Chisholm Julia, Mandeville Henry, Adams Madeleine, Minard-Collin Veronique, Rogers Timothy, Kelsey Anna, Shipley Janet, van Rijn Rick R, de Vries Isabelle, van Ewijk Roelof, de Keizer Bart, Gatz Susanne A, Casanova Michela, Hjalgrim Lisa Lyngsie, Firth Charlotte, Wheatley Keith, Kearns Pamela, Liu Wenyu, Kirkham Amanda, Rees Helen, Bisogno Gianni, Wasti Ajla, Wakeling Sara, Heenen Delphine, Tweddle Deborah A, Merks Johannes H M, Jenney Meriel
Children and Young People's Unit, Royal Marsden Hospital and Institute of Cancer Research, Sutton SM2 5PT, UK.
Children's Hospital for Wales, Cardiff CF14 4XW, UK.
Cancers (Basel). 2024 Feb 29;16(5):998. doi: 10.3390/cancers16050998.
The Frontline and Relapsed Rhabdomyosarcoma (FaR-RMS) clinical trial is an overarching, multinational study for children and adults with rhabdomyosarcoma (RMS). The trial, developed by the European Soft Tissue Sarcoma Study Group (EpSSG), incorporates multiple different research questions within a multistage design with a focus on (i) novel regimens for poor prognostic subgroups, (ii) optimal duration of maintenance chemotherapy, and (iii) optimal use of radiotherapy for local control and widespread metastatic disease. Additional sub-studies focusing on biological risk stratification, use of imaging modalities, including [F]FDG PET-CT and diffusion-weighted MRI imaging (DWI) as prognostic markers, and impact of therapy on quality of life are described. This paper forms part of a Special Issue on rhabdomyosarcoma and outlines the study background, rationale for randomisations and sub-studies, design, and plans for utilisation and dissemination of results.
一线及复发性横纹肌肉瘤（FaR-RMS）临床试验是一项针对儿童和成人横纹肌肉瘤（RMS）的综合性跨国研究。该试验由欧洲软组织肉瘤研究组（EpSSG）开展，在多阶段设计中纳入了多个不同的研究问题，重点关注：（i）预后不良亚组的新型治疗方案；（ii）维持化疗的最佳疗程；（iii）局部控制和广泛转移性疾病放疗的最佳应用。还描述了其他子研究，重点关注生物学风险分层、成像模态的应用，包括将[F]FDG PET-CT和扩散加权MRI成像（DWI）作为预后标志物，以及治疗对生活质量的影响。本文是横纹肌肉瘤特刊的一部分，概述了研究背景、随机分组及子研究的基本原理、设计以及结果利用和传播计划。
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