In the modern era of targeted and immune-based therapies, investigator and patient expectations of availability and efficacy in phase I trials have increased. We assessed availability of, and benefit from, early drug development trials, specifically in patients with gastrointestinal cancers. We reviewed computerized referral records of the Early Drug Development Service at our institution to identify patients internally referred from our Gastrointestinal Oncology Service in calendar year 2018. End points were treatment on a trial, 3- and 6-month progression-free survival (PFS), and any tumor shrinkage. Of 394 gastrointestinal cancer patients referred in 2018, 54 enrolled on a trial and 53 (13.5%) were treated (1 withdrew before treatment): 34 on immune-based and 19 on targeted (3 to phase II basket) studies. None of the 52 patients who had exhausted standard therapy achieved 6-month PFS, two (3.8%) met 3-month PFS with tumor growth below Response Evaluation Criteria in Solid Tumors progression at 3 months, and both came off study for progression at 4 months. One patient who was to receive an irinotecan-based regimen as standard therapy instead received irinotecan plus an investigational targeted agent and remained stable for 8 months. No patients achieved any degree of tumor shrinkage. The most common reasons for nonaccrual were lack of available protocol treatment openings and failure to meet eligibility criteria for specific trials. Thus, availability and benefit from investigational treatment in this treatment-refractory gastrointestinal cancer patient population was extremely modest. Expectations regarding both availability and efficacy of phase I investigational therapy in gastrointestinal cancer patients likely exceed what our experience suggests.

中文翻译：

---

对于胃肠道癌患者，I期试验和篮式试验的有效性和有效性缺乏。

在靶向和基于免疫疗法的现代时代，研究人员和患者对I期临床试验可获得性和疗效的期望有所提高。我们评估了早期药物开发试验的可用性，并从中受益，尤其是在胃肠道癌患者中。我们审查了本机构早期药物开发服务的计算机转诊记录，以识别在2018日历年从胃肠道肿瘤服务部门内部转诊的患者。终点为试验，3个月和6个月无进展生存期（PFS）的治疗，以及任何肿瘤缩小。在2018年转诊的394位胃肠道癌症患者中，有54位参加了试验，有53位（13.5％）得到了治疗（1位在治疗前退出）：基于免疫的研究34位，针对有针对性的研究（II期3至3期研究）。用尽标准疗法的52例患者中没有一个达到6个月的PFS，两个（3.8％）达到3个月的PFS，且肿瘤生长低于3个月时实体瘤进展的缓解评估标准，并且都在4岁时退出研究个月。一位接受基于伊立替康的方案作为标准疗法的患者改为接受伊立替康加研究性靶向药物，并保持稳定8个月。没有患者达到任何程度的肿瘤缩小。不能进行应计的最常见原因是缺乏可用的方案治疗开口和未能满足特定试验的入选标准。因此，在这种难治性胃肠道癌症患者人群中，研究治疗的可得性和收益是非常有限的。