PDF(Sci-hub)
Abstract：
OBJECTIVE: Neurofibromatosis type 2 (NF2) is a rare autosomal dominant disorder associated primarily with bilateral schwannomas seen on the superior vestibular branches of the eighth cranial nerves. Significant morbidity can result from surgical treatment of these tumors. Meningiomas, ependymomas, and other benign central nervous system tumors are also common in NF2. The lack of effective treatments for NF2 marks an unmet medical need.
METHODS: Here, we provide recommendations from a workshop, cochaired by Drs. D. Gareth Evans and Marco Giovannini, of 36 international researchers, physicians, representatives of the biotechnology industry, and patient advocates on how to accelerate progress toward NF2 clinical trials.
RESULTS: Workshop participants reached a consensus that, based on current knowledge, the time is right to plan and implement NF2 clinical trials. Obstacles impeding NF2 clinical trials and how to address them were discussed, as well as the candidate therapeutic pipeline for NF2.
CONCLUSIONS: Both phase 0 and phase II NF2 trials are near-term options for NF2 clinical trials. The number of NF2 patients in the population remains limited, and successful recruitment will require ongoing collaboration efforts between NF2 clinics.
METHODS: Here, we provide recommendations from a workshop, cochaired by Drs. D. Gareth Evans and Marco Giovannini, of 36 international researchers, physicians, representatives of the biotechnology industry, and patient advocates on how to accelerate progress toward NF2 clinical trials.
RESULTS: Workshop participants reached a consensus that, based on current knowledge, the time is right to plan and implement NF2 clinical trials. Obstacles impeding NF2 clinical trials and how to address them were discussed, as well as the candidate therapeutic pipeline for NF2.
CONCLUSIONS: Both phase 0 and phase II NF2 trials are near-term options for NF2 clinical trials. The number of NF2 patients in the population remains limited, and successful recruitment will require ongoing collaboration efforts between NF2 clinics.
摘要：
目的：2型神经纤维瘤病（NF2）是一种罕见的常染色体显性遗传病，主要与第八颅神经前庭上支的双侧神经鞘瘤有关。这些肿瘤的手术治疗可导致显著的发病率。脑膜瘤,室管膜瘤,和其他良性中枢神经系统肿瘤在NF2中也很常见。缺乏有效的NF2治疗标志着未满足的医疗需求。
方法：这里，我们提供研讨会的建议，由博士担任联合主席D.GarethEvans和MarcoGiovannini,在36名国际研究人员中，医师,生物技术行业的代表，和患者倡导者如何加快NF2临床试验的进展。
结果：研讨会参与者达成共识，根据目前的知识，现在是规划和实施NF2临床试验的时候了.讨论了阻碍NF2临床试验的障碍以及如何解决这些障碍，以及NF2的候选治疗管道。
结论：0期和II期NF2试验都是NF2临床试验的近期选择。NF2患者在人群中的数量仍然有限，成功的招募将需要NF2诊所之间持续的合作努力。
方法：这里，我们提供研讨会的建议，由博士担任联合主席D.GarethEvans和MarcoGiovannini,在36名国际研究人员中，医师,生物技术行业的代表，和患者倡导者如何加快NF2临床试验的进展。
结果：研讨会参与者达成共识，根据目前的知识，现在是规划和实施NF2临床试验的时候了.讨论了阻碍NF2临床试验的障碍以及如何解决这些障碍，以及NF2的候选治疗管道。
结论：0期和II期NF2试验都是NF2临床试验的近期选择。NF2患者在人群中的数量仍然有限，成功的招募将需要NF2诊所之间持续的合作努力。
