Insights into pediatric rhabdomyosarcoma research: Challenges and goals.

Journal Article (Journal Article;Review)

Overall survival rates for pediatric patients with high-risk or relapsed rhabdomyosarcoma (RMS) have not improved significantly since the 1980s. Recent studies have identified a number of targetable vulnerabilities in RMS, but these discoveries have infrequently translated into clinical trials. We propose streamlining the process by which agents are selected for clinical evaluation in RMS. We believe that strong consideration should be given to the development of combination therapies that add biologically targeted agents to conventional cytotoxic drugs. One example of this type of combination is the addition of the WEE1 inhibitor AZD1775 to the conventional cytotoxic chemotherapeutics, vincristine and irinotecan.

Full Text

Duke Authors

Cited Authors

  • Yohe, ME; Heske, CM; Stewart, E; Adamson, PC; Ahmed, N; Antonescu, CR; Chen, E; Collins, N; Ehrlich, A; Galindo, RL; Gryder, BE; Hahn, H; Hammond, S; Hatley, ME; Hawkins, DS; Hayes, MN; Hayes-Jordan, A; Helman, LJ; Hettmer, S; Ignatius, MS; Keller, C; Khan, J; Kirsch, DG; Linardic, CM; Lupo, PJ; Rota, R; Shern, JF; Shipley, J; Sindiri, S; Tapscott, SJ; Vakoc, CR; Wexler, LH; Langenau, DM

Published Date

  • October 2019

Published In

Volume / Issue

  • 66 / 10

Start / End Page

  • e27869 -

PubMed ID

  • 31222885

Pubmed Central ID

  • PMC6707829

Electronic International Standard Serial Number (EISSN)

  • 1545-5017

Digital Object Identifier (DOI)

  • 10.1002/pbc.27869


  • eng

Conference Location

  • United States