Recommendations for imaging tumor response in neurofibromatosis clinical trials
Recommendations for imaging tumor response in neurofibromatosis clinical trials
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DOI：
10.1212/01.wnl.0000435744.57038.af
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发表时间：
2013-11-19
期刊：
影响因子：
9.9
通讯作者：
Widemann, Brigitte C.
中科院分区：
文献类型：
--
作者：
Dombi, Eva;Ardern-Holmes, Simone L.;Widemann, Brigitte C.
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）可导致大量的发病率。针对这些肿瘤的临床试验已经成为可能。由于疾病表现和NF相关肿瘤自然史的差异，用于实体癌的缓解标准（一维/RECIST [实体瘤缓解评价标准]和二维/世界卫生组织）适用性有限。良性NF肿瘤没有标准化的反应标准。REiNS（神经纤维瘤病和神经鞘瘤病的反应评价）委员会的肿瘤测量工作组的目标是提出共识的指导方针，为NF tumors.Methods的临床试验中的成像反应的评价：目前使用的成像终点，NF临床试验的设计，和NF相关肿瘤的自然史的知识，特别是PN和VS，进行了审查。共识的建议，为今后的研究反应评价的基础上，这一审查和专家组members.Results：建议MRI与体积分析，以敏感和可重复地评估肿瘤大小的变化在临床试验。体积分析需要遵守特定的成像建议。选择20%的体积变化来指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使跨study.Conclusions的结果有意义的比较：拟议的成像反应评价指南，沿着与验证的临床结果的措施，将最大限度地提高能力，以确定潜在的活性药物与NF和良性肿瘤患者。
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.