Morris Katrina A, Golding John F, Axon Patrick R, Afridi Shazia, Blesing Claire, Ferner Rosalie E, Halliday Dorothy, Jena Raj, Pretorius Pieter M, Evans D Gareth, McCabe Martin G, Parry Allyson
Nuffield Department of Neurosciences, University of Oxford, John Radcliffe Hospital, Oxford, UK (K.A.M., A.P.); University of New South Wales, St Vincent's Clinical School, Darlinghurst, Australia (K.A.M.); University of Westminster, London, UK (J.F.G.); Institute of Psychiatry, King's College, London, UK (J.F.G., R.E.F.); Addenbrooke's Hospital, Cambridge, UK (P.R.A.); Department of Neurology, Guy's & St Thomas' Hospital, London, UK (S.A., R.E.F.); Oxford University Hospitals NHS Trust, Oxford, UK (D.H., C.B., A.P., P.M.P.); Department of Oncology, University of Cambridge, Addenbrooke's Hospital, Cambridge, UK (R.J.); Genomic Medicine, Institute of Human Development, MAHSC, University of Manchester, St Mary's Hospital, Manchester, UK (D.G.E.); Centre for Paediatric, Teenage and Young Adult Cancer, Institute of Cancer Sciences, University of Manchester, Manchester, UK (M.G.M.).
Neurooncol Pract. 2016 Dec;3(4):281-289. doi: 10.1093/nop/npv065. Epub 2016 Jan 7.
NF2 patients develop multiple nervous system tumors including bilateral vestibular schwannomas (VS). The tumors and their surgical treatment are associated with deafness, neurological disability, and mortality.Medical treatment with bevacizumab has been reported to reduce VS growth and to improve hearing. In addition to evaluating these effects, this study also aimed to determine other important consequences of treatment including patient-reported quality of life and the impact of treatment on surgical VS rates.
Patients treated with bevacizumab underwent serial prospective MRI, audiology, clinical, CTCAE-4.0 adverse events, and NFTI-QOL quality-of-life assessments. Tumor volumetrics were classified according to the REiNs criteria and annual VS surgical rates reviewed.
Sixty-one patients (59% male), median age 25 years (range, 10-57), were reviewed. Median follow-up was 23 months (range, 3-53). Partial volumetric tumor response (all tumors) was seen in 39% and 51% had stabilization of previously growing tumors. Age and pretreatment growth rate were predictors of response. Hearing was maintained or improved in 86% of assessable patients. Mean NFTI-QOL scores improved from 12.0 to 10.7 ( < .05). Hypertension was observed in 30% and proteinuria in 16%. Twelve treatment breaks occurred due to adverse events. The rates of VS surgery decreased after the introduction of bevacizumab.
Treatment with bevacizumab in this large, UK-wide cohort decreased VS growth rates and improved hearing and quality of life. The potential risk of surgical iatrogenic damage was also reduced due to an associated reduction in VS surgical rates. Ongoing follow-up of this cohort will determine the long-term benefits and risks of bevacizumab treatment.
神经纤维瘤病2型（NF2）患者会出现多种神经系统肿瘤，包括双侧前庭神经鞘瘤（VS）。这些肿瘤及其手术治疗与耳聋、神经功能障碍和死亡率相关。据报道，使用贝伐单抗进行药物治疗可减少VS生长并改善听力。除了评估这些效果外，本研究还旨在确定治疗的其他重要后果，包括患者报告的生活质量以及治疗对VS手术率的影响。
接受贝伐单抗治疗的患者接受了系列前瞻性磁共振成像（MRI）、听力学、临床、癌症治疗和不良反应通用术语标准（CTCAE）4.0不良事件以及神经纤维瘤病生活质量指数（NFTI-QOL）生活质量评估。根据神经鞘瘤反应评估标准（REiNs）对肿瘤体积进行分类，并回顾年度VS手术率。
共纳入61例患者（59%为男性），中位年龄25岁（范围10 - 57岁）。中位随访时间为23个月（范围3 - 53个月）。39%的患者出现部分肿瘤体积反应（所有肿瘤），51%先前生长的肿瘤病情稳定。年龄和治疗前生长率是反应的预测因素。86%可评估的患者听力得以维持或改善。NFTI-QOL平均得分从12.0提高到10.7（P < 0.05）。30%的患者出现高血压，16%的患者出现蛋白尿。因不良事件发生了12次治疗中断。引入贝伐单抗后VS手术率下降。
在这个全英国范围的大型队列中，使用贝伐单抗治疗降低了VS生长率，改善了听力和生活质量。由于VS手术率的相关降低，手术医源性损伤的潜在风险也降低了。对该队列的持续随访将确定贝伐单抗治疗的长期益处和风险。
