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Financial toxicity in patients with glioblastoma. 胶质母细胞瘤患者的经济毒性。
IF 3.2 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-16 DOI: 10.1007/s11060-024-04835-8
Danielle Terrell, Joseph Camarano, Stephen Whipple, Bharat Guthikonda, Robbie Beyl, W Christopher Newman

Purpose: There has been mounting interest in understanding the impact of financial toxicity (FT) in various cancer types; however, it remains poorly understood and understudied within neuro-oncology-especially as it relates to neurosurgical components of patient care.

Methods: Retrospective, single-center study of patients who underwent craniotomy for resection of glioblastoma from 2020 to 2022. OIBEE™ (Austin, Texas) software was queried to identify the subset of these patients who had a bad debt charged to their account. These patients were deemed to qualify as experiencing FT. Chi Square analysis was conducted between FT and non-FT patient groups. Additionally, survival analyses were performed to determine predictors of progression free and overall survival.

Results: 74 patients were included in this sample. 33/74 (44%) met criteria for FT. The average bad debt amount was $7,476.76 and the median debt amount was $2,015.96, with the average time to financial toxicity after surgery being approximately 127 days. FT patients were significantly younger at diagnosis than those who were not FT (64.6 years- non-FT vs. 59.0 years- FT, p = 0.0344). FT patients were more likely to have undergone subtotal resections rather than a gross total resection compared to non-FT patients (FT GTR 27.3%, non-FT GTR 52.4%, p = 0.028). Hospital length of stay was significantly longer for FT patients compared to non-FT patients (LOS FT 9.5 days, non-FT 6.5 days, p = 0.0312).

Conclusion: Glioblastoma patients are at high risk of experiencing FT with our series showing no significant impact on overall survival. Larger studies are needed to understand the impact of FT on patient outcomes.

目的:人们越来越关注了解财务毒性(FT)对各种癌症类型的影响;然而,在神经肿瘤学领域,人们对财务毒性的了解和研究仍然很少,尤其是与患者护理的神经外科部分有关的财务毒性:对 2020 年至 2022 年期间接受开颅手术切除胶质母细胞瘤的患者进行回顾性单中心研究。通过查询 OIBEE™(德克萨斯州奥斯汀)软件,确定这些患者中哪些人的账户被记入坏账。这些患者被认定为 FT 患者。在 FT 和非 FT 患者组之间进行了 Chi Square 分析。此外,还进行了生存分析,以确定无进展生存期和总生存期的预测因素:74名患者被纳入该样本。33/74(44%)的患者符合 FT 标准。平均坏账金额为 7,476.76 美元,中位数为 2,015.96 美元,术后出现财务毒性的平均时间约为 127 天。与非 FT 患者相比,FT 患者确诊时的年龄明显更小(非 FT 患者 64.6 岁,FT 患者 59.0 岁,P = 0.0344)。与非 FT 患者相比,FT 患者更有可能接受次全切除术,而不是全切除术(FT GTR 27.3%,非 FT GTR 52.4%,p = 0.028)。与非FT患者相比,FT患者的住院时间明显更长(LOS FT 9.5天,非FT 6.5天,p = 0.0312):结论:胶质母细胞瘤患者发生FT的风险很高,但我们的研究结果显示,FT对患者的总生存率没有明显影响。需要进行更大规模的研究,以了解FT对患者预后的影响。
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引用次数: 0
DTI fiber-tracking parameters adjacent to gliomas: the role of tract irregularity value in operative planning, resection, and outcome. 邻近胶质瘤的 DTI 纤维追踪参数:束不规则值在手术规划、切除和预后中的作用。
IF 3.2 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-15 DOI: 10.1007/s11060-024-04848-3
Daniele Armocida, Andrea Bianconi, Giuseppa Zancana, Tingting Jiang, Alessandro Pesce, Fulvio Tartara, Diego Garbossa, Maurizio Salvati, Antonio Santoro, Carlo Serra, Alessandro Frati

Purpose: The goal of glioma surgery is maximal tumor resection associated with minimal post-operative morbidity. Diffusion tensor imaging-tractography/fiber tracking (DTI-FT) is a valuable white-matter (WM) visualization tool for diagnosis and surgical planning. Still, it assumes a descriptive role since the main DTI metrics and parameters showed several limitations in clinical use. New applications and quantitative measurements were recently applied to describe WM architecture that surround the tumor area. The brain adjacent tumor area (BAT) is defined as the region adjacent to the gross tumor volume, which contains signal abnormalities on T2-weighted or FLAIR sequences. The DTI-FT analysis of the BAT can be adopted as predictive values and a guide for safe tumor resection.

Methods: This is an observational prospective study on an extensive series of glioma patients who performed magnetic resonance imaging (MRI) with pre-operative DTI-FT analyzed on the BAT by two different software. We examined DTI parameters of Fractional anisotropy (FA mean, min-max), Mean diffusivity (MD), and the shape-metric "tract irregularity" (TI) grade, comparing it with the surgical series' clinical, radiological, and outcome data.

Results: The population consisted of 118 patients, with a mean age of 60.6 years. 82 patients suffering from high-grade gliomas (69.5%), and 36 from low-grade gliomas (30.5%). A significant inverse relationship exists between the FA mean value and grading (p = 0.001). The relationship appears directly proportional regarding MD values (p = 0.003) and TI values (p = 0.005). FA mean and MD values are susceptible to significant variations with tumor and edema volume (p = 0.05). TI showed an independent relationship with grading regardless of tumor radiological features and dimensions, with a direct relationship with grading, ki67% (p = 0,05), PFS (p < 0.001), and EOR (p < 0.01).

Conclusion: FA, MD, and TI are useful predictive measures of the clinical behavior of glioma, and TI could be helpful for tumor grading identification and surgical planning.

目的:胶质瘤手术的目标是最大限度地切除肿瘤,同时将术后发病率降至最低。弥散张量成像-痕量成像/纤维追踪(DTI-FT)是诊断和手术规划的重要白质(WM)可视化工具。不过,由于主要的 DTI 指标和参数在临床应用中存在一些局限性,因此它仍只能发挥描述性作用。最近,新的应用和定量测量被用于描述肿瘤周围的 WM 结构。脑邻近肿瘤区(BAT)被定义为与肿瘤体积相邻的区域,该区域在 T2 加权或 FLAIR 序列上包含信号异常。对 BAT 的 DTI-FT 分析可作为预测值和安全切除肿瘤的指南:这是一项前瞻性观察研究,研究对象是一系列胶质瘤患者,这些患者均接受了磁共振成像(MRI)检查,术前使用两种不同的软件对 BAT 进行了 DTI-FT 分析。我们检查了各向异性分数(FA 平均值、最小值-最大值)、平均弥散度(MD)和形状度量 "道不规则性"(TI)等级等 DTI 参数,并将其与手术系列的临床、放射学和结果数据进行了比较:研究对象包括 118 名患者,平均年龄为 60.6 岁。82名患者患有高级别胶质瘤(69.5%),36名患者患有低级别胶质瘤(30.5%)。FA 平均值与分级之间存在明显的反比关系(p = 0.001)。MD值(p = 0.003)和TI值(p = 0.005)之间呈正比关系。FA 平均值和 MD 值容易随肿瘤和水肿体积的变化而发生显著变化(p = 0.05)。无论肿瘤的放射学特征和尺寸如何,TI与分级都有独立的关系,与分级、ki67%(p = 0.05)、PFS(p 结论:FA、MD和TI值对肿瘤的分级、ki67%(p = 0.05)和PFS(p = 0.005)有直接关系:FA、MD和TI是预测胶质瘤临床表现的有用指标,TI有助于肿瘤分级鉴定和手术规划。
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引用次数: 0
Social media "SoMe" in neuro-oncology: a review of the literature. 神经肿瘤学中的社交媒体 "SoMe":文献综述。
IF 3.2 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-14 DOI: 10.1007/s11060-024-04845-6
Benjamin R Klein, David J Levi, Ashish H Shah, Michael E Ivan, Allan D Levi

Purpose: This article examines the current state of social media (SoMe) in neuro-oncology and neurosurgical oncology. The goal of this paper is to provide thorough discourse regarding benefits and disadvantages of being a neurosurgical oncologist on SoMe, while discussing the place SoMe will have in cranial tumor-based practices going forward.

Methods: The author's performed a rigorous literature review on the topic. Included information was pertinent to the history of SoMe in neurosurgical oncology and its impact on the field of neuro-oncology. Incorporated as well are the benefits of being a neurosurgical oncologist on SoMe, the drawbacks of participation on SoMe platforms, and knowledge that facilitates discussion about the future of SoMe in neurosurgical oncology.

Results: SoMe plays an important role in neuro-oncology and neurosurgical oncology. SoMe continues to exponentially grow in the healthcare sphere as more providers utilize SoMe platforms. We report objective negative and positive outcomes of SoMe in neurosurgical oncology and neuro-oncology. Here, we summarize these results and provide dialogue describing the effect SoMe is having on the many different aspects of neurosurgical oncology and neuro-oncology.

Conclusion: Although SoMe platforms improve social presence and patient outreach, the use of SoMe can also adversely affect one's career by exposing clinicians to unchecked societal, legal and professional consequences. While using SoMe as a vessel to propagate career initiatives, neurosurgical oncologists should exercise caution with the content they choose to circulate.

目的:本文探讨了社交媒体(SoMe)在神经肿瘤学和神经外科肿瘤学中的应用现状。本文旨在深入探讨作为神经外科肿瘤学家在 SoMe 上的利弊,同时讨论 SoMe 在颅脑肿瘤实践中的未来地位:作者对该主题进行了严格的文献综述。其中包括有关 SoMe 在神经肿瘤外科的历史及其对神经肿瘤学领域的影响的信息。还包括作为神经外科肿瘤学家在SoMe平台上的益处、参与SoMe平台的弊端,以及有助于讨论SoMe在神经外科肿瘤学领域的未来的知识:SoMe在神经肿瘤学和神经外科肿瘤学中发挥着重要作用。随着越来越多的医疗服务提供者使用 SoMe 平台,SoMe 在医疗保健领域继续呈指数级增长。我们报告了 SoMe 在神经肿瘤外科和神经肿瘤学中的客观负面和正面结果。在此,我们总结了这些结果,并提供了对话,描述了SoMe对神经外科肿瘤学和神经肿瘤学许多不同方面的影响:尽管 SoMe 平台提高了社交影响力和患者外展能力,但使用 SoMe 也会使临床医生面临无法控制的社会、法律和专业后果,从而对其职业生涯产生不利影响。神经外科肿瘤学家在使用 SoMe 作为传播职业倡议的渠道时,应谨慎选择传播的内容。
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引用次数: 0
Brain volume loss after cranial irradiation: a controlled comparison study between photon vs proton radiotherapy for WHO grade 2-3 gliomas. 颅脑照射后脑容量损失:针对WHO 2-3级胶质瘤的光子与质子放疗对照比较研究。
IF 3.2 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-14 DOI: 10.1007/s11060-024-04850-9
Melissa M Gardner, Sebastian F Winter, Franziska Stahl, Elizabeth R Gerstner, Helen A Shih, Janet Sherman, Jorg Dietrich, Michael W Parsons

Purpose: Radiation therapy (RT) is an integral treatment component in patients with glioma but associated with neurotoxicity. Proton RT (PRT), as compared with photon RT (XRT), reduces excess radiation to nontarget tissue. We used a retrospective method to evaluate brain imaging metrics of neurotoxicity after treatment with PRT and XRT for glioma.

Methods: We analyzed brain volume change in thirty-four patients with WHO grade 2-3 gliomas treated with either PRT (n = 17) or XRT (n = 17). Both groups were carefully matched by demographic/clinical criteria and assessed longitudinally for two years post-radiotherapy. Brain volume change was measured as ventricular volume expansion in the tumor free hemisphere (contralateral to RT target) as a proxy indicator of brain volume loss. We further assessed the impact of volumetric changes on cognition in PRT patients, who completed neuropsychological testing as part of an outcome study.

Results: We found significant ventricular volume increases in the contralesional hemisphere in both groups at two years post-RT (F(1, 31) = 18.45, p < 0.000, partial η2 = 0.373), with greater volume change observed in XRT (26.55%) vs. PRT (12.03%) (M = 12.03%, SD = 16.26; F(1,31) = 4.26, p = 0.048, partial η2 = 0.121). Although, there was no group-level change on any cognitive test in PRT treated patients, individual changes on cognitive screening, working memory, processing speed and visual memory tasks correlated with contralesional brain volume loss.

Conclusion: This study suggests progressive brain volume loss following cranial irradiation, with greater severity after XRT vs. PRT. Radiation-induced brain volume loss appears to be associated with measurable cognitive changes on an individual level. Prospective studies are warranted to validate these findings and their impacts on long-term cognitive function and quality of life. An improved understanding of the structural and functional consequences of cranial radiation is essential to develop neuroprotective strategies.

目的:放射治疗(RT)是胶质瘤患者不可或缺的治疗手段,但也会产生神经毒性。与光子放射治疗(XRT)相比,质子放射治疗(PRT)可减少对非靶组织的过量辐射。我们采用了一种回顾性方法来评估脑胶质瘤PRT和XRT治疗后神经毒性的脑成像指标:我们分析了 34 名接受 PRT(17 人)或 XRT(17 人)治疗的 WHO 2-3 级胶质瘤患者的脑容量变化。两组患者均按照人口统计学/临床标准进行了仔细配对,并在放疗后两年内进行了纵向评估。脑容量变化以无肿瘤半球(RT靶点的对侧)脑室容量的扩大作为脑容量损失的替代指标。我们进一步评估了体积变化对 PRT 患者认知能力的影响,这些患者完成了神经心理学测试,作为结果研究的一部分:结果:我们发现,RT 术后两年时,两组患者对侧大脑半球的脑室容积均有明显增加(F(1,31)= 18.45,P 结论:PRT 术后两年,两组患者对侧大脑半球的脑室容积均有明显增加:这项研究表明,颅脑照射后脑容量会逐渐减少,XRT 与 PRT 相比更为严重。辐射引起的脑容量损失似乎与个体水平上可测量的认知变化有关。需要进行前瞻性研究来验证这些发现及其对长期认知功能和生活质量的影响。进一步了解颅脑辐射的结构和功能后果对于制定神经保护策略至关重要。
{"title":"Brain volume loss after cranial irradiation: a controlled comparison study between photon vs proton radiotherapy for WHO grade 2-3 gliomas.","authors":"Melissa M Gardner, Sebastian F Winter, Franziska Stahl, Elizabeth R Gerstner, Helen A Shih, Janet Sherman, Jorg Dietrich, Michael W Parsons","doi":"10.1007/s11060-024-04850-9","DOIUrl":"https://doi.org/10.1007/s11060-024-04850-9","url":null,"abstract":"<p><strong>Purpose: </strong>Radiation therapy (RT) is an integral treatment component in patients with glioma but associated with neurotoxicity. Proton RT (PRT), as compared with photon RT (XRT), reduces excess radiation to nontarget tissue. We used a retrospective method to evaluate brain imaging metrics of neurotoxicity after treatment with PRT and XRT for glioma.</p><p><strong>Methods: </strong>We analyzed brain volume change in thirty-four patients with WHO grade 2-3 gliomas treated with either PRT (n = 17) or XRT (n = 17). Both groups were carefully matched by demographic/clinical criteria and assessed longitudinally for two years post-radiotherapy. Brain volume change was measured as ventricular volume expansion in the tumor free hemisphere (contralateral to RT target) as a proxy indicator of brain volume loss. We further assessed the impact of volumetric changes on cognition in PRT patients, who completed neuropsychological testing as part of an outcome study.</p><p><strong>Results: </strong>We found significant ventricular volume increases in the contralesional hemisphere in both groups at two years post-RT (F(1, 31) = 18.45, p < 0.000, partial η2 = 0.373), with greater volume change observed in XRT (26.55%) vs. PRT (12.03%) (M = 12.03%, SD = 16.26; F(1,31) = 4.26, p = 0.048, partial η2 = 0.121). Although, there was no group-level change on any cognitive test in PRT treated patients, individual changes on cognitive screening, working memory, processing speed and visual memory tasks correlated with contralesional brain volume loss.</p><p><strong>Conclusion: </strong>This study suggests progressive brain volume loss following cranial irradiation, with greater severity after XRT vs. PRT. Radiation-induced brain volume loss appears to be associated with measurable cognitive changes on an individual level. Prospective studies are warranted to validate these findings and their impacts on long-term cognitive function and quality of life. An improved understanding of the structural and functional consequences of cranial radiation is essential to develop neuroprotective strategies.</p>","PeriodicalId":16425,"journal":{"name":"Journal of Neuro-Oncology","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2024-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142467910","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Congress of Neurological Surgeons systematic review and evidence‑based guidelines on the management of recurrent diffuse low-grade glioma: update. 神经外科医师大会关于复发性弥漫性低级别胶质瘤治疗的系统回顾和循证指南:更新版。
IF 3.2 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-14 DOI: 10.1007/s11060-024-04838-5
Kevin Morrow, Andrew Sloan, Jeffrey J Olson, D Ryan Ormond
<p><p>Target population These recommendations apply to adult patients with recurrent WHO grade 2 infiltrative diffuse glioma (oligodendroglioma, astrocytoma).Questions and Recommendations:Imaging Q1: In adult patients with suspected recurrence of histologically proven WHO grade 2 diffuse glioma, do advanced imaging techniques using magnetic resonance spectroscopy, perfusion weighted imaging, diffusion weighted imaging or PET provide superior assessment of tumor recurrence and histologic progression compared to standard MRI neuroimaging?Recommendation Level III: In adult patients with suspected recurrence of histologically proven WHO grade 2 diffuse glioma, advanced imaging techniques using magnetic resonance spectroscopy, perfusion weighted imaging, diffusion weighted imaging or PET are suggested for identification of tumor recurrence or histologic progression.Pathology Q1: In adult patients with suspected recurrence of histologically proven WHO grade 2 diffuse glioma, is molecular testing for IDH-1, IDH-2, and TP53 Mutations and MGMT promotor methylation mutation warranted for predicting survival and formulating treatment recommendations?Recommendation Level III: It is suggested that IDH mutation status be determined for diagnostic purposes. TP53 mutations occur early in WHO grade 2 diffuse glioma pathogenesis, remain stable, and are not suggested as a marker of predisposition to malignant transformation at recurrence or other measures of prognosis. Assessment of MGMT status is suggested as an adjunct to assessing prognosis. Assessment of CDK2NA status is suggested since this is associated with malignant progression of WHO grade 2 diffuse gliomas.Q2: In adult patients with suspected recurrence of histologically proven WHO Grade 2 diffuse glioma, is testing of proliferation indices (MIB-1 and/or BUdR) warranted for predicting survival and formulating treatment recommendations?Recommendation Level III: It is suggested that proliferative indices (MIB-1 or BUdR) be measured in WHO grade 2 diffuse glioma as higher proliferation indices are associated with increased likelihood of recurrence and shorter progression free and overall survival.Chemotherapy Q1: In adult patients with suspected recurrence of histologically proven WHO grade 2 diffuse glioma, does addition of temozolomide (TMZ), other cytotoxic agents or targeted agents to their treatment regimen improve PFS and/or OS?Recommendation Level III: Temozolomide is suggested in the therapy of recurrent WHO grade 2 diffuse glioma as it may improve clinical symptoms. PCV is suggested in the therapy of WHO grade 2 diffuse glioma at recurrence as it may improve clinical symptoms with the strongest evidence being for oligodendrogliomas. TMZ is suggested as the initial choice for recurrent WHO grade 2 diffuse glioma. Carboplatin is not suggested as there is no significant benefit from carboplatin as single agent therapy for recurrent WHO grade 2 diffuse gliomas. There is insufficient evidence to make any
这些建议适用于复发的WHO 2级浸润性弥漫性胶质瘤(少突胶质细胞瘤、星形细胞瘤)的成人患者。问题与建议:影像学问题1:对于组织学证实的WHO 2级弥漫性胶质瘤疑似复发的成人患者,与标准磁共振神经影像学相比,使用磁共振波谱、灌注加权成像、弥散加权成像或正电子发射计算机断层显像的高级影像学技术是否能提供更好的肿瘤复发和组织学进展评估?建议 III 级:对于组织学证实为 WHO 2 级弥漫性胶质瘤疑似复发的成人患者,建议使用磁共振波谱成像、灌注加权成像、弥散加权成像或 PET 等高级成像技术来鉴别肿瘤复发或组织学进展。病理问题 1:对于组织学证实的 WHO 2 级弥漫性胶质瘤疑似复发的成人患者,是否有必要进行 IDH-1、IDH-2 和 TP53 突变以及 MGMT 启动子甲基化突变的分子检测,以预测生存率并制定治疗建议?建议 III 级:建议为诊断目的确定 IDH 突变状态。TP53突变发生在WHO 2级弥漫性胶质瘤发病的早期,并保持稳定,不建议将其作为复发时恶性转化倾向的标志或其他预后指标。建议将 MGMT 状态评估作为评估预后的辅助手段。问题2:对于组织学证实为WHO 2级弥漫性胶质瘤的疑似复发成人患者,是否有必要检测增殖指数(MIB-1和/或BUdR)以预测生存率并制定治疗建议?建议 III 级:建议对 WHO 2 级弥漫性胶质瘤检测增殖指数(MIB-1 或 BUdR),因为增殖指数越高,复发的可能性越大,无进展生存期和总生存期越短。化疗问题1:对于组织学证实的WHO 2级弥漫性胶质瘤疑似复发的成人患者,在治疗方案中添加替莫唑胺(TMZ)、其他细胞毒性药物或靶向药物是否能改善PFS和/或OS?建议等级III:建议在治疗复发的WHO 2级弥漫性胶质瘤时使用替莫唑胺,因为它可以改善临床症状。建议将 PCV 用于治疗复发的 WHO 2 级弥漫性胶质瘤,因为它可以改善临床症状,其中证据最充分的是少突胶质瘤。建议将 TMZ 作为复发的 WHO 2 级弥漫性胶质瘤的初始选择。不建议使用卡铂,因为卡铂作为复发性WHO 2级弥漫性胶质瘤的单药治疗并无明显疗效。放疗问题1:对于组织学证实为WHO 2级弥漫性胶质瘤的疑似复发成人患者,在治疗方案中加入放疗是否能改善PFS和/或OS?建议等级III:如果既往未接受过放疗,建议在复发时进行放疗。问题2:对于既往接受过放疗、组织学证实为WHO 2级弥漫性胶质瘤疑似复发的成人患者,在治疗方案中增加再次放疗或质子治疗是否能改善PFS和/或OS?建议等级III:建议在WHO 2级弥漫性胶质瘤复发的情况下考虑再次放疗,因为它可能会为PFS和OS带来益处。 问题1:对于组织学证实为WHO 2级弥漫性胶质瘤疑似复发的成人患者,手术切除是否能改善PFS和/或OS?目前尚无足够证据就手术治疗的价值或切除范围与复发的WHO 2级弥漫性胶质瘤生存率的关系提出新的具体建议。
{"title":"Congress of Neurological Surgeons systematic review and evidence‑based guidelines on the management of recurrent diffuse low-grade glioma: update.","authors":"Kevin Morrow, Andrew Sloan, Jeffrey J Olson, D Ryan Ormond","doi":"10.1007/s11060-024-04838-5","DOIUrl":"https://doi.org/10.1007/s11060-024-04838-5","url":null,"abstract":"&lt;p&gt;&lt;p&gt;Target population These recommendations apply to adult patients with recurrent WHO grade 2 infiltrative diffuse glioma (oligodendroglioma, astrocytoma).Questions and Recommendations:Imaging Q1: In adult patients with suspected recurrence of histologically proven WHO grade 2 diffuse glioma, do advanced imaging techniques using magnetic resonance spectroscopy, perfusion weighted imaging, diffusion weighted imaging or PET provide superior assessment of tumor recurrence and histologic progression compared to standard MRI neuroimaging?Recommendation Level III: In adult patients with suspected recurrence of histologically proven WHO grade 2 diffuse glioma, advanced imaging techniques using magnetic resonance spectroscopy, perfusion weighted imaging, diffusion weighted imaging or PET are suggested for identification of tumor recurrence or histologic progression.Pathology Q1: In adult patients with suspected recurrence of histologically proven WHO grade 2 diffuse glioma, is molecular testing for IDH-1, IDH-2, and TP53 Mutations and MGMT promotor methylation mutation warranted for predicting survival and formulating treatment recommendations?Recommendation Level III: It is suggested that IDH mutation status be determined for diagnostic purposes. TP53 mutations occur early in WHO grade 2 diffuse glioma pathogenesis, remain stable, and are not suggested as a marker of predisposition to malignant transformation at recurrence or other measures of prognosis. Assessment of MGMT status is suggested as an adjunct to assessing prognosis. Assessment of CDK2NA status is suggested since this is associated with malignant progression of WHO grade 2 diffuse gliomas.Q2: In adult patients with suspected recurrence of histologically proven WHO Grade 2 diffuse glioma, is testing of proliferation indices (MIB-1 and/or BUdR) warranted for predicting survival and formulating treatment recommendations?Recommendation Level III: It is suggested that proliferative indices (MIB-1 or BUdR) be measured in WHO grade 2 diffuse glioma as higher proliferation indices are associated with increased likelihood of recurrence and shorter progression free and overall survival.Chemotherapy Q1: In adult patients with suspected recurrence of histologically proven WHO grade 2 diffuse glioma, does addition of temozolomide (TMZ), other cytotoxic agents or targeted agents to their treatment regimen improve PFS and/or OS?Recommendation Level III: Temozolomide is suggested in the therapy of recurrent WHO grade 2 diffuse glioma as it may improve clinical symptoms. PCV is suggested in the therapy of WHO grade 2 diffuse glioma at recurrence as it may improve clinical symptoms with the strongest evidence being for oligodendrogliomas. TMZ is suggested as the initial choice for recurrent WHO grade 2 diffuse glioma. Carboplatin is not suggested as there is no significant benefit from carboplatin as single agent therapy for recurrent WHO grade 2 diffuse gliomas. There is insufficient evidence to make any","PeriodicalId":16425,"journal":{"name":"Journal of Neuro-Oncology","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2024-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142467912","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Development and application of explainable artificial intelligence using machine learning classification for long-term facial nerve function after vestibular schwannoma surgery. 针对前庭分裂瘤手术后的长期面神经功能,开发和应用利用机器学习分类的可解释人工智能。
IF 3.2 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-11 DOI: 10.1007/s11060-024-04844-7
Lukasz Przepiorka, Sławomir Kujawski, Katarzyna Wójtowicz, Edyta Maj, Andrzej Marchel, Przemysław Kunert

Purpose: Vestibular schwannomas (VSs) represent the most common cerebellopontine angle tumors, posing a challenge in preserving facial nerve (FN) function during surgery. We employed the Extreme Gradient Boosting machine learning classifier to predict long-term FN outcomes (classified as House-Brackmann grades 1-2 for good outcomes and 3-6 for bad outcomes) after VS surgery.

Methods: In a retrospective analysis of 256 patients, comprehensive pre-, intra-, and post-operative factors were examined. We applied the machine learning (ML) classifier Extreme Gradient Boosting (XGBoost) for the following binary classification: long-term good and bad FN outcome after VS surgery To enhance the interpretability of our model, we utilized an explainable artificial intelligence approach.

Results: Short-term FN function (tau = 0.6) correlated with long-term FN function. The model exhibited an average accuracy of 0.83, a ROC AUC score of 0.91, and Matthew's correlation coefficient score of 0.62. The most influential feature, identified through SHapley Additive exPlanations (SHAP), was short-term FN function. Conversely, large tumor volume and absence of preoperative auditory brainstem responses were associated with unfavorable outcomes.

Conclusions: We introduce an effective ML model for classifying long-term FN outcomes following VS surgery. Short-term FN function was identified as the key predictor of long-term function. This model's excellent ability to differentiate bad and good outcomes makes it useful for evaluating patients and providing recommendations regarding FN dysfunction management.

目的:前庭分裂瘤(VS)是最常见的小脑角肿瘤,在手术过程中保护面神经(FN)功能是一项挑战。我们采用了极端梯度提升机器学习分类器来预测VS手术后面神经的长期预后(House-Brackmann分级1-2级预后好,3-6级预后差):在对 256 例患者进行的回顾性分析中,对术前、术中和术后的综合因素进行了研究。为了提高模型的可解释性,我们采用了一种可解释的人工智能方法:结果:短期 FN 功能(tau = 0.6)与长期 FN 功能相关。该模型的平均准确率为 0.83,ROC AUC 得分为 0.91,马修相关系数为 0.62。通过 SHapley Additive exPlanations(SHAP)确定的最有影响的特征是短期 FN 功能。相反,肿瘤体积大和术前无听性脑干反应与不利的结果有关:结论:我们引入了一个有效的 ML 模型,用于对 VS 手术后的长期 FN 结果进行分类。短期 FN 功能是预测长期功能的关键因素。该模型区分不良和良好结果的能力极强,因此可用于评估患者和提供有关 FN 功能障碍管理的建议。
{"title":"Development and application of explainable artificial intelligence using machine learning classification for long-term facial nerve function after vestibular schwannoma surgery.","authors":"Lukasz Przepiorka, Sławomir Kujawski, Katarzyna Wójtowicz, Edyta Maj, Andrzej Marchel, Przemysław Kunert","doi":"10.1007/s11060-024-04844-7","DOIUrl":"https://doi.org/10.1007/s11060-024-04844-7","url":null,"abstract":"<p><strong>Purpose: </strong>Vestibular schwannomas (VSs) represent the most common cerebellopontine angle tumors, posing a challenge in preserving facial nerve (FN) function during surgery. We employed the Extreme Gradient Boosting machine learning classifier to predict long-term FN outcomes (classified as House-Brackmann grades 1-2 for good outcomes and 3-6 for bad outcomes) after VS surgery.</p><p><strong>Methods: </strong>In a retrospective analysis of 256 patients, comprehensive pre-, intra-, and post-operative factors were examined. We applied the machine learning (ML) classifier Extreme Gradient Boosting (XGBoost) for the following binary classification: long-term good and bad FN outcome after VS surgery To enhance the interpretability of our model, we utilized an explainable artificial intelligence approach.</p><p><strong>Results: </strong>Short-term FN function (tau = 0.6) correlated with long-term FN function. The model exhibited an average accuracy of 0.83, a ROC AUC score of 0.91, and Matthew's correlation coefficient score of 0.62. The most influential feature, identified through SHapley Additive exPlanations (SHAP), was short-term FN function. Conversely, large tumor volume and absence of preoperative auditory brainstem responses were associated with unfavorable outcomes.</p><p><strong>Conclusions: </strong>We introduce an effective ML model for classifying long-term FN outcomes following VS surgery. Short-term FN function was identified as the key predictor of long-term function. This model's excellent ability to differentiate bad and good outcomes makes it useful for evaluating patients and providing recommendations regarding FN dysfunction management.</p>","PeriodicalId":16425,"journal":{"name":"Journal of Neuro-Oncology","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2024-10-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142400438","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Advanced imaging reveals enhanced malignancy in glioblastomas involving the subventricular zone: evidence of increased infiltrative growth and perfusion. 高级成像显示脑室下区胶质母细胞瘤的恶性程度增强:浸润性生长和灌注增加的证据。
IF 3.2 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-10 DOI: 10.1007/s11060-024-04849-2
Michael Griessmair, Severin Schramm, Julian Ziegenfeuter, Julian Canisius, Kirsten Jung, Claire Delbridge, Friederike Schmidt-Graf, Meike Mitsdoerffer, Claus Zimmer, Bernhard Meyer, Marie-Christin Metz, Benedikt Wiestler

Background: Glioblastoma's infiltrative growth and heterogeneity are influenced by neural, molecular, genetic, and immunological factors, with the precise origin of these tumors remaining elusive. Neurogenic zones might serve as the tumor stem cells' nest, with tumors in contact with these zones exhibiting worse outcomes and more aggressive growth patterns. This study aimed to determine if these characteristics are reflected in advanced imaging, specifically diffusion and perfusion data.

Methods: In this monocentric retrospective study, 137 glioblastoma therapy-naive patients (IDH-wildtype, grade 4) with advanced preoperative MRI, including perfusion and diffusion imaging, were analyzed. Tumors and neurogenic zones were automatically segmented. Advanced imaging metrics, including cerebral blood volume (CBV) from perfusion imaging, tissue volume mask (TVM), and free water corrected fractional anisotropy (FA-FWE) from diffusion imaging, were extracted.

Results: SVZ infiltration positively correlated with CBV, indicating higher perfusion in tumors. Significant CBV differences were noted between high and low SVZ infiltration cases at specific percentiles. Negative correlation was observed with TVM and positive correlation with FA-FWE, suggesting more infiltrative tumor growth. Significant differences in TVM and FA-FWE values were found between high and low SVZ infiltration cases.

Discussion: Glioblastomas with SVZ infiltration exhibit distinct imaging characteristics, including higher perfusion and lower cell density per voxel, indicating a more infiltrative growth and higher vascularization. Stem cell-like characteristics in SVZ-infiltrating cells could explain the increased infiltration and aggressive behavior. Understanding these imaging and biological correlations could enhance the understanding of glioblastoma evolution.

背景:胶质母细胞瘤的浸润性生长和异质性受神经、分子、遗传和免疫因素的影响,而这些肿瘤的确切起源仍难以捉摸。神经源区可能是肿瘤干细胞的巢穴,与这些区域接触的肿瘤会表现出更差的预后和更具侵袭性的生长模式。本研究旨在确定这些特征是否反映在高级成像中,特别是弥散和灌注数据中:在这项单中心回顾性研究中,分析了137名未接受治疗的胶质母细胞瘤患者(IDH-野生型,4级)的术前高级磁共振成像,包括灌注和弥散成像。对肿瘤和神经源区进行了自动分割。提取了先进的成像指标,包括灌注成像的脑血容量(CBV)、组织容积掩膜(TVM)和弥散成像的自由水校正分数各向异性(FA-FWE):结果:SVZ浸润与CBV呈正相关,表明肿瘤的灌注较高。在特定百分位数上,高SVZ浸润和低SVZ浸润病例的CBV差异显著。TVM与CBV呈负相关,FA-FWE与CBV呈正相关,表明肿瘤的浸润性生长更强。高SVZ浸润和低SVZ浸润病例的TVM和FA-FWE值存在显著差异:讨论:有SVZ浸润的胶质母细胞瘤表现出独特的成像特征,包括较高的灌注和较低的每体素细胞密度,表明肿瘤的浸润性生长和血管化程度较高。SVZ浸润细胞的干细胞样特征可以解释浸润增加和侵袭行为。了解这些成像和生物学相关性有助于加深对胶质母细胞瘤演变的理解。
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引用次数: 0
In-patient neurosurgical tumor treatments for malignant glioma patients in Germany. 德国恶性胶质瘤患者的神经外科肿瘤住院治疗。
IF 3.2 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-10 DOI: 10.1007/s11060-024-04784-2
Marcel A Kamp, Larissa Fink, Marie-Therese Forster, Carolin Weiss Lucas, Aaron Lawson McLean, Anna Lawson McLean, Christian Freyschlag, Klaus-Peter Stein, Dorothee Wiewrodt, Felix Muehlensiepen, Florian H Ebner, Marion Rapp, Niklas Thon, Michael Sabel, Nazife Dinc, Christiane von Saß, Marco Stein, Christine Jungk

Objective: Treatment for malignant gliomas involves multiple disciplines, including neurosurgery, radiation therapy, medical and neuro-oncology, and palliative medicine, with function-preserving neurosurgical tumor removal being crucial. However, real-world data on hospital cases, treatment types, especially regarding surgical approaches, and the associated complication and mortality rates in Germany are lacking.

Methods: We analyzed data on hospital cases involving malignant gliomas (ICD-10-GM code C71) from the German §21 Hospital Remuneration Act, provided by the Institute for the Hospital Remuneration System (InEK GmbH), from 2019 to 2022. Our focus was on neuro-oncological operations defined by the German Cancer Society (DKG) and included specific operation and procedure (OPS) codes.

Results: From 2019 to 2022, there were 101,192 hospital cases involving malignant gliomas in Germany. Neurosurgical tumor removal was performed in 27,193 cases (26.9%). Microsurgical techniques were used in 95% of surgeries, intraoperative navigation systems in 84%, fluorescence-guided surgeries in 45.6%, and intraoperative neurophysiological monitoring (IONM) in 46.4%. Surgical or medical complications occurred in 2903 cases (10.7%). The hospital mortality rate was 2.7%. Mortality was significantly higher in patients aged 65 and older (Odds ratio 2.9, p < 0.0001), and lower in cases using fluorescence-guided procedures (Odds ratio 0.8, p = 0.015) and IONM (Odds ratio 0.5, p < 0.0001).

Conclusions: Over the course of 4 years, over 100,000 hospital cases involving adult patients diagnosed with malignant gliomas were treated in Germany, with 27,193 cases undergoing tumor removal using various modern surgical techniques. The hospital mortality rate was 2.7%.

目的:恶性胶质瘤的治疗涉及多个学科,包括神经外科、放射治疗、内科和神经肿瘤科以及姑息医学,其中保留功能的神经外科肿瘤切除术至关重要。然而,德国缺乏有关医院病例、治疗类型(尤其是手术方法)以及相关并发症和死亡率的真实数据:我们分析了德国医院薪酬系统研究所(InEK GmbH)提供的2019年至2022年德国§21医院薪酬法案中涉及恶性胶质瘤(ICD-10-GM代码C71)的住院病例数据。我们的研究重点是德国癌症协会(DKG)定义的神经肿瘤手术,包括具体的手术和程序(OPS)代码:从 2019 年到 2022 年,德国共有 101,192 例涉及恶性胶质瘤的住院病例。其中27193例(26.9%)进行了神经外科肿瘤切除术。95%的手术使用了显微外科技术,84%的手术使用了术中导航系统,45.6%的手术使用了荧光引导手术,46.4%的手术使用了术中神经电生理监测(IONM)。2903例(10.7%)发生了手术或医疗并发症。住院死亡率为 2.7%。65 岁及以上患者的死亡率明显较高(Odds ratio 2.9,P 结论):在4年的时间里,德国医院共治疗了10万多例确诊为恶性胶质瘤的成年患者,其中27193例患者接受了各种现代外科技术的肿瘤切除术。住院死亡率为 2.7%。
{"title":"In-patient neurosurgical tumor treatments for malignant glioma patients in Germany.","authors":"Marcel A Kamp, Larissa Fink, Marie-Therese Forster, Carolin Weiss Lucas, Aaron Lawson McLean, Anna Lawson McLean, Christian Freyschlag, Klaus-Peter Stein, Dorothee Wiewrodt, Felix Muehlensiepen, Florian H Ebner, Marion Rapp, Niklas Thon, Michael Sabel, Nazife Dinc, Christiane von Saß, Marco Stein, Christine Jungk","doi":"10.1007/s11060-024-04784-2","DOIUrl":"https://doi.org/10.1007/s11060-024-04784-2","url":null,"abstract":"<p><strong>Objective: </strong>Treatment for malignant gliomas involves multiple disciplines, including neurosurgery, radiation therapy, medical and neuro-oncology, and palliative medicine, with function-preserving neurosurgical tumor removal being crucial. However, real-world data on hospital cases, treatment types, especially regarding surgical approaches, and the associated complication and mortality rates in Germany are lacking.</p><p><strong>Methods: </strong>We analyzed data on hospital cases involving malignant gliomas (ICD-10-GM code C71) from the German §21 Hospital Remuneration Act, provided by the Institute for the Hospital Remuneration System (InEK GmbH), from 2019 to 2022. Our focus was on neuro-oncological operations defined by the German Cancer Society (DKG) and included specific operation and procedure (OPS) codes.</p><p><strong>Results: </strong>From 2019 to 2022, there were 101,192 hospital cases involving malignant gliomas in Germany. Neurosurgical tumor removal was performed in 27,193 cases (26.9%). Microsurgical techniques were used in 95% of surgeries, intraoperative navigation systems in 84%, fluorescence-guided surgeries in 45.6%, and intraoperative neurophysiological monitoring (IONM) in 46.4%. Surgical or medical complications occurred in 2903 cases (10.7%). The hospital mortality rate was 2.7%. Mortality was significantly higher in patients aged 65 and older (Odds ratio 2.9, p < 0.0001), and lower in cases using fluorescence-guided procedures (Odds ratio 0.8, p = 0.015) and IONM (Odds ratio 0.5, p < 0.0001).</p><p><strong>Conclusions: </strong>Over the course of 4 years, over 100,000 hospital cases involving adult patients diagnosed with malignant gliomas were treated in Germany, with 27,193 cases undergoing tumor removal using various modern surgical techniques. The hospital mortality rate was 2.7%.</p>","PeriodicalId":16425,"journal":{"name":"Journal of Neuro-Oncology","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2024-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142400439","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Impact of upfront adjuvant chemoradiation on survival in patients with molecularly defined oligodendroglioma: the benefits of PCV over TMZ. 前期辅助化疗对分子定义少突胶质细胞瘤患者生存期的影响:PCV比TMZ更有优势。
IF 3.2 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-09 DOI: 10.1007/s11060-024-04829-6
Jordina Rincon-Torroella, Maureen Rakovec, Anita L Kalluri, Kelly Jiang, Carly Weber-Levine, Megan Parker, Divyaansh Raj, Josh Materi, Sadra Sepehri, Abel Ferres, Karisa C Schreck, Iban Aldecoa, Calixto-Hope G Lucas, Haris I Sair, Kristin J Redmond, Matthias Holdhoff, Jon Weingart, Henry Brem, Josep J González Sánchez, Xiaobu Ye, Chetan Bettegowda

Purpose: Oligodendroglioma is an adult-type diffuse glioma defined by 1p/19q codeletion and IDH1/2 mutation. Treatment includes surgery followed by observation alone in select low-grade tumors, or combination radiation and chemotherapy with procarbazine, lomustine, and vincristine (PCV) or temozolomide (TMZ). While prospective studies investigating treatments for molecularly defined oligodendrogliomas are ongoing, this retrospective study analyzes the relationship between adjuvant regimens and progression-free survival (PFS).

Methods: Adults with IDH-mutant, 1p/19q codeleted oligodendroglioma (WHO grade 2 or 3) who underwent surgery between 2005 and 2021 were identified. Clinical data, disease characteristics, treatment, and outcomes were collected.

Results: A total of 207 patients with grade 2 and 70 with grade 3 oligodendrogliomas were identified. Median (IQR) follow-up was 57 (87) months. Patients with grade 3 tumors who received adjuvant radiation and PCV had longer median PFS (> 110 months) than patients who received radiation and TMZ (52 months, p = 0.008) or no adjuvant chemoradiation (83 months, p = 0.03), which was not seen in grade 2 tumors (p = 0.8). In multivariate analysis, patients who received PCV chemotherapy (Relative Risk [95% CI] = 0.24[0.05-1.08] and radiotherapy (0.46[0.21-1.02]) trended towards longer PFS, independently of grade.

Conclusion: Adjuvant radiation and PCV are associated with improved PFS over radiation with TMZ in patients with grade 3 molecularly defined oligodendrogliomas, and all-grade patients treated with PCV trended towards decreased risk of recurrence and progression. These results highlight the importance of ongoing clinical trials investigating these treatments.

目的:少突胶质细胞瘤是一种成人型弥漫性胶质瘤,定义为1p/19q编码缺失和IDH1/2突变。治疗方法包括对部分低级别肿瘤进行手术,然后进行单纯观察,或联合放疗和丙卡巴嗪、洛莫司汀和长春新碱(PCV)或替莫唑胺(TMZ)化疗。尽管针对分子定义的少突胶质瘤治疗方法的前瞻性研究正在进行中,但这项回顾性研究分析了辅助治疗方案与无进展生存期(PFS)之间的关系:方法:对2005年至2021年期间接受手术治疗的IDH突变、1p/19q编码缺失的少突胶质细胞瘤(WHO 2级或3级)成人患者进行鉴定。收集了临床数据、疾病特征、治疗方法和结果:结果:共发现207例2级和70例3级少突胶质细胞瘤患者。随访中位数(IQR)为57(87)个月。与接受放射治疗和TMZ(52个月,P = 0.008)或未接受辅助化疗(83个月,P = 0.03)的患者相比,接受放射治疗和PCV辅助治疗的3级肿瘤患者的中位PFS(大于110个月)更长,而2级肿瘤患者的中位PFS则不长(P = 0.8)。在多变量分析中,接受PCV化疗(相对风险[95% CI] = 0.24[0.05-1.08])和放疗(0.46[0.21-1.02])的患者有延长PFS的趋势,与分级无关:结论:对于分子定义为3级的少突胶质细胞瘤患者,与TMZ放疗相比,辅助放疗和PCV可改善患者的PFS,而接受PCV治疗的所有级别患者的复发和进展风险均呈下降趋势。这些结果凸显了正在进行的研究这些治疗方法的临床试验的重要性。
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引用次数: 0
Clinical trial design for novel targeted agents in neuro-oncology. 神经肿瘤学新型靶向药物的临床试验设计。
IF 3.2 2区 医学 Q2 CLINICAL NEUROLOGY Pub Date : 2024-10-08 DOI: 10.1007/s11060-024-04846-5
Mary Jane Lim-Fat, Katrina Roberto, Patrick Y Wen

Biomarker-based clinical trials investigating targeted treatments for brain tumors have surged due to better access to biomarker testing and a deeper grasp of the molecular basis of tumor development. The design of clinical trials is crucial for evaluating safety, confirming effectiveness, and affirming the clinical advantage of novel agents, with the goal of approval by regulatory authorities to expand patient treatment options. Given some challenges unique to brain tumors, early-stage clinical trials for novel targeted agents must integrate pharmacokinetics and tissue-based pharmacodynamic assessments to establish timely go-no-go decisions for larger studies. Surgical window-of-opportunity trials can allow for the comparison of treated and untreated tumor samples, verifying target engagement and its modulatory effects for evidence of biological activity. Due to the challenges of achieving the required sample sizes to power efficacy analyses, later-stage trials of targeted therapies can include basket trials which test one drug on multiple tumor types, while umbrella trials evaluate several biomarkers within a single histology. Platform trials can also be leveraged to investigate multiple biomarker-driven hypotheses within a unified protocol and can incorporate Bayesian algorithms for adaptive randomization. Selecting appropriate endpoints, such as progression-free survival or overall response rate, is crucial and novel response assessment criteria need to be considered in the context of the tumor and therapy being investigated. Lastly, inclusivity in trials is essential to address health disparities and engage diverse patient populations to ensure real-world impact. Future trials should build upon the knowledge gained from both initial achievements and past setbacks in the field.

由于更容易获得生物标记物检测以及对肿瘤发生的分子基础有了更深入的了解,研究脑肿瘤靶向治疗的基于生物标记物的临床试验激增。临床试验的设计对于评估安全性、确认有效性和肯定新型药物的临床优势至关重要,其目标是获得监管机构的批准,以扩大患者的治疗选择。鉴于脑肿瘤所特有的一些挑战,新型靶向药物的早期临床试验必须整合药代动力学和基于组织的药效学评估,以便为更大规模的研究及时做出 "去还是不去 "的决定。外科机会之窗试验可以对治疗和未治疗的肿瘤样本进行比较,验证靶点参与及其调节作用,以获得生物活性证据。由于难以达到疗效分析所需的样本量,靶向疗法的后期试验可包括篮式试验,即对多种肿瘤类型的一种药物进行测试,而伞式试验则对单一组织学中的几种生物标记物进行评估。平台试验也可用于在统一方案中研究多种生物标记物驱动的假设,并可采用贝叶斯算法进行自适应随机化。选择适当的终点(如无进展生存期或总体反应率)至关重要,需要根据所研究的肿瘤和疗法来考虑新的反应评估标准。最后,试验的包容性对于解决健康差异和吸引不同患者群体参与以确保对现实世界产生影响至关重要。未来的试验应建立在从该领域的初步成就和过去挫折中获得的知识基础之上。
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引用次数: 0
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Journal of Neuro-Oncology
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