一线分子靶向治疗晚期脊索瘤：疗效与预后因素。法国肉瘤研究组（GSF/GETO）和法国神经肿瘤学家协会（ANOCEF）的一项回顾性研究。
Advanced chordoma treated by first-line molecular targeted therapies: Outcomes and prognostic factors. A retrospective study of the French Sarcoma Group (GSF/GETO) and the Association des Neuro-Oncologues d'Expression Française (ANOCEF).
作者信息
Lebellec Loïc, Chauffert Bruno, Blay Jean-Yves, Le Cesne Axel, Chevreau Christine, Bompas Emmanuelle, Bertucci François, Cupissol Didier, Fabbro Michel, Saada-Bouzid Esma, Duffaud Florence, Feuvret Loïc, Bonneville-Levard Alice, Bay Jacques-Olivier, Vauleon Elodie, Vinceneux Armelle, Noel Georges, Penel Nicolas, Mir Olivier
机构信息
Department of General Oncology, Centre Oscar Lambret, Lille, France.
Department of Medical Oncology, University Hospital (CHU), Amiens, France.
出版信息
Eur J Cancer. 2017 Jul;79:119-128. doi: 10.1016/j.ejca.2017.03.037. Epub 2017 May 4.
BACKGROUND
To assess the role of first-line Molecular Targeted Therapies (MTTs) in Advanced chordoma (AC) patients.
METHODS
Retrospective study of 80 patients treated between January 2004 and December 2015 at 15 major French Sarcoma or Neurooncology Centres.
RESULTS
The sex ratio M/F was 46/34. The median age was 59 (6-86) years. The primary sites were the sacrum (50, 62.5%), mobile spine (12, 15.0%), and skull base (18, 22.5%). Metastases were present in 28 patients (36.0%). The first line of MTTs consisted of imatinib (62, 77.5%), sorafenib (11, 13.7%), erlotinib (5, 6.3%), sunitinib (1, 1.2%) and temsirolimus (1, 1.2%). The reported responses were: partial response (5, 6.3%), stable disease (58, 72.5%), or progressive disease (10, 12.5%). Symptomatic improvement was seen in 28/66 assessable patients (42.4%) and was associated with an objective response occurrence (p = 0.005), imatinib (p = 0.020) or erlotinib use (p = 0.028). The median progression-free survival (PFS) was 9.4°months (95% CI, [6.8-16.1]). Two independent factors of poor prognosis for PFS were identified: a skull-based primary location (HR = 2.5, p = 0.019), and the interval between diagnosis and MTT of <52months (HR = 2.8, p < 0.001). The median overall survival (OS) was 4.4°years (95% CI, [3.8-5.6]). Four independent factors of poor prognosis for OS were identified: the presence of liver metastases (HR = 13.2, p < 0.001), pain requiring opioids (HR = 2.9, p = 0.012), skull-based primary location (HR = 19.7, p < 0.001), and prior radiotherapy (photon alone) (HR = 2.5, p = 0.024). The PFS and OS did not significantly differ between the MTT.
CONCLUSIONS
The prognostic factors identified require validation in an independent database but are potently useful to guide treatment decisions and design further clinical trials.
背景
评估一线分子靶向治疗（MTT）在晚期脊索瘤（AC）患者中的作用。
方法
对2004年1月至2015年12月期间在法国15个主要肉瘤或神经肿瘤中心接受治疗的80例患者进行回顾性研究。
结果
男女比例为46/34。中位年龄为59岁（6 - 86岁）。原发部位为骶骨（50例，62.5%）、活动脊柱（12例，15.0%）和颅底（18例，22.5%）。28例患者（36.0%）出现转移。一线MTT包括伊马替尼（62例，77.5%）、索拉非尼（11例，13.7%）、厄洛替尼（5例，6.3%）、舒尼替尼（1例，1.2%）和替西罗莫司（1例，1.2%）。报告的反应为：部分缓解（5例，6.3%）、疾病稳定（58例，72.5%）或疾病进展（10例，12.5%）。66例可评估患者中有28例（42.4%）出现症状改善，且与客观反应的发生相关（p = 0.005）、伊马替尼的使用（p = 0.020）或厄洛替尼的使用（p = 0.028）。中位无进展生存期（PFS）为9.4个月（95%置信区间，[6.8 - 16.1]）。确定了两个PFS预后不良的独立因素：颅底原发部位（HR = 2.5，p = 0.019），以及诊断与MTT之间的间隔<52个月（HR = 2.8，p < 0.001）。中位总生存期（OS）为4.4年（95%置信区间，[3.8 - 5.6]）。确定了四个OS预后不良的独立因素：肝转移的存在（HR = 13.2，p < 0.001）、需要使用阿片类药物止痛（HR = 2.9，p = 0.012）、颅底原发部位（HR = 19.7，p < 0.001）以及既往放疗（仅光子放疗）（HR = 2.5，p = 0.024）。MTT之间的PFS和OS无显著差异。
结论
所确定的预后因素需要在独立数据库中进行验证，但对指导治疗决策和设计进一步的临床试验非常有用。
Zotero 插件