Prioritization of novel agents for further investigation in pediatric non-rhabdomyosarcoma soft tissue sarcomas: A report from the Children's Oncology Group
Jacquelyn N. Crane, Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; Children's Hospital of Philadelphia, Philadelphia, PA, USA, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. Electronic address: cranej2@chop.edu.
David P. Douglass, Arkansas Children's Hospital, Little Rock, AR, USA; Department of Pediatrics, Division of Hematology/Oncology, University of Arkansas for Medical Sciences, Little Rock, AR, USA. Electronic address: DPDouglass@uams.edu.
Sapna Oberoi, Department of Pediatrics and Child Health, University of Manitoba, Winnipeg, Manitoba, Canada; Department of Pediatric Hematology/Oncology, Cancer Care Manitoba, Winnipeg, Manitoba, Canada, University of Manitoba, Winnipeg, Manitoba, Canada. Electronic address: soberoi@cancercare.mb.ca.
Natalie B. Collins, Harvard Medical School, Boston, MA, USA; Dana-Farber/Boston Children's Cancer and Blood Disorders Center Boston, Boston, MA, USA. Electronic address: natalie_collins@dfci.harvard.edu.
Ajay Gupta, Division of Pediatric Oncology, Department of Pediatrics, Roswell Park Comprehensive Cancer Center, University at Buffalo Jacobs School of Medicine and Biomedical Sciences, Buffalo, NY, USA. Electronic address: Ajay.Gupta@RoswellPark.org.
Abstract
Non-rhabdomyosarcoma soft tissue sarcoma (NRSTS) is a group of over 70 tumors that occur across the age range and account for approximately 4 % of childhood cancers. Patients with metastatic or relapsed NRSTS have a dismal prognosis. The histologic, molecular, and clinical heterogeneity of NRSTS and the rarity of individual types pose unique challenges to the study of these diseases. The Children's Oncology Group (COG) Soft Tissue Sarcoma (STS) Committee established a NRSTS Novel Agents Working Group to identify and prioritize agents for further study in pediatric NRSTS, similar to recent efforts in Ewing sarcoma, rhabdomyosarcoma and osteosarcoma. We report the framework that was used to evaluate agents and the results of this evaluation. Immune checkpoint inhibitors and tyrosine kinase inhibitors were identified by the COG STS Committee as candidate classes of agents for inclusion in phase 2 or phase 3 clinical trials of pediatric NRSTS. Gaps in the existing literature and practical barriers were identified for additional classes of agents to guide future drug development efforts.
