Rechberger Julian S, Lu Victor M, Zhang Liang, Power Erica A, Daniels David J
Department of Neurologic Surgery, Mayo Clinic, 200 First St. SW, Rochester, MN, 55905, USA.
Mayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Rochester, MN, USA.
Childs Nerv Syst. 2020 Jan;36(1):39-46. doi: 10.1007/s00381-019-04363-1. Epub 2019 Sep 6.
Diffuse intrinsic pontine glioma (DIPG) is a lethal high-grade pediatric brainstem tumor without a cure. Despite numerous clinical trials over the last decades, the prognosis has remained poor. The aim of this update was to report on the status and outcomes of all clinical trials for DIPG performed to better understand the landscape of research efforts for this diagnosis to date.
The ClinicalTrials.gov database was reviewed in May 2019 for all possible interventional clinical trials that included DIPG as a diagnosis of primary investigation. These were then screened against selection criteria to identify pertinent clinical trials.
Ninety-five clinical trials satisfied all inclusion criteria, with 55 (58%) trials specific to the DIPG diagnosis only. In terms of the most prevalent design features, 42 (44%) were phase I trials, with median expected start and completion years in 2011 (range, 1994-2020) and 2018 (range, 2005-2047), respectively. Median target number of patients to enroll was 38 (range, 1-1500), and the most common primary outcome was safety and toxicity (56%). There were 69 (73%) trials originating from the USA, with 49 (52%) of them being single institutional. Only 10 (11%) trials have reported results to date.
To date, 95 clinical trials investigating DIPG with specific emphasis have been registered on ClinicalTrials.gov. There were only a small number of trials that had study results available, and they uniformly reported non-significant improvement to prognosis. Given the rarity and lethality of DIPG, which limits the accumulation of large cohorts, our results mandate the need for more robust, systematic clinical trial design to minimize redundancies and maximize yield in the future.
弥漫性脑桥内生型胶质瘤（DIPG）是一种致命的儿童高级别脑干肿瘤，无法治愈。尽管在过去几十年中进行了大量临床试验，但其预后仍然很差。本次更新的目的是报告所有针对DIPG开展的临床试验的现状和结果，以便更好地了解迄今为止针对该诊断的研究工作情况。
2019年5月对ClinicalTrials.gov数据库进行了检索，查找所有将DIPG作为主要研究诊断的可能的介入性临床试验。然后根据入选标准对这些试验进行筛选，以确定相关的临床试验。
95项临床试验符合所有纳入标准，其中55项（58%）试验仅针对DIPG诊断。就最常见的设计特征而言，42项（44%）为I期试验，预期开始和完成年份的中位数分别为2011年（范围为1994 - 2020年）和2018年（范围为2005 - 2047年）。计划招募的患者目标数量中位数为38名（范围为1 - 1500名），最常见的主要结局是安全性和毒性（56%）。有69项（73%）试验来自美国，其中49项（52%）为单机构试验。迄今为止，只有10项（11%）试验报告了结果。
迄今为止，在ClinicalTrials.gov上已注册了95项专门针对DIPG进行研究的临床试验。只有少数试验有研究结果，且均报告对预后无显著改善。鉴于DIPG的罕见性和致死性限制了大型队列的积累，我们的结果表明需要更稳健、系统的临床试验设计，以尽量减少冗余并在未来最大化研究产出。
