Development of a core outcome set for clinical trials in childhood asthma: a survey of clinicians, parents, and young people
Development of a core outcome set for clinical trials in childhood asthma: a survey of clinicians, parents, and young people
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DOI：
10.1186/1745-6215-13-103
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发表时间：
2012-07-02
期刊：
影响因子：
2.5
通讯作者：
Smyth, Rosalind L.
中科院分区：
文献类型：
--
作者：
Sinha, Ian P.;Gallagher, Ruairi;Smyth, Rosalind L.
背景：在儿童哮喘的临床试验中，经常测量反映短期疾病活动的结果，而很少评估功能状态、生活质量（QoL）和长期治疗效果。在这些领域的结果选择和测量方面，研究之间也存在不一致性。核心结局集的开发有可能减少试验之间的异质性，导致更有可能测量相关结果的研究，并通过降低结果报告偏倚的风险和确保所有试验提供可用信息来提高证据合成的价值。方法：通过英国儿科呼吸学会确定的儿科医生和专科护士完成了两轮德尔菲调查。在诊所招募的18岁以下儿童的父母单独队列参加每一轮。13岁以上的青少年哮喘患者参加了第一轮试验。学龄前儿童和学龄儿童的结果分别确定。我们确定了临床医生和家长/年轻人在常规临床评估中认为重要的结果。在第1轮中，46名临床医生在决定是否调整儿童哮喘治疗方案时提出了他们认为重要的结果，并使用开放式问题询问了49名父母/年轻人，他们如何判断他们的孩子（对于年轻人，他们自己）的哮喘治疗是否合适。两名研究人员独立地将反应分类为适当的、相应的结果。在第2轮中，43名临床医生和50名家长对至少10%的第1轮应答者建议的每个结果的重要性从0-4分进行评分，并选择最重要的三个结果。结果：在对学龄和学龄前哮喘儿童进行常规治疗的共同决策时，最重要的结果是白天和夜间症状、加重、生活质量和死亡率。父母和临床医生的结果大致一致，但父母更强调长期治疗效果。结论：我们开发了一种方法来确定临床医生、家长和年轻人在评估哮喘常规治疗时最相关的结果。日间和夜间症状、加重、生活质量和死亡率是哮喘儿童常规治疗的所有临床试验中应测量和报告的特别重要的结果。
Background: In clinical trials in childhood asthma, outcomes reflecting short-term disease activity are frequently measured, whilst functional status, quality of life (QoL), and long-term treatment effects are rarely assessed. There is also non-uniformity across studies in the selection and measurement of outcomes within these domains. The development of a core outcome set has the potential to reduce heterogeneity between trials, lead to research that is more likely to have measured relevant outcomes, and enhance the value of evidence synthesis by reducing the risk of outcome reporting bias and ensuring that all trials contribute usable information.Methods: Paediatricians and specialist nurses, identified through the British Paediatric Respiratory Society, completed a two-round Delphi survey. Separate cohorts of parents of children younger than 18 years, recruited in clinics, participated in each round. Young people with asthma, aged at least 13 years, participated in the first round. Outcomes were identified separately for preschool and school-aged children. We identified outcomes considered important in routine clinical assessment by clinicians and parents/young people. In round 1, 46 clinicians suggested outcomes they considered important when deciding whether to adjust a child's asthma therapy regime, and 49 parents/young people were asked, using open questions, how they judged whether their child's (for young people, their own) asthma therapy was appropriate. Two researchers independently classified responses into appropriate, corresponding outcomes. In round 2, 43 clinicians and 50 parents scored, from 0-4, the importance of each outcome suggested by at least 10 % of round 1 responders and selected the three most important.Results: The most important outcomes, when making shared decisions about regular therapies for school-aged and preschool children with asthma, were daytime and nocturnal symptoms, exacerbations, QoL, and mortality. Results from parents and clinicians were generally concordant, but parents placed more emphasis on long-term treatment effects.Conclusions: We have developed a methodology to identify outcomes of most relevance to clinicians, parents, and young people when evaluating regularly administered therapies for asthma. Daytime and nocturnal symptoms, exacerbations, QoL, and mortality are particularly important outcomes that should be measured and reported in all clinical trials of regular therapies for children with asthma.