Background: It is unclear as to where we stand with respect to utilizing emerging focused ultrasound (FUS) technology in the setting of brain tumor treatment in pediatric patients, such as malignant diffuse intrinsic pontine glioma, and various adult counterparts. Correspondingly, the aim of this study was to present a contemporary summary of all pertinent clinical trials to date. Methods: The ClinicalTrials.gov database was reviewed in January 2025 for all possible clinical trials involving FUS in the management of brain tumors. These were then screened against selection criteria to identify pertinent clinical trials. Results: A total of 30 clinical trials were identified. The majority were focused on adult patients (24/30, 80%), with the most common tumor type being glioblastoma (GBM) (14/30, 47%). There were also trials focused on pediatric patients only (5/30, 17%), as well as diffuse intrinsic pontine glioma (DIPG) (5/30, 17%). The most prevalent primary outcome of interest was safety (26/30, 87%). The majority of trials were active, either recruiting currently (12/30, 40%), or active but not recruiting currently (3/30, 10%). North America (22/30, 73%) was the most common location for the primary coordinating institution, and the median number of institutions per trial was one. The median expected start year for all trials was 2021, and the completion year was 2024. To date, there have been no results (interim or final) formally reported, although preliminary reports in the literature indicate this to be a safe procedure. Anecdotal trends suggest later trials target the blood-brain barrier more, involve more pediatric patients, and are more based in the United States. Conclusion: There exists a number of early-stage clinical trials investigating FUS to treat a variety of brain tumors in pediatric patients, as well as adult patients, with a significant clinical potential to improve outcomes. To date, no official results have been published, however anecdotal evidence is promising, and a number of results are expected soon.
背景：目前尚不明确聚焦超声（FUS）这一新兴技术在儿童脑肿瘤（如恶性弥漫内生型脑桥胶质瘤）及各类成人脑肿瘤治疗中的应用现状。为此，本研究旨在系统梳理迄今所有相关临床试验的最新进展。方法：于2025年1月检索ClinicalTrials.gov数据库中所有涉及FUS治疗脑肿瘤的临床试验，依据筛选标准确定相关研究。结果：共纳入30项临床试验。其中多数研究针对成人患者（24/30，80%），最常见肿瘤类型为胶质母细胞瘤（14/30，47%）。另有专门针对儿童患者（5/30，17%）及弥漫内生型脑桥胶质瘤（5/30，17%）的试验。主要研究终点以安全性评估最为普遍（26/30，87%）。多数试验处于活跃状态，包括正在招募（12/30，40%）和活跃但暂停招募（3/30，10%）两类。主要协调机构多位于北美地区（22/30，73%），单中心试验占比最高。所有试验的中位启动年份为2021年，中位完成年份为2024年。截至目前虽无正式公布的阶段性或最终结果，但文献初步报告显示该技术具有安全性。现有趋势表明，后期试验更侧重于血脑屏障靶向治疗，更多纳入儿童患者，且美国主导的研究比例增加。结论：目前已有若干早期临床试验探索FUS治疗儿童及成人脑肿瘤的临床应用，该技术具有改善预后的重要潜力。虽尚无官方结果公布，但现有证据显示前景可观，多项研究成果有望近期发布。
肿瘤(癌症)患者之家