神经纤维瘤病临床试验中肿瘤反应的影像学推荐建议。
Recommendations for imaging tumor response in neurofibromatosis clinical trials.
机构信息
From the Pediatric Oncology Branch (E.D., B.C.W.), National Cancer Institute, Bethesda, MD; Department of Neurology (S.L.A.-H.), The Children's Hospital at Westmead, Sydney, Australia; Department of Medical Genetics (D. B.-V.), Mayo Clinic, Rochester, MN; Neurosurgical Service (F.G.B.), Department of Radiology (G.J.H.), and Department of Neurology and Cancer Center (S.R.P.), Massachusetts General Hospital, Boston, MA; Department of Neuroradiology (S.C.), King's College Hospital, London, UK; Department of Genetic Medicine (D.G.E.), MAHSC, St Mary's Hospital, Manchester, UK; Division of Oncology (M.J.F.) and Department of Radiology (D.J.), The Children's Hospital of Philadelphia; Department of Pediatrics (M.J.F.), The Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA; Department of Neurosurgery (S.G.), Hôpital Beaujon, Clichy, France; Division of Pediatric Hematology/Oncology and NYU Cancer Institute (M.A.K.), NYU Langone Medical Center, New York, NY; Department of Genetics (B.R.K.), University of Alabama at Birmingham, Birmingham, AL; Department of Neurology (V.M.), University Medical Center Hamburg-Eppendorf, Hamburg, Germany; Department of Radiology (T.Y.P.), Boston Children's Hospital, Boston, MA; and Department of Pediatrics (K.R., C.-S.S.), Riley Hospital for Children, Indianapolis, IN.
出版信息
Neurology. 2013 Nov 19;81(21 Suppl 1):S33-40. doi: 10.1212/01.wnl.0000435744.57038.af.
OBJECTIVE
Neurofibromatosis (NF)-related benign tumors such as plexiform neurofibromas (PN) and vestibular schwannomas (VS) can cause substantial morbidity. Clinical trials directed at these tumors have become available. Due to differences in disease manifestations and the natural history of NF-related tumors, response criteria used for solid cancers (1-dimensional/RECIST [Response Evaluation Criteria in Solid Tumors] and bidimensional/World Health Organization) have limited applicability. No standardized response criteria for benign NF tumors exist. The goal of the Tumor Measurement Working Group of the REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) committee is to propose consensus guidelines for the evaluation of imaging response in clinical trials for NF tumors.
METHODS
Currently used imaging endpoints, designs of NF clinical trials, and knowledge of the natural history of NF-related tumors, in particular PN and VS, were reviewed. Consensus recommendations for response evaluation for future studies were developed based on this review and the expertise of group members.
RESULTS
MRI with volumetric analysis is recommended to sensitively and reproducibly evaluate changes in tumor size in clinical trials. Volumetric analysis requires adherence to specific imaging recommendations. A 20% volume change was chosen to indicate a decrease or increase in tumor size. Use of these criteria in future trials will enable meaningful comparison of results across studies.
CONCLUSIONS
The proposed imaging response evaluation guidelines, along with validated clinical outcome measures, will maximize the ability to identify potentially active agents for patients with NF and benign tumors.
目的
神经纤维瘤病（NF）相关的良性肿瘤，如丛状神经纤维瘤（PN）和前庭神经鞘瘤（VS），可导致严重的发病率。针对这些肿瘤的临床试验已经问世。由于 NF 相关肿瘤的临床表现和自然史存在差异，用于实体瘤的反应标准（一维/RECIST [实体瘤反应评价标准]和二维/世界卫生组织）的适用性有限。没有用于良性 NF 肿瘤的标准化反应标准。REiNS（神经纤维瘤病和神经鞘瘤病反应评估）委员会肿瘤测量工作组的目标是提出用于 NF 肿瘤临床试验中评估成像反应的共识指南。
方法
审查了目前使用的成像终点、NF 临床试验设计以及 NF 相关肿瘤（特别是 PN 和 VS）的自然史知识。根据这一审查和小组成员的专业知识，制定了未来研究反应评估的共识建议。
结果
建议使用 MRI 进行容积分析，以在临床试验中灵敏且可重复地评估肿瘤大小的变化。容积分析需要遵守特定的成像建议。选择 20%的体积变化来表示肿瘤大小的减小或增大。在未来的试验中使用这些标准将能够在研究之间进行有意义的结果比较。
结论
拟议的成像反应评估指南，以及经过验证的临床结局衡量标准，将最大限度地提高识别 NF 患者和良性肿瘤潜在有效药物的能力。
Zotero 插件