哮喘联盟

摘要
   综述目的：设计针对疗效和哮喘治疗循证指南金标准的随机对照试验，研究干预措施是否有助于让某些患者处于理想环境下。对实用临床试验和观察性研究进行评价，以了解其在现实生活中的疗效或有效性。综述总结了疗效和有效性试验的优点和不足、近期哮喘治疗有效性试验的结果，以促使进行更多的有效性研究。
   近期发现：近期实用临床试验和观察性研究在不同现实患者人群中评价干预后的转归，这些患者包括吸烟者、不同依从性、不同吸入器使用和不同基线哮喘控制状态的患者。对于临床实践中有关白三烯受体拮抗剂和皮质激素吸入治疗（ICS）有效性、补充指南有关吸烟者干预有效性，研究结果对其构成挑战。同时，研究结果开始阐述有关指南中ICS和吸入装置之间存在的差距。主动参与对观察性研究的研究方法进行提炼，使得哮喘转归能更好的整合进入所有类型的试验。
   总结：实用临床试验和观察性研究的结果是告知指南推荐措施和医疗服务提供者、患者和政策制定者进行决策的重要证据组成部分。

                                                                                           （刘国梁 审校）
                          CurrOpinAllergyClinImmunol.2013Feb;13(1):50-7.doi:10.1097/ACI.0b013e32835ad059.


Efficacy versus effectiveness trials: informing guidelines for asthma management.

Price D, Hillyer EV, van der Molen T.

Source
aAcademic Primary Care, Division of Applied Health Sciences, University of Aberdeen, Aberdeen bResearch in Real Life Ltd, Cambridge, UK cDepartment of General Practice, Groningen Research Institute for Asthma and COPD, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.

Abstract
PURPOSE OF REVIEW: Randomized controlled trials, known as efficacy trials and long considered the gold standard for evidence-based asthma guidelines, are designed to test whether interventions have a benefit for selective patient populations under ideal conditions. The goal of pragmatic trials and observational studies instead is to understand real-life efficacy, known as effectiveness. This review summarizes the strengths and limitations of efficacy and effectiveness trials, results of recent effectiveness trials in asthma and initiatives promoting effectiveness research.
RECENT FINDINGS: Recent pragmatic trials and observational studies have examined outcomes of interventions for diverse real-life patient populations, including smokers and patients with variable adherence, inhaler technique and baseline asthma control. Study results challenge practice guidelines regarding relative effectiveness of leukotriene receptor antagonists and inhaled corticosteroids (ICS); supplement guidelines with regard to effectiveness of interventions in smokers; and begin to address gaps in guidelines regarding choice of ICS and inhaler device. Initiatives are ongoing to refine methods of observational research and to harmonize asthma outcomes for better integration of results from all types of trials.
SUMMARY: Results of pragmatic trials and observational studies are an important component of the evidence needed to inform guideline recommendations and decision-making by healthcare providers, patients and policymakers.

CurrOpinAllergyClinImmunol.2013Feb;13(1):50-7.doi:10.1097/ACI.0b013e32835ad059.