Advancing neurofibromatosis and schwannomatosis clinical trial design: Consensus recommendations from the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration.
作者信息
Merker Vanessa L, Gross Andrea M, Widemann Brigitte C, Plotkin Scott R
机构信息
Department of Neurology and Cancer Center, Massachusetts General Hospital, Boston, MA, USA.
Pediatric Oncology Branch, National Cancer Institute, Bethesda, MD, USA.
出版信息
Clin Trials. 2024 Feb;21(1):3-5. doi: 10.1177/17407745231201345. Epub 2023 Sep 29.
Abstract
摘要
相似文献
1
Advancing neurofibromatosis and schwannomatosis clinical trial design: Consensus recommendations from the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration.神经纤维瘤病和施万细胞瘤病临床试验设计进展：神经纤维瘤病和施万细胞瘤病反应评估（REiNS）国际协作组的共识建议
Clin Trials. 2024 Feb;21(1):3-5. doi: 10.1177/17407745231201345. Epub 2023 Sep 29.
2
Recommendations for the collection and annotation of biosamples for analysis of biomarkers in neurofibromatosis and schwannomatosis clinical trials.神经纤维瘤病和雪旺细胞瘤临床试验中生物标志物分析用生物样本采集和注释的推荐意见。
Clin Trials. 2024 Feb;21(1):40-50. doi: 10.1177/17407745231203330. Epub 2023 Oct 31.
3
Patient-reported outcomes in neurofibromatosis and schwannomatosis clinical trials.神经纤维瘤病和雪旺氏细胞瘤病临床试验中的患者报告结局。
Neurology. 2013 Nov 19;81(21 Suppl 1):S6-14. doi: 10.1212/01.wnl.0000435747.02780.bf.
4
Enhancing Neurofibromatosis Clinical Trial Outcome Measures Through Patient Engagement: Lessons From REiNS.通过患者参与增强神经纤维瘤病临床试验的结果测量：REiNS 的经验教训。
Neurology. 2021 Aug 17;97(7 Suppl 1):S4-S14. doi: 10.1212/WNL.0000000000012430. Epub 2021 Jul 6.
5
Conclusions and future directions for the REiNS International Collaboration.REiNS 国际合作的结论和未来方向。
Neurology. 2013 Nov 19;81(21 Suppl 1):S41-4. doi: 10.1212/01.wnl.0000435748.79908.c5.
6
Patient-reported measures of tinnitus for individuals with neurofibromatosis type 2-related schwannomatosis: Recommendations for clinical trials.神经纤维瘤病 2 型相关雪旺氏细胞瘤患者的耳鸣患者报告测量：临床试验建议。
Clin Trials. 2024 Feb;21(1):18-28. doi: 10.1177/17407745231217279. Epub 2024 Feb 6.
7
Achieving consensus for clinical trials: the REiNS International Collaboration.达成临床试验共识：REiNS 国际合作。
Neurology. 2013 Nov 19;81(21 Suppl 1):S1-5. doi: 10.1212/01.wnl.0000435743.49414.b6.
8
Current whole-body MRI applications in the neurofibromatoses: NF1, NF2, and schwannomatosis.当前全身磁共振成像在神经纤维瘤病中的应用：1型神经纤维瘤病、2型神经纤维瘤病和施万细胞瘤病
Neurology. 2016 Aug 16;87(7 Suppl 1):S31-9. doi: 10.1212/WNL.0000000000002929.
9
From process to progress-2017 International Conference on Neurofibromatosis 1, Neurofibromatosis 2 and Schwannomatosis.从进程到进展——2017 年神经纤维瘤病 1 型、神经纤维瘤病 2 型和施万细胞瘤国际会议。
Am J Med Genet A. 2019 Jun;179(6):1098-1106. doi: 10.1002/ajmg.a.61112. Epub 2019 Mar 25.
10
Potential endpoints for assessment of bone health in persons with neurofibromatosis type 1.评估 1 型神经纤维瘤病患者骨骼健康的潜在终点。
Clin Trials. 2024 Feb;21(1):29-39. doi: 10.1177/17407745231201338. Epub 2023 Sep 29.
本文引用的文献
1
Patient-reported measures of tinnitus for individuals with neurofibromatosis type 2-related schwannomatosis: Recommendations for clinical trials.神经纤维瘤病 2 型相关雪旺氏细胞瘤患者的耳鸣患者报告测量：临床试验建议。
Clin Trials. 2024 Feb;21(1):18-28. doi: 10.1177/17407745231217279. Epub 2024 Feb 6.
2
Recommendations for assessing appearance concerns related to plexiform and cutaneous neurofibromas in neurofibromatosis 1 clinical trials.评估 1 型神经纤维瘤病临床试验中丛状和皮肤神经纤维瘤相关外观问题的建议。
Clin Trials. 2024 Feb;21(1):6-17. doi: 10.1177/17407745231205577. Epub 2023 Dec 23.
3
Perspectives of adults with neurofibromatosis regarding the design of psychosocial trials: Results from an anonymous online survey.神经纤维瘤病患者对心理社会临床试验设计的看法：一项匿名在线调查的结果。
Clin Trials. 2024 Feb;21(1):73-84. doi: 10.1177/17407745231209224. Epub 2023 Nov 14.
4
Gene-targeted therapy for neurofibromatosis and schwannomatosis: The path to clinical trials.针对神经纤维瘤病和神经鞘瘤病的基因靶向治疗：通往临床试验之路。
Clin Trials. 2024 Feb;21(1):51-66. doi: 10.1177/17407745231207970. Epub 2023 Nov 8.
5
Recommendations for the collection and annotation of biosamples for analysis of biomarkers in neurofibromatosis and schwannomatosis clinical trials.神经纤维瘤病和雪旺细胞瘤临床试验中生物标志物分析用生物样本采集和注释的推荐意见。
Clin Trials. 2024 Feb;21(1):40-50. doi: 10.1177/17407745231203330. Epub 2023 Oct 31.
6
Potential endpoints for assessment of bone health in persons with neurofibromatosis type 1.评估 1 型神经纤维瘤病患者骨骼健康的潜在终点。
Clin Trials. 2024 Feb;21(1):29-39. doi: 10.1177/17407745231201338. Epub 2023 Sep 29.
7
Perspectives of adolescents with neurofibromatosis 1 and cutaneous neurofibromas: Implications for clinical trials.神经纤维瘤病 1 型伴皮肤神经纤维瘤青少年的观点：对临床试验的启示。
Clin Trials. 2024 Feb;21(1):67-72. doi: 10.1177/17407745231178839. Epub 2023 Jun 2.
8
FDA regulatory considerations for the review of drugs intended to treat pediatric cancers and rare tumors.美国食品药品监督管理局（FDA）对用于治疗儿童癌症和罕见肿瘤药物审评的监管考量。
Curr Opin Pediatr. 2023 Feb 1;35(1):48-54. doi: 10.1097/MOP.0000000000001201. Epub 2022 Nov 11.
9
Updated diagnostic criteria and nomenclature for neurofibromatosis type 2 and schwannomatosis: An international consensus recommendation.神经纤维瘤病 2 型和雪旺细胞瘤病的更新诊断标准和命名：国际共识建议。
Genet Med. 2022 Sep;24(9):1967-1977. doi: 10.1016/j.gim.2022.05.007. Epub 2022 Jun 9.
10
Enhancing Neurofibromatosis Clinical Trial Outcome Measures Through Patient Engagement: Lessons From REiNS.通过患者参与增强神经纤维瘤病临床试验的结果测量：REiNS 的经验教训。
Neurology. 2021 Aug 17;97(7 Suppl 1):S4-S14. doi: 10.1212/WNL.0000000000012430. Epub 2021 Jul 6.
Zotero 插件