Insights into pediatric rhabdomyosarcoma research: Challenges and goals Review


Authors: Yohe, M. E.; Heske, C. M.; Stewart, E.; Adamson, P. C.; Ahmed, N.; Antonescu, C. R.; Chen, E.; Collins, N.; Ehrlich, A.; Galindo, R. L.; Gryder, B. E.; Hahn, H.; Hammond, S.; Hatley, M. E.; Hawkins, D. S.; Hayes, M. N.; Hayes-Jordan, A.; Helman, L. J.; Hettmer, S.; Ignatius, M. S.; Keller, C.; Khan, J.; Kirsch, D. G.; Linardic, C. M.; Lupo, P. J.; Rota, R.; Shern, J. F.; Shipley, J.; Sindiri, S.; Tapscott, S. J.; Vakoc, C. R.; Wexler, L. H.; Langenau, D. M.
Review Title: Insights into pediatric rhabdomyosarcoma research: Challenges and goals
Abstract: 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. © 2019 Wiley Periodicals, Inc.
Keywords: genomics; rhabdomyosarcoma; cancer biology; early-phase clinical trials
Journal Title: Pediatric Blood and Cancer
Volume: 66
Issue: 10
ISSN: 1545-5009
Publisher: Wiley Periodicals, Inc  
Date Published: 2019-10-01
Start Page: e27869
Language: English
DOI: 10.1002/pbc.27869
PUBMED: 31222885
PROVIDER: scopus
PMCID: PMC6707829
DOI/URL:
Notes: Review -- Export Date: 30 August 2019 -- Source: Scopus
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  1. Leonard H Wexler
    191 Wexler
  2. Cristina R Antonescu
    895 Antonescu