Bisogno Gianni, Sparber-Sauer Monika, Rodeberg David, Merks Johannes H M, Koscielniak Ewa, Wolden Suzanne L, De Salvo Gian Luca, Del Bianco Paola, Xue Wei, Ebinger Martin, Vokuhl Christian, Venkatramani Rajkumar, Volchenboum Samuel L, Furner Brian, Minard Colin Veronique, Hawkins Douglas S
Department of Women's and Children's Health, University of Padua, Padua, Italy.
Pediatric Hematology Oncology Division, University Hospital of Padua, Padua, Italy.
Cancer. 2025 Jul 15;131(14):e35974. doi: 10.1002/cncr.35974.
BACKGROUND: The International Soft Tissue Sarcoma Consortium (INSTRuCT) was established in 2017 to enhance international collaboration. This study describes the characteristics of rhabdomyosarcoma (RMS) patients in the INSTRuCT database and examines differences across contributing groups. METHODS: INSTRuCT includes data Children's Oncology Group (COG), Cooperative Weichteilsarkom Studiengruppe (CWS), and European Paediatric Soft Tissue Sarcoma Study Group (EpSSG) along with prior studies from Malignant Mesenchymal Tumour Committee (MMT) and Italian Soft Tissue Sarcoma Committee (STSC). Data standardization was supported by the University of Chicago's Pediatric Cancer Data Commons. Pseudonymized patient-level data from clinical trials were harmonized. Differences across groups were assessed using χ or Kruskal-Wallis tests. RESULTS: As of March 2025, INSTRuCT includes 6972 RMS patients from 16 trials (1990-2016). Embryonal RMS was the most common histology in all groups (range, 45.4%-62.2%). Alveolar RMS was less frequent in EpSSG (26.8%) although the rate of RMS fusion-positive was comparable across groups (74.6%-81.9%). COG and EpSSG had more T1 tumors, (53.2% and 51.4%) with COG reporting more tumors <5 cm (52%). Nodal involvement was least reported in MMT (15.4%). Metastatic patients were less represented in MMT (11%) and EpSSG (13.3%). Tumor site distribution varied: genitourinary nonbladder/prostate RMS was more common in COG, whereas head and neck nonparameningeal and orbital RMS were more represented in MMT and STSC. MMT had fewer completely resected tumors (8.9%). CONCLUSION: Differences among RMS study populations reflect evolving diagnostic criteria and treatment strategies that should be considered in future analyses. INSTRuCT offers a valuable international data set for RMS research.
背景:国际软组织肉瘤联盟(INSTRuCT)于2017年成立,以加强国际合作。本研究描述了INSTRuCT数据库中横纹肌肉瘤(RMS)患者的特征,并探讨了各参与组之间的差异。 方法:INSTRuCT纳入了儿童肿瘤协作组(COG)、软组织肉瘤合作研究组(CWS)和欧洲儿科软组织肉瘤研究组(EpSSG)的数据,以及恶性间叶肿瘤委员会(MMT)和意大利软组织肉瘤委员会(STSC)之前的研究数据。芝加哥大学儿科癌症数据共享中心支持数据标准化。对来自临床试验的匿名患者水平数据进行了整合。使用χ检验或Kruskal-Wallis检验评估组间差异。 结果:截至2025年3月,INSTRuCT包括来自16项试验(1990 - 2016年)的6972例RMS患者。胚胎型RMS是所有组中最常见的组织学类型(范围为45.4% - 62.2%)。在EpSSG中,肺泡型RMS较少见(26.8%),尽管各组中RMS融合阳性率相当(74.6% - 81.9%)。COG和EpSSG的T1期肿瘤较多(分别为53.2%和51.4%),COG报告的肿瘤<5 cm的比例更高(52%)。MMT报告的淋巴结受累最少(15.4%)。MMT和EpSSG中转移性患者的比例较低(分别为11%和13.3%)。肿瘤部位分布各不相同:泌尿生殖系统非膀胱/前列腺RMS在COG中更常见,而头颈部非脑膜和眼眶RMS在MMT和STSC中更常见。MMT中完全切除的肿瘤较少(8.9%)。 结论:RMS研究人群之间的差异反映了不断演变的诊断标准和治疗策略,在未来的分析中应予以考虑。INSTRuCT为RMS研究提供了一个有价值的国际数据集。
Pediatr Blood Cancer. 2022-11