Children's Oncology Group's 2023 blueprint for research: Soft tissue sarcomas

Abstract

In the United States, approximately 850–900 children and adolescents each year are diagnosed with soft tissue sarcomas (STS). STS are divided into rhabdomyosarcoma (RMS) and non‐rhabdomyosarcoma STS (NRSTS). RMS and NRSTS are risk stratified into low‐, intermediate‐, and high‐risk categories, with 5‐year survival rates of approximately 90%, 50%–70%, and 20%, respectively. Recent key achievements from the Children's Oncology Group (COG) STS Committee include the identification of new molecular prognostic factors for RMS, development and validation of a novel risk stratification system for NRSTS, successful completion of a collaborative NRSTS clinical trial with adult oncology consortia, and collaborative development of the INternational Soft Tissue SaRcoma ConsorTium (INSTRuCT). Current COG trials for RMS are prospectively evaluating a new risk stratification system that incorporates molecular findings, de‐intensification of therapy for a very low‐risk subgroup, and augmented therapy approaches for intermediate‐ and high‐risk RMS. Trials for NRSTS exploring novel targets and local control modalities are in development.

Event‐free survival in relapsed and refractory rhabdomyosarcoma treated on cooperative group phase II trials: A report from the Children's Oncology Group

“…While rhabdomyosarcoma (RMS) cure rates exceed 70%, certain subsets of patients remain at higher risk of recurrence, including those with unfavorable histology, primary disease at unfavorable sites, and metastatic disease at diagnosis. 1,2 Relapsed and refractory RMS (RRMS), excluding patients with initially low-risk features, carries a dismal prognosis, with a 5-year overall survival (OS) from first recurrence of less than 20%. 3 Ongoing investigation of novel therapies and treatment strategies is essential to improve survival for RRMS.…”

“…7,8 While the myelosuppressive toxicity seen with rebeccamycin analog halted its further development, vinorelbine has been further developed in multiple RMS COG studies: a relapsed combination trial (ARST0921), a randomized phase 3 intermediate-risk trial where it was incorporated into maintenance chemotherapy with oral cyclophosphamide (ARST1431), and an ongoing high-risk randomized phase III clinical trial (ARST2031). 2,9 The use of radiologic response as an activity endpoint in RRMS provides multiple limitations. First, several studies have shown conflicting findings between the relationship of an early radiographic response and long-term outcome in newly diagnosed RMS.…”