Required efficacy for novel therapies in BCG‐unresponsive non‐muscle invasive bladder cancer: Do current recommendations really reflect clinically meaningful outcomes?
Required efficacy for novel therapies in BCG‐unresponsive non‐muscle invasive bladder cancer: Do current recommendations really reflect clinically meaningful outcomes?
复制标题
BCG 无反应非肌层浸润性膀胱癌新疗法所需的疗效：当前的建议是否真正反映了具有临床意义的结果？
作者：
M. Wettstein;D. Naimark;T. Hermanns;J. Herrera;Ardalan E. Ahmad;M. Jewett;G. Kulkarni
单臂试验目前是一种公认的研究设计，用于研究新型疗法（NT）在膀胱内卡介苗（BCG）免疫治疗无效的非肌层浸润性膀胱癌（NMIBC）中的疗效，因为随机对照试验要么不可行（对照药物：早期根治性环磷酰胺; ERC），要么不符合伦理（对照药物：安慰剂）。为了指导此类单组试验的设计，专家组发表了具有临床意义的结果的建议。本研究的目的是定量验证这些建议的适当性。
Single‐arm trials are currently an accepted study design to investigate the efficacy of novel therapies (NT) in non‐muscle invasive bladder cancer (NMIBC) unresponsive to intravesical Bacillus Calmette‐Guérin (BCG) immunotherapy as randomized controlled trials are either unfeasible (comparator: early radical cystectomy; ERC), or unethical (comparator: placebo). To guide the design of such single‐arm trials, expert groups published recommendations for clinically meaningful outcomes. The aim of this study was to quantitatively verify the appropriateness of these recommendations.
影响因子：
45.3
作者：
Kohn, Christine G.;Zeichner, Simon B.;Flowers, Christopher R.
通讯作者：
Flowers, Christopher R.
影响因子：
45.3
作者：
Herr, HW;Faulkner, JR;Crawford, ED
通讯作者：
Crawford, ED