【佳學基因檢測】2 型神經(jīng)纖維瘤病相關前庭神經(jīng)鞘瘤 II 期抗腫瘤藥物研究的建議反應標準

1年靶向藥物要多少錢評價

探索明白《J Neurooncol》在?2009 May;93(1):61-77發(fā)表了一篇題目為《2 型神經(jīng)纖維瘤病相關前庭神經(jīng)鞘瘤 II 期抗腫瘤藥物研究的建議反應標準》腫瘤靶向藥物治療基因檢測臨床研究文章。該研究由Scott R Plotkin,?Chris Halpin,?Jaishri O Blakeley,?William H Slattery rd,?D Bradley Welling,?Susan M Chang,?Jay S Loeffler,?Gordon J Harris,?A Gregory Sorensen,?Michael J McKenna,?Fred G Barker nd等完成。促進了腫瘤的正確治療與個性化用藥的發(fā)展，進一步強調(diào)了基因信息檢測與分析的重要性。

腫瘤靶向藥物及正確治療臨床研究內(nèi)容關鍵詞：

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腫瘤靶向治療基因檢測臨床應用結(jié)果

2型神經(jīng)纖維瘤?。∟F2）是一種以多發(fā)性神經(jīng)鞘瘤，尤其是前庭神經(jīng)鞘瘤（VS）和腦膜瘤為特征的抑癌基因綜合征。目前正在探索抗癌藥物試驗，但 NF2 沒有標準化的終點。我們回顧了 NF2 臨床試驗的挑戰(zhàn)，并提出了用于初始 II 期研究的可能反應標準。我們建議在此類試驗中使用兩個主要的反應標準。客觀放射學反應定義為 VS 體積持久減少 20% 或更多，基于通過內(nèi)耳道收集 3 毫米或更細切口的增強后 T1 加權 MRI 圖像。聽力反應被定義為在聽力學中使用 50 個單詞的記錄列表在單詞識別分數(shù)方面的統(tǒng)計顯著改善。概述了結(jié)合放射學反應和聽力反應的可能復合終點。我們強調(diào)應對評估中的陷阱，并提出指導方針，以盡量減少對應對的誤解。我們還確定了 NF2 的研究目標，以促進未來的試驗進行，例如確定對未經(jīng)治療的 NF2 相關 VS 的腫瘤進展時間和可測量聽力損失時間的預期，以及這兩個終點與患者預后因素（如年齡）的關系、基線腫瘤體積和疾病嚴重程度的測量）。這些數(shù)據(jù)將有助于未來使用基于腫瘤大小和聽力穩(wěn)定性的端點，這可能更適合測試某些藥物。我們鼓勵在為該人群開發(fā) II 期試驗的早期采用標準化終點，以促進不同藥物試驗之間結(jié)果的比較。

腫瘤發(fā)生與反復轉(zhuǎn)移國際數(shù)據(jù)庫描述：

Neurofibromatosis type 2 (NF2) is a tumor suppressor gene syndrome characterized by multiple schwannomas, especially vestibular schwannomas (VS), and meningiomas. Anticancer drug trials are now being explored, but there are no standardized endpoints in NF2. We review the challenges of NF2 clinical trials and suggest possible response criteria for use in initial phase II studies. We suggest two main response criteria in such trials. Objective radiographic response is defined as a durable 20% or greater reduction in VS volume based on post-contrast T1-weighted MRI images collected with 3 mm or finer cuts through the internal auditory canal. Hearing response is defined as a statistically significant improvement in word recognition scores using 50-word recorded lists in audiology. A possible composite endpoint incorporating both radiographic response and hearing response is outlined. We emphasize pitfalls in response assessment and suggest guidelines to minimize misinterpretations of response. We also identify research goals in NF2 to facilitate future trial conduct, such as identifying the expectations for time to tumor progression and time to measurable hearing loss in untreated NF2-related VS, and the relation of both endpoints to patient prognostic factors (such as age, baseline tumor volume, and measures of disease severity). These data would facilitate future use of endpoints based on stability of tumor size and hearing, which might be more appropriate for testing certain drugs. We encourage adoption of standardized endpoints early in the development of phase II trials for this population to facilitate comparison of results across trials of different agents.