Seattle Children's Hospital, University of Washington, and Fred Hutchinson Cancer Research Center, Seattle, WA 98015, USA.
Pediatr Blood Cancer. 2013 Jun;60(6):1001-8. doi: 10.1002/pbc.24435. Epub 2012 Dec 19.
In the US, approximately 850-900 children are diagnosed each year with soft tissue sarcomas (STS). Key findings from recent Children's Oncology Group (COG) clinical trials include safe reduction in therapy for low risk rhabdomyosarcoma (RMS), validation of FOXO1 fusion as a prognostic factor, a modest improvement in outcome for high-risk RMS, and a biologically designed non-cytotoxic therapy for pediatric desmoid tumor. Planned Phase 2 trials include targeted agents for VEGF/PDGF, mTOR, and IGF-1R for children with RMS and VEGF for children with non-RMS STS (NRSTS). For RMS, COG Phase 3 trials potentially will explore VEGF/mTOR inhibition or chemotherapy interval compression. For NRSTS, a COG Phase 3 trial will explore VEGF inhibition.
在美国，每年大约有 850-900 名儿童被诊断患有软组织肉瘤（STS）。最近儿童肿瘤学组（COG）临床试验的主要发现包括：横纹肌肉瘤（RMS）低风险患者的治疗安全减少，FOXO1 融合作为预后因素得到验证，高危 RMS 患者的结果略有改善，以及针对儿科硬纤维瘤的生物学设计的非细胞毒性治疗。计划进行的 2 期试验包括针对 RMS 患儿的 VEGF/PDGF、mTOR 和 IGF-1R 的靶向药物，以及针对非 RMS STS（NRSTS）患儿的 VEGF。对于 RMS，COG 的 3 期试验可能会探索 VEGF/mTOR 抑制或化疗间隔压缩。对于 NRSTS，COG 的 3 期试验将探索 VEGF 抑制。
