Division of Gastroenterology, Oregon Health and Science University, Portland, OR, USA.
Medical University of South Carolina, Department of Biostatistics, Charleston, SC, USA.
Pancreatology. 2023 Mar;23(2):187-191. doi: 10.1016/j.pan.2022.12.011. Epub 2022 Dec 21.
Using the ongoing NIDDK-funded multicenter randomized clinical trial, Sphincterotomy for Acute Recurrent Pancreatitis (SHARP) as an example, this article discusses the rationale and key aspects of study design that need to be considered when conducting a clinical trial of endoscopic therapy in acute pancreatitis. SHARP, the first trial using a sham ERCP in the placebo group, is designed to address a decades long controversy in clinical pancreatology, i.e. whether minor papilla sphincterotomy benefits patients with idiopathic acute recurrent pancreatitis who also have pancreas divisum. Although the trial has already enrolled and randomized over 5 times the number of subjects enrolled in the only randomized trial in this area published in 1992 (107 vs. 19), recruitment has been challenging and we are at ∼46% of target recruitment. The review discusses the challenges in the execution of the trial and strategies the SHARP team has used to address these, which investigators planning or considering treatment trials in pancreatitis may find helpful. It will also inform the general gastroenterologists the importance of discussing and referring potentially eligible subjects to centers participating in clinical trials. Developing evidence-based treatment will provide a solid scientific basis for physicians to recommend evidence-based treatments for pancreatitis.
以正在进行的 NIDDK 资助的多中心随机临床试验(Sphincterotomy for Acute Recurrent Pancreatitis,SHARP)为例,本文讨论了在进行急性胰腺炎内镜治疗临床试验时需要考虑的研究设计的基本原理和关键方面。SHARP 是第一个在安慰剂组中使用假内镜逆行胰胆管造影术(ERCP)的试验,旨在解决临床胰腺病学中长达数十年的争议,即对于伴有胰腺分裂的特发性复发性胰腺炎患者,小乳头括约肌切开术是否有益。尽管该试验已经招募并随机分配了超过 1992 年发表的该领域唯一一项随机试验(107 例 vs. 19 例)中招募的患者数量的 5 倍,但招募工作具有挑战性,我们目前仅达到目标招募人数的 46%。本文综述了试验执行过程中的挑战,以及 SHARP 团队为解决这些挑战所采用的策略,这可能对计划或考虑进行胰腺炎治疗试验的研究人员有所帮助。它还将使一般胃肠病学家认识到讨论和向参与临床试验的中心转诊潜在合格患者的重要性。制定基于证据的治疗方法将为医生为胰腺炎患者推荐基于证据的治疗方法提供坚实的科学基础。