Department of Surgery, University of Pittsburgh Medical Center, Pittsburgh, PA, USA.
Division of GI Surgical Oncology, UPMC Pancreatic Cancer Center, University of Pittsburgh Medical Center, UPMC Cancer Pavilion, Pittsburgh, PA, USA.
J Gastrointest Surg. 2022 Aug;26(8):1628-1636. doi: 10.1007/s11605-022-05387-w. Epub 2022 Jun 17.
Hepatopancreaticobiliary (HPB) diseases carry high morbidity despite efforts aimed at their reduction. An assessment of their trial characteristics is paramount to determine trial design adequacy and highlight areas for improvement. As such, the aim of this study is to assess HPB surgery trial characteristics, summarize logistic, financial, and practical reasons behind early discontinuation, and propose potential interventions to prevent this in the future.
All clinical trials investigating HPB surgery registered on ClinicalTrials.gov from October 1st, 2007 (inclusive), to April 20th, 2021 (inclusive), were examined. Trial characteristics were collected including, but not limited to, study phase, duration, patient enrollment size, location, and study design. Peer-reviewed publications associated with the selected trials were also assessed to determine outcome reporting.
A total of 1776 clinical trials conducted in 43 countries were identified, the majority of which were conducted in the USA. Of these trials, 32% were reported as "completed" whereas 12% were "discontinued." The most common cause of trial discontinuation was low accrual, which was reported in 37% of terminated studies. These resulted in 413 published studies. Most trials had multiple assignment, randomized, or open-label designs. Treatment was the most common study objective (73%) with pharmacological therapy being the most commonly studied intervention.
The main reasons for early discontinuation of clinical trials in HPB surgery are poor patient recruitment and inadequate funding. Improved trial design, recruitment strategies and increased funding are needed to prevent trial discontinuation and increase publication rates of HPB surgery clinical trials.
尽管人们努力降低肝胆管疾病的发病率，但这些疾病的发病率仍然很高。评估其试验特征对于确定试验设计的充分性和突出需要改进的领域至关重要。因此，本研究旨在评估肝胆管手术试验特征，总结早期终止的逻辑、财务和实际原因，并提出未来预防这种情况的潜在干预措施。
检查了 2007 年 10 月 1 日（含）至 2021 年 4 月 20 日（含）期间在 ClinicalTrials.gov 上注册的所有肝胆管手术临床试验。收集了试验特征，包括但不限于研究阶段、持续时间、患者入组规模、地点和研究设计。还评估了与选定试验相关的同行评审出版物，以确定结果报告。
确定了 1776 项在 43 个国家进行的临床试验，其中大多数在美国进行。这些试验中，32%报告为“完成”，12%报告为“中止”。试验中止的最常见原因是低入组率，在 37%终止的研究中报告了这一原因。这导致了 413 项已发表的研究。大多数试验都有多种分配、随机或开放标签设计。治疗是最常见的研究目标（73%），药物治疗是最常研究的干预措施。
肝胆管手术临床试验早期终止的主要原因是患者招募不佳和资金不足。需要改进试验设计、招募策略和增加资金，以防止肝胆管手术临床试验的终止并提高其发表率。
