Department of Neurosurgery, Penn State Hershey Medical Center, Hershey, Pennsylvania, USA.
Penn State Cancer Institute, Hershey, Pennsylvania, USA.
Neurosurgery. 2021 Jul 15;89(2):196-203. doi: 10.1093/neuros/nyab169.
The therapeutic challenge of glioblastoma (GBM) has catalyzed the development of clinical trials to evaluate novel interventions. With increased understanding of GBM biology and technological advances, the neurosurgeon's role in neuro-oncology has evolved.
To evaluate the current landscape of procedure-based clinical trials for GBM to characterize this evolution, gain insight into past failures, and accordingly outline implications for future research and practice that may inform future studies.
The ClinicalTrials.gov database was searched for surgical/procedural trials in individuals with GBM. Demographics, specific intervention, trial phase, and main outcome measures were abstracted.
A total of 224 of 2311 GBM trials (9.7%) were identified as procedural, with the majority being based in the United States (155/224, 69.2%), single-center (155/224, 69.2%), and not randomized (176/224, 78.6%). Primary and recurrent GBMs were evenly addressed. The leading interventions were local delivery of therapeutics (50.0%), surgical techniques (33.9%), such as image-guided surgery, and novel device applications (14.3%). Phase I designs predominated (82/224, 36.6%). The top primary outcome was safety/tolerability/feasibility (88/224, 39.3%), followed by survival (46/224, 20.5%). Approximately 17% of studies were terminated, withdrawn, or suspended. Fifty-two linked publications were identified, among which 42 were classified as having a positive result.
Procedural interventions comprised ∼10% of all registered GBM trials. Local delivery of therapeutics, use of surgical imaging techniques and novel device applications, predominantly through phase I designs, represent the evolved role of the neurosurgeon in neuro-oncology. Improved reporting of trial designs, outcomes, and results are needed to better inform the field and increase efficiency.
胶质母细胞瘤（GBM）的治疗挑战促使人们开展临床试验，以评估新的干预措施。随着对 GBM 生物学的深入了解和技术进步，神经外科医生在神经肿瘤学中的角色也发生了演变。
评估目前 GBM 基于手术的临床试验现状，以描述这一演变过程，深入了解过去的失败，并据此为未来的研究和实践提出建议，为未来的研究提供信息。
在 ClinicalTrials.gov 数据库中搜索针对 GBM 患者的手术/程序试验。提取试验的人口统计学、具体干预措施、试验阶段和主要结局指标。
共确定了 2311 项 GBM 试验中的 224 项（9.7%）为手术/程序试验，其中大多数来自美国（155/224，69.2%）、单中心（155/224，69.2%），且未随机（176/224，78.6%）。原发和复发性 GBM 均有涉及。主要干预措施包括局部药物输送（50.0%）、手术技术（33.9%），如影像引导手术和新型设备应用（14.3%）。以Ⅰ期设计为主（82/224，36.6%）。主要的初始结果是安全性/耐受性/可行性（88/224，39.3%），其次是生存（46/224，20.5%）。约 17%的研究被终止、撤回或暂停。共确定了 52 篇相关文献，其中 42 篇被归类为阳性结果。
手术干预占所有登记的 GBM 试验的约 10%。局部药物输送、使用手术影像技术和新型设备应用，主要通过Ⅰ期设计，代表了神经外科医生在神经肿瘤学中的角色演变。需要更好地报告试验设计、结果和结果，以更好地为该领域提供信息并提高效率。
