文章：

脊索瘤：临床试验全面系统综述

Chordoma: A Comprehensive Systematic Review of Clinical Trials

原文发布日期：11 December 2023

DOI: 10.3390/cancers15245800

类型: Article

开放获取: 是

英文摘要：

This systematic review aims to characterize ongoing clinical trials and therapeutic treatment options for chordoma, a rare notochordal remnant tumor that primarily affects the cranial base, mobile spine, and sacrum. While radical surgical resection remains the cornerstone for chordoma management, unique technical challenges posed by its proximity to critical neurovascular structures confer a tendency towards disease recurrence which often requires additional treatment modalities. In an attempt to better understand the current treatment landscape, a systematic review was designed to identify clinical trials directed at chordoma. A total of 108 chordoma trials were identified from four clinical trial databases; fifty-one trials were included in the final analysis, of which only 14 were designated as completed (27.5%). Aggregate data suggests most chordoma interventions are repurposed from other neoplasms that share common molecular pathways, with a recent emphasis on combination therapeutics within and across drug classes. Naturally, the publication and dissemination of clinical trial results remain a concern (n= 4, 28.6%), highlighting the need for enhanced reporting and transparency measures. Active clinical trial efforts are quite promising, with a renewed focus on novel biotherapeutic targets and deciphering the natural history, as well as survivorship of this complex disease.

摘要翻译： 

本系统综述旨在梳理脊索瘤（一种罕见的脊索残余肿瘤，主要累及颅底、活动脊柱和骶骨）的现有临床试验及治疗方案特征。尽管根治性手术切除仍是脊索瘤治疗的基石，但由于肿瘤毗邻重要神经血管结构带来的特殊技术挑战，疾病易出现复发，往往需要联合其他治疗方式。为深入理解当前治疗现状，本研究通过系统综述方法识别针对脊索瘤的临床试验。从四个临床试验数据库中共筛选出108项脊索瘤试验，其中51项纳入最终分析，仅14项（27.5%）被标记为已完成。汇总数据显示，多数脊索瘤干预方案源自具有共同分子通路的其他肿瘤的疗法再利用，近期研究重点转向同类药物及跨类别药物的联合治疗。值得注意的是，临床试验结果的公布与传播仍显不足（n=4，28.6%），凸显了加强报告规范与透明度的必要性。当前活跃的临床试验前景可观，研究重点正重新聚焦于新型生物治疗靶点的探索，以及对此复杂疾病自然史和生存状况的解析。

原文链接：

Chordoma: A Comprehensive Systematic Review of Clinical Trials