Which and How Many Patients Should Be Included in Randomised Controlled Trials to Demonstrate the Efficacy of Biologics in Primary Sjögren's Syndrome?
作者: Valérie Devauchelle-PensecJacques-Eric GottenbergSandrine Jousse-JoulinJean-Marie BerthelotAleth PerdrigerEric HachullaPierre Yves HatronXavier PuechalVéronique Le GuernJean SibiliaLaurent ChicheVincent GoebOlivier VittecoqClaire LarrocheAnne Laure FauchaisGilles HayemJacques MorelCharles ZarnitskyJean Jacques DubostPhilippe DieudéJacques Olivier PersDivi CornecRaphaele SerorXavier MarietteEmmanuel NowakAlain Saraux
刊名: PLoS ONE, 2017, Vol.10 (9)
来源数据库: Directory of Open Access Journals
DOI: 10.1371/journal.pone.0133907
原始语种摘要: The goal of this study was to determine how the choice of the primary endpoint influenced sample size estimates in randomised controlled trials (RCTs) of treatments for primary Sjögren's syndrome (pSS).We reviewed all studies evaluating biotechnological therapies in pSS to identify their inclusion criteria and primary endpoints. Then, in a large cohort (ASSESS), we determined the proportion of patients who would be included in RCTs using various inclusion criteria sets. Finally, we used the population of a large randomised therapeutic trial in pSS (TEARS) to assess the impact of various primary objectives and endpoints on estimated sample sizes. These analyses were performed only for the endpoints indicating greater efficacy of rituximab compared to the placebo.We identified 18 studies....
全文获取路径: DOAJ  (合作)
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影响因子:3.73 (2012)

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关键词翻译
关键词翻译
  • estimate 估计
  • smallest 最小的
  • percent 百分率
  • logical 逻辑的
  • score 得分
  • improvement 改良
  • evidence 
  • criteria 准则
  • dryness 干度
  • develop 发展