poly-ADP-ribose polymerase (PARP) inhibitors in combination with cytotoxic agents, mitogen-activated protein kinase (MEK) inhibitors in combination with type 1 insulin-like growth factor receptor (IGFR1) inhibitors, histone deacetylase (HDAC) inhibitors, and novel cytotoxic agents.

Original languageEnglish (US)
Article number1416
JournalJournal of Clinical Medicine
Volume10
Issue number7
DOIs
StatePublished - Apr 1 2021

Keywords

  • Clinical trials
  • Metastasis
  • New agents
  • Relapse
  • Rhabdomyosarcoma

ASJC Scopus subject areas

  • Medicine(all)

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