Cheng Vinton W T, Heywood Richard, Zakaria Rasheed, Burger Rebecca, Zucker Kieran, Kannan Siddarth, Putra Muhammad Alifian Remifta, Fitzpatrick Amanda, Doherty Gary, Sanghera Paul, Jenkinson Michael D, Palmieri Carlo
Leeds Institute of Medical Research, St James's University Hospital, University of Leeds, Leeds, UK.
Department of Cancer and Genomic Sciences, University of Birmingham, Birmingham, UK.
Neuro Oncol. 2024 Dec 5;26(12):2193-2207. doi: 10.1093/neuonc/noae140.
Recent studies have challenged the notion that patients with brain metastasis (BM) or leptomeningeal metastasis (LM) should be excluded from systemic therapy clinical trials. This scoping study summarizes the BM/LM clinical studies published between 2010 and 2023.
MEDLINE, CINAHL, CAB Abstracts, PsycINFO, Cochrane Library, HINARI, International Pharmaceutical Abstracts, PubMed, Scopus, Web of Science, and EMBASE electronic databases were searched on June 21, 2021. An updated search was performed on February 21, 2023. Eligible studies investigated a therapeutic intervention in solid tumor patients with BM and/or LM and reported a patient outcome. Extracted study-level data, including study type, publication date, geographical location, number of BM/LM patients in the study, primary tumor type, and type of therapeutic intervention, were collected.
4921 unique studies were eligible for analysis. The key finding is that BM/LM clinical research is expanding globally, both in observational studies and clinical trials. Despite the shift over time toward a higher proportion of systemic therapy trials, the majority still do not include patients with symptomatic disease and lack reporting of BM/LM-specific endpoints. Globally, there has been a trend to more international collaboration in BM/LM clinical studies.
Our analysis of the BM/LM literature charts the evolving landscape of studies involving this previously excluded population. Given the increasing clinical research activity, particularly involving late-stage systemic therapy trials, it is imperative that due consideration is given to the intracranial activity of new investigational agents. Wider adoption of standardized reporting of intracranial-specific endpoints will facilitate the evaluation of relative intracranial efficacy.
最近的研究对脑转移（BM）或软脑膜转移（LM）患者应被排除在全身治疗临床试验之外的观念提出了挑战。这项范围界定研究总结了2010年至2023年间发表的BM/LM临床研究。
于2021年6月21日检索了MEDLINE、CINAHL、CAB文摘数据库、PsycINFO、Cochrane图书馆、HINARI、国际药学文摘数据库、PubMed、Scopus、科学网和EMBASE电子数据库。2023年2月21日进行了更新检索。符合条件的研究调查了对患有BM和/或LM的实体瘤患者的治疗干预，并报告了患者结局。收集了提取的研究层面数据，包括研究类型、发表日期、地理位置、研究中BM/LM患者数量、原发肿瘤类型和治疗干预类型。
4921项独特研究符合分析条件。关键发现是，BM/LM临床研究在全球范围内不断扩展，包括观察性研究和临床试验。尽管随着时间推移，全身治疗试验的比例有所增加，但大多数试验仍未纳入有症状疾病的患者，且缺乏对BM/LM特异性终点的报告。在全球范围内，BM/LM临床研究有更多国际合作的趋势。
我们对BM/LM文献的分析描绘了涉及这一先前被排除人群的研究不断演变的格局。鉴于临床研究活动日益增加，尤其是涉及晚期全身治疗试验，必须充分考虑新研究药物的颅内活性。更广泛地采用颅内特异性终点的标准化报告将有助于评估相对颅内疗效。
