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Pacenta HL, Allen-Rhoades W, Langenau D, et al. Prioritization of Novel Agents for Patients with Rhabdomyosarcoma: A Report from the Children's Oncology Group (COG) New Agents for Rhabdomyosarcoma Task Force. J Clin Med. 2021;10(7)doi: 10.3390/jcm10071416.
Pacenta, H. L., Allen-Rhoades, W., Langenau, D., Houghton, P. J., Keller, C., Heske, C. M., Deel, M. D., Linardic, C. M., Shern, J. F., Stewart, E., Turpin, B., Harrison, D. J., Khan, J., Mascarenhas, L., Skapek, S. X., Meyer, W. H., Hawkins, D. S., Chen, E. Y., Amatruda, J. F., Hingorani, P., & Laetsch, T. W. (2021). Prioritization of Novel Agents for Patients with Rhabdomyosarcoma: A Report from the Children's Oncology Group (COG) New Agents for Rhabdomyosarcoma Task Force. Journal of clinical medicine, 10(7), . https://doi.org/10.3390/jcm10071416
Pacenta, Holly L, et al. "Prioritization of Novel Agents for Patients with Rhabdomyosarcoma: A Report from the Children's Oncology Group (COG) New Agents for Rhabdomyosarcoma Task Force." Journal of clinical medicine vol. 10,7 (2021). doi: https://doi.org/10.3390/jcm10071416
Pacenta HL, Allen-Rhoades W, Langenau D, Houghton PJ, Keller C, Heske CM, Deel MD, Linardic CM, Shern JF, Stewart E, Turpin B, Harrison DJ, Khan J, Mascarenhas L, Skapek SX, Meyer WH, Hawkins DS, Chen EY, Amatruda JF, Hingorani P, Laetsch TW. Prioritization of Novel Agents for Patients with Rhabdomyosarcoma: A Report from the Children's Oncology Group (COG) New Agents for Rhabdomyosarcoma Task Force. J Clin Med. 2021 Apr 01;10(7). doi: 10.3390/jcm10071416. PMID: 33915882; PMCID: PMC8037615.
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J Clin Med. 2021 Apr 01;10(7). doi: 10.3390/jcm10071416.

Prioritization of Novel Agents for Patients with Rhabdomyosarcoma: A Report from the Children's Oncology Group (COG) New Agents for Rhabdomyosarcoma Task Force.

Journal of clinical medicine

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 Hingorani, Theodore W Laetsch

Affiliations

  1. Cook Children's Medical Center, Division of Hematology and Oncology, Fort Worth, TX 76014, USA.
  2. Department of Pediatrics and Harold C. Simmons Comprehensive Cancer Center, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
  3. Division of Pediatric Oncology, Mayo Clinic, Rochester, MN 55902, USA.
  4. Molecular Pathology Unit, Massachusetts General Hospital Research Institute, Charlestown, MA 02114, USA.
  5. Department of Molecular Medicine, Greehey Children's Cancer Research Institute, San Antonio, TX 78229, USA.
  6. Children's Cancer Therapy Development Institute, Beaverton, OR 97005, USA.
  7. Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, Bethesda, MD 20892, USA.
  8. Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, USA.
  9. Department of Oncology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
  10. Division of Oncology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.
  11. Division of Pediatrics, University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
  12. Genetics Branch, Center for Cancer Research, National Cancer Institute, Bethesda, MD 20892, USA.
  13. Cancer and Blood Disease Institute, Children's Hospital of Los Angeles, Los Angeles, CA 90027, USA.
  14. Keck School of Medicine, University of Southern California, Los Angeles, CA 90033, USA.
  15. Department of Pediatrics, Jimmy Everest Section of Pediatric Hematology/Oncology, University of Oklahoma Health Sciences Center, Oklahoma, OK 73104, USA.
  16. Division of Hematology/Oncology, Seattle Children's Hospital, University of Washington, Seattle, WA 98105, USA.
  17. Department of Laboratory Medicine and Pathology, University of Washington, Seattle, WA 98195, USA.
  18. Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
  19. Department of Pediatrics and Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA 19104, USA.

PMID: 33915882 PMCID: PMC8037615 DOI: 10.3390/jcm10071416

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.

Keywords: clinical trials; metastasis; new agents; relapse; rhabdomyosarcoma

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