Abstract
Simple SummaryNeurofibromatosis type 2 (NF2) is a rare genetic hereditary disease characterized by multiple central nervous system tumors, most frequently bilateral vestibular schwannomas (VSs). No chemotherapeutic agents are available for clinical use, and surgery and radiotherapy are the only therapeutic options available now. Still, neither treatment option alleviates hearing loss in patients with NF2 and VS; they may even exacerbate it. However, bevacizumab has been reported to be effective in suppressing the tumor’s growth and has shown unprecedented efficacy in improving hearing. We describe a new ongoing and novel clinical trial, BeatNF2, a randomized, double-blinded, placebo-controlled, multicenter trial to assess bevacizumab’s efficacy and safety in patients with NF2. The study’s primary endpoint is improved hearing function 24 weeks after the beginning of the treatment protocol.Neurofibromatosis type 2 (NF2) causes bilateral vestibular schwannomas (VSs), leading to deafness. VS is treated by surgery or radiation, but neither treatments prevent hearing loss. Bevacizumab was found to be effective in suppressing the tumor’s growth and may help to improve hearing. We are conducting a randomized, double-blind, multicenter clinical trial to verify the efficacy and safety of bevacizumab in NF2-related VS. The primary objective is to evaluate the efficacy of bevacizumab in improving hearing in the affected ear. One of the secondary objectives is to evaluate bevacizumab’s efficacy in rechallenge treatment in relapsed cases. Sixty patients will randomly receive either bevacizumab or a placebo and will be clinically observed for 48 weeks in the initial intervention phase. In the first half (24 weeks), they will receive either 5 mg/kg of bevacizumab or a placebo drug. In the second half, all patients will receive 5 mg/kg of bevacizumab. If hearing function deteriorated in a patient who had shown improvement during the first phase, a rechallenge dose with bevacizumab would be offered.
Highlights
In 2009, the vascular endothelial growth factor (VEGF) antibody bevacizumab was reported to be effective in suppressing the growth of vestibular schwannomas (VSs) [20], and subsequent follow-up reports have shown unprecedented efficacy, whereby more than 50% of treated patients exhibited tumor shrinkage and, surprisingly, hearing improvement at a rate of just over 50% [21]
Improvement of maximum word recognition score (WRS) is defined as an increase of 20% points or greater over baseline score and a WRS of 50% points or above
Patients with Neurofibromatosis type 2 (NF2) associated VS will be randomly assigned in a 1-to-1 ratio to receive either bevacizumab or placebo
Summary
Neurofibromatosis type 2 (NF2) is an autosomal dominant disease that frequently causes bilateral vestibular schwannomas (VSs; known as acoustic neuromas). NF2 is designated an incurable disease by the Ministry of Health, Labor, and Welfare in Japan. Between 2009 and 2013, the national NF2 registry of the Japanese Ministry of Health and Welfare documented 807 NF2 patients (44% male, 56% female). In 587 patients for whom longitudinal disability data were available, the significant independent risk factors of progression included age of
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