Bevacizumab monotherapy

贝伐单抗单药治疗
  • 文章类型: Journal Article
    背景:贝伐单抗在脑放射性坏死(CRN)治疗中显示出优异的疗效,但是由于药品价格高昂,其经济负担仍然很重。本研究旨在从中国付款人的角度评估贝伐单抗治疗CRN的成本-效果。
    方法:建立了一个决策树模型来比较贝伐单抗和皮质类固醇用于CRN治疗的成本和健康结果。疗效和安全性数据来自NCT01621880试验,比较了贝伐单抗单药与糖皮质激素治疗鼻咽癌患者CRN的有效性和安全性,并证明贝伐单抗引起的反应明显高于皮质类固醇(65.5%vs.31.5%,P<0.001),两组之间的不良事件无显着差异。“非重复”状态的效用值是从真实世界数据中导出的。成本和其他效用值是从权威的中国网络数据库和已发表的文献中收集的。主要结果是总成本,质量调整寿命年(QALYs),和增量成本效益比(ICER)。通过单向和概率敏感性分析对模型的不确定性进行评估。
    结果:贝伐单抗治疗增加0.12(0.48vs.0.36)QALY与皮质类固醇治疗相比,以及2010年的增量成本(4260美元与2160美元)。由此产生的ICER为$16,866/QALY,低于中国38223美元/QALY的支付意愿门槛。贝伐单抗的价格,体重,复发状态的效用值是ICER的关键影响参数。概率敏感性分析显示,贝伐单抗的成本-效果概率为84.9%。
    结论:与皮质类固醇相比,贝伐单抗是中国CRN治疗的一种经济选择。
    BACKGROUND: Bevacizumab shows superior efficacy in cerebral radiation necrosis (CRN) therapy, but its economic burden remains heavy due to the high drug price. This study aims to evaluate the cost-effectiveness of bevacizumab for CRN treatment from the Chinese payers\' perspective.
    METHODS: A decision tree model was developed to compare the costs and health outcomes of bevacizumab and corticosteroids for CRN therapy. Efficacy and safety data were derived from the NCT01621880 trial, which compared the effectiveness and safety of bevacizumab monotherapy with corticosteroids for CRN in nasopharyngeal cancer patients, and demonstrated that bevacizumab invoked a significantly higher response than corticosteroids (65.5% vs. 31.5%, P < 0.001) with no significant differences in adverse events between two groups. The utility value of the \"non-recurrence\" status was derived from real-world data. Costs and other utility values were collected from an authoritative Chinese network database and published literature. The primary outcomes were total costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER). The uncertainty of the model was evaluated via one-way and probabilistic sensitivity analyses.
    RESULTS: Bevacizumab treatment added 0.12 (0.48 vs. 0.36) QALYs compared to corticosteroid therapy, along with incremental costs of $ 2010 ($ 4260 vs. $ 2160). The resultant ICER was $ 16,866/QALY, which was lower than the willingness-to-pay threshold of $ 38,223/QALY in China. The price of bevacizumab, body weight, and the utility value of recurrence status were the key influential parameters for ICER. Probabilistic sensitivity analysis revealed that the probability of bevacizumab being cost-effectiveness was 84.9%.
    CONCLUSIONS: Compared with corticosteroids, bevacizumab is an economical option for CRN treatment in China.
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  • 文章类型: Journal Article
    OBJECTIVE: Our aim was to compare the clinical outcomes of patients treated with bevacizumab combined with corticosteroids and those with bevacizumab monotherapy from a radiation-induced brain necrosis (RN) registry cohort (NCT03908502).
    METHODS: We utilized clinical data from a prospective RN registry cohort (NCT03908502) from July 2017 to June 2020. Patients were considered eligible if they had symptomatic RN after radiotherapy for nasopharyngeal carcinoma (NPC) and received bevacizumab (5 mg/kg, two to four cycles) with a minimum follow-up time of 3 months. The primary outcome was a 2-month response rate determined by MRI and clinical symptoms. Secondary outcomes included quality of life [evaluated by the abbreviated World Health Organization Quality of Life (WHOQOL-BREF) questionnaire] and cognitive function (evaluated by the Montreal Cognitive Assessment scale) at 2 months, RN recurrence during follow-up, and adverse events.
    RESULTS: A total of 123 patients (34 in the combined therapy group and 89 in the monotherapy group) were enrolled in our study with a median follow-up time of 0.97 year [interquartile range (IQR) = 0.35-2.60 years]. The clinical efficacy of RN did not differ significantly between patients in these two groups [odds ratio (OR) = 1.642, 95%CI = 0.584-4.614, p = 0.347]. Furthermore, bevacizumab combined with corticosteroids did not reduce recurrence compared with bevacizumab monotherapy [hazard ratio (HR) = 1.329, 95%CI = 0.849-2.079, p = 0.213]. The most common adverse events of bevacizumab were hypertension (17.89%), followed by nosebleed (8.13%) and fatigue (8.13%). There was no difference in grade 2 or more severe adverse events between the two groups (p = 0.811).
    CONCLUSIONS: Our results showed that the treatment strategy of combining bevacizumab with corticosteroids did not lead to better clinical outcomes for RN patients with a background of radiotherapy for nasopharyngeal carcinoma.
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  • 文章类型: Journal Article
    恶性黑色素瘤的发病率在世界范围内正在上升,转移性疾病的生存率仍然很低。最近,新的治疗方案已经可用。尽管如此,需要预测性生物标志物来优化该患者组的治疗.在这项研究中,我们研究了60种血管生成因子在接受抗血管内皮生长因子A抗体贝伐单抗治疗的转移性黑色素瘤患者中的预测价值.35名患者被纳入临床II期试验,基线血清样本通过多重蛋白阵列分析。高血清浓度的激活素A与治疗的客观反应(OR)显着相关(p=0.014)。通过免疫组织化学进一步研究表明与治疗反应有临界关联的候选蛋白。激活素A的强表达,白细胞介素-1β,转移灶中尿激酶型纤溶酶原激活物受体与OR显着相关(分别为p=0.011,p=0.003和p=0.007),以及激活的血管生成的标记,如更多的增殖血管和肾小球微血管增生的存在。我们的发现表明,这些蛋白质可能是贝伐单抗单药治疗的潜在预测标志物。
    The incidence of malignant melanoma is rising worldwide and survival for metastatic disease is still poor. Recently, new treatment options have become available. Still, predictive biomarkers are needed to optimise treatment for this patient group. In this study, we investigated the predictive value of 60 angiogenic factors in patients with metastatic melanoma treated with the anti-vascular endothelial growth factor A antibody bevacizumab. Thirty-five patients were included in a clinical phase II trial and baseline serum samples were analysed by multiplex protein array. High-serum concentration of Activin A was significantly associated with objective response (OR) to treatment (p = 0.014). Candidate proteins that indicated a borderline association with treatment response were further investigated by immunohistochemistry. Strong expression of Activin A, interleukin-1β, and urokinase-type plasminogen activator receptor in metastases was significantly associated with OR (p = 0.011, p = 0.003, and p = 0.007, respectively), as well as with markers of activated angiogenesis, such as higher number of proliferating vessels and the presence of glomeruloid microvascular proliferations. Our findings indicate that these proteins may be potential predictive markers for treatment with bevacizumab monotherapy.
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