Neuro-Oncology Practice最新文献

{"title":"Platform trial design for Neurofibromatosis type 1, NF2-related Schwannomatosis and non-NF2-related Schwannomatosis: a potential model for rare diseases","authors":"B. Dhaenens, Günter Heimann, Annette Bakker, M. Nievo, Rosalie E. Ferner, D Gareth Evans, P. Wolkenstein, J. Leubner, C. Potratz, C. Carton, Uche Iloeje, George Kirk, J. Blakeley, Scott Plotkin, Michael J Fisher, Ae Rang Kim, P. Driever, Amedeo A Azizi, B. Widemann, A. Gross, Tom Parke, Eric Legius, R. Oostenbrink","doi":"10.1093/nop/npae001","DOIUrl":"https://doi.org/10.1093/nop/npae001","url":null,"abstract":"\u0000 \u0000 \u0000 Neurofibromatosis type 1, NF2-related schwannomatosis and non-NF2-related schwannomatosis (grouped under the abbreviation ‘NF’) are rare hereditary tumour predisposition syndromes. Due to the low prevalence, variability in the range and severity of manifestations, as well as limited treatment options, these conditions require innovative trial designs to accelerate the development of new treatments.\u0000 \u0000 \u0000 \u0000 Within European Patient-Centric Clinical Trial Platforms (EU-PEARL), we designed two platform-basket trials in NF. The trials were designed by a team of multidisciplinary NF experts and trial methodology experts.\u0000 \u0000 \u0000 \u0000 The trial will consist of an observational and a treatment period. The observational period will serve as a longitudinal natural history study. The platform trial design and randomisation to a sequence of available interventions allow for the addition of interventions during the trial. If a drug does not meet the predetermined efficacy endpoint or reveals unacceptable toxicities, participants may stop treatment on that arm and re-enter the observational period, where they can be re-randomised to a different treatment arm if eligible. Intervention-specific eligibility criteria and endpoints are listed in Intervention-Specific-Appendices (ISAs), allowing the flexibility and adaptability needed for highly variable and rare conditions like NF.\u0000 \u0000 \u0000 \u0000 These innovative platform-basket trials for NF may serve as a model for other rare diseases, as they will enhance the chance of identifying beneficial treatments through optimal learning from a small number of patients. The goal of these trials is to identify beneficial treatments for NF more rapidly and at lower cost than traditional, single-agent clinical trials.\u0000","PeriodicalId":506567,"journal":{"name":"Neuro-Oncology Practice","volume":"36 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-01-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139385146","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Towards gender equity in neuro-oncology","authors":"S. Sahebjam, Heather Leeper","doi":"10.1093/nop/npad079","DOIUrl":"https://doi.org/10.1093/nop/npad079","url":null,"abstract":"","PeriodicalId":506567,"journal":{"name":"Neuro-Oncology Practice","volume":" 6","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139144762","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Radiotherapy intensification for atypical and malignant meningiomas: a systematic review","authors":"S. Gaito, L. Goyal, R. Rieu, A. France, N. Burnet, C. Barker, S. Pan, R. Colaco, G. Minniti, F. Roncaroli, E. Smith, M. Aznar, G. Whitfield","doi":"10.1093/nop/npad077","DOIUrl":"https://doi.org/10.1093/nop/npad077","url":null,"abstract":"The outcomes of non-benign (WHO Grade 2 and 3) meningiomas are suboptimal and radiotherapy (RT) dose intensification strategies have been investigated. The purpose of this review is to report on clinical practice and outcomes with particular attention to RT doses and techniques. The PICO criteria (Population, Intervention, Comparison and Outcomes) were used to frame the research question, directed at outlining the clinical outcomes in patients with G2-3 meningiomas treated with radiotherapy (RT). The same search strategy was run in Embase and MEDLINE and, after deduplication, returned 1807 records. These were manually screened for relevance and 25 were included. Tumour outcomes and toxicities are not uniformly reported in the selected studies since different endpoints and time points have been used by different authors. Many risk factors for worse outcomes are described, the most common being suboptimal RT. This includes no or delayed RT, low doses, older techniques. A positive association between RT dose and progression-free survival (PFS) has been highlighted by analysing the studies in this review (10/25) that report the same endpoint (5y-PFS). This literature review has shown that standard practice RT leads to suboptimal tumour control rates in G2-3 meningiomas, with a significant proportion of disease recurring after a relatively short follow-up. Randomised controlled trials are needed in this setting to define the optimal RT approach. Given the increasing data to suggest a benefit of higher RT doses for high-risk meningiomas, novel RT technologies with highly conformal dose distributions are preferential to achieve optimal target coverage and organs at risk (OARs) sparing.","PeriodicalId":506567,"journal":{"name":"Neuro-Oncology Practice","volume":"21 4","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139173527","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Embryonal tumor with multi-layered rosettes (ETMR) located in the brainstem: Promising results after multimodal treatment including interstitial brachytherapy (SBT)","authors":"S. Jünger, D. Rueß, C. Kabbasch, Gerrit Gielen, Torsten Pietsch T, Pascal Johann, Pablo Landgraf, Martin Kocher, R. Goldbrunner, Thorsten Simon, M. Ruge","doi":"10.1093/nop/npad075","DOIUrl":"https://doi.org/10.1093/nop/npad075","url":null,"abstract":"","PeriodicalId":506567,"journal":{"name":"Neuro-Oncology Practice","volume":"299 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139205242","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Response to van Zandvoort et al. “Using a single neuropsychological task as a red flag creates false security”","authors":"Robin Grant","doi":"10.1093/nop/npad074","DOIUrl":"https://doi.org/10.1093/nop/npad074","url":null,"abstract":"","PeriodicalId":506567,"journal":{"name":"Neuro-Oncology Practice","volume":"14 9","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139237576","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Using a single neuropsychological task as a red flag creates false security","authors":"M. V. van Zandvoort, I. H. Huenges Wajer, Mariska Mantione, C. Ruis","doi":"10.1093/nop/npad073","DOIUrl":"https://doi.org/10.1093/nop/npad073","url":null,"abstract":"","PeriodicalId":506567,"journal":{"name":"Neuro-Oncology Practice","volume":"29 46","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139237482","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}