Larynx Preservation Clinical Trial Design: Summary of Key Recommendations of a Consensus Panel
Larynx Preservation Clinical Trial Design: Summary of Key Recommendations of a Consensus Panel
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DOI：
10.1634/theoncologist.2010-s3-25
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发表时间：
2010-10-01
期刊：
影响因子：
5.8
通讯作者：
Ang, K. Kian
中科院分区：
文献类型：
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作者：
Ang, K. Kian
召开了一个国际共识小组，为局部晚期喉癌和下咽癌患者进行保留喉部的第三阶段临床试验制定指南。根据他们的建议，未来的试验人群应该包括T2或T3喉或下咽鳞状细胞癌患者，不考虑进行部分喉切除术，并应排除那些喉部功能障碍或70岁以上的患者。基线和治疗后的功能评估应包括言语和吞咽评估。此外，应定期使用简单、有效的仪器评估嗓音。关于终结点，主要终结点应该考虑生存和功能。因此，该小组创建了无喉-食道功能障碍(LED)生存的新终点，包括死亡、局部复发、全喉或部分喉切除、气管切开=2年或喂养管=2年的事件。推荐的次要终点是不受LED影响、总存活率、无进展存活率、局部区域控制、气管切开时间、喉切除时间、停止喂养管时间以及生活质量/患者报告的结果。未来的探索性相关生物标志物研究应包括表皮生长因子受体、切除修复交叉互补组1基因、E-钙粘附素和β-连环素、表观调节素和两端调节素以及TP53突变。为了推进喉保存的研究，需要在几个关键领域进行修订的试验设计。有了一致的方法，临床试验可以更有效地评估和量化新的治疗方案对局部晚期喉癌和下咽癌患者的治疗益处。《肿瘤学家》2010；15(增刊3)：25-29
An international consensus panel was convened to develop guidelines for the conduct of phase III clinical trials of larynx preservation in patients with locally advanced laryngeal and hypopharyngeal cancer. According to their recommendations, future trial populations should include patients with T2 or T3 laryngeal or hypopharyngeal squamous cell carcinoma not considered for partial laryngectomy and should exclude those with laryngeal dysfunction or aged >70 years. Baseline and post-treatment functional assessments should include speech and swallowing evaluations. Furthermore, voice should be routinely assessed with a simple, validated instrument. Regarding endpoints, the primary endpoint should capture survival and function. As a result, the panel created a new endpoint of laryngoesophageal dysfunction (LED)-free survival, which includes the events of death, local relapse, total or partial laryngectomy, tracheotomy at >= 2 years, or feeding tube at >= 2 years. Recommended secondary endpoints are freedom from LED, overall survival, progression-free survival, locoregional control, time to tracheotomy, time to laryngectomy, time to discontinuation of feeding tube, and quality of life/patient-reported outcomes. Future exploratory correlative biomarker studies should include epidermal growth factor receptor, excision repair cross-complementation group 1 gene, E-cadherin and beta-catenin, epiregulin and amphiregulin, and TP53 mutation.Revised trial designs in several key areas are needed to advance the study of larynx preservation. With consistent methodologies, clinical trials can more effectively evaluate and quantify the therapeutic benefit of novel treatment options for patients with locally advanced laryngeal and hypopharyngeal cancer. The Oncologist 2010;15(suppl 3):25-29