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哮喘状况测量指标之间的一致性:对中重度哮喘低收入儿童的前瞻性研究。

Agreement among measures of asthma status: a prospective study of low-income children with moderate to severe asthma.

作者信息

Sharek Paul J, Mayer Michelle L, Loewy Lisa, Robinson Thomas N, Shames Richard S, Umetsu Dale T, Bergman David A

机构信息

Division of General Pediatrics, Stanford University School of Medicine, Palo Alto, California, USA.

出版信息

Pediatrics. 2002 Oct;110(4):797-804. doi: 10.1542/peds.110.4.797.

Abstract

BACKGROUND

Because no validated "gold standard" for measuring asthma outcomes exists, asthma interventions are often evaluated using a large number of disease status measures. Some of these measures may be redundant, whereas others may be complementary. Use of multiple outcomes may lead to ambiguous results, increased type I error rates, and be an inefficient use of resources including caregiver and patient/participant time and effort. Understanding the relationship between these measures may facilitate more parsimonious and valid evaluation strategies without loss of information.

OBJECTIVE

To assess the relationships between multiple measures of asthma disease status over time.

DESIGN/METHODS: We used data from a randomized, controlled trial of a comprehensive disease management program involving 119 disadvantaged inner-city children aged 5 to 12 years with moderate to severe asthma. Spearman correlations were calculated between the following asthma disease status measures: parent-reported disease symptoms, parent-reported health care utilization, functional health status using the American Academy of Pediatrics' validated Child Health Survey for Asthma (CHSA), diary data (symptom scores, night wakings, and bronchodilator use), and pulmonary function tests at baseline, 32 weeks, 52 weeks, and changes from baseline to 52 weeks.

RESULTS

Ninety-four (79%) of randomized patients participated at baseline and 52 weeks. Completion rates for outcome measures ranged from 79% (CHSA, spirometry data) to 64% (diary data). At baseline, asthma symptoms, health care utilization, and individual domains from the CHSA were significantly correlated (r = 0.21-0.53). These correlations were stable over the 52-week follow-up. Forced expiratory volume in 1 second and diary data did not correlate to any other measures at baseline, and these measures correlated only inconsistently with other measures at 32 weeks and 52 weeks. Baseline to 52-week changes in asthma symptoms, utilization, and the CHSA domains were significantly correlated (0.22-0.56), as were baseline to 52-week changes in symptom days, night wakings, and the CHSA domains (r = 0.24-0.64). Baseline to 52-week changes in forced expiratory volume in 1 second and diary data did not correlate with other measures.

CONCLUSIONS

These results suggest that asthma status and change in asthma status over time after introduction of a disease management intervention are best characterized by parent-reported symptoms, parent-reported utilization, and functional health status measures. Asthma diaries and pulmonary function tests did not seem to provide additional benefit, although they may play an important role in individual patient management. Our findings suggest a parsimonious evaluation strategy would include collection of key data elements regarding symptoms, utilization, and functional health status only, without loss of vital response information.

摘要

背景

由于不存在用于衡量哮喘治疗效果的经过验证的“金标准”,哮喘干预措施通常使用大量疾病状态指标进行评估。其中一些指标可能是冗余的,而其他指标可能是互补的。使用多个结果指标可能会导致结果不明确、I型错误率增加,并且会低效利用包括护理人员以及患者/参与者的时间和精力在内的资源。了解这些指标之间的关系可能有助于制定更简洁有效的评估策略,同时又不会丢失信息。

目的

评估哮喘疾病状态的多个指标随时间的关系。

设计/方法:我们使用了一项综合疾病管理项目的随机对照试验数据,该项目涉及119名年龄在5至12岁、患有中度至重度哮喘的内城区贫困儿童。计算了以下哮喘疾病状态指标之间的Spearman相关性:家长报告的疾病症状、家长报告的医疗保健利用情况、使用美国儿科学会验证的儿童哮喘健康调查(CHSA)得出的功能健康状况、日记数据(症状评分、夜间醒来次数和支气管扩张剂使用情况),以及在基线、32周、52周时的肺功能测试,以及从基线到52周的变化情况。

结果

94名(79%)随机分组的患者在基线和52周时参与研究。结果指标的完成率从79%(CHSA、肺活量测定数据)到64%(日记数据)不等。在基线时,哮喘症状、医疗保健利用情况以及CHSA中的各个领域显著相关(r = 0.21 - 0.53)。这些相关性在52周的随访期间保持稳定。第1秒用力呼气量和日记数据在基线时与任何其他指标均无相关性,并且这些指标在32周和52周时与其他指标的相关性也不一致。哮喘症状、利用情况以及CHSA领域从基线到52周的变化显著相关(0.22 - 0.56),症状天数、夜间醒来次数以及CHSA领域从基线到52周的变化也是如此(r = 0.24 - 0.64)。第1秒用力呼气量和日记数据从基线到52周的变化与其他指标不相关。

结论

这些结果表明,在引入疾病管理干预措施后,哮喘状态以及哮喘状态随时间的变化最好通过家长报告的症状、家长报告的利用情况以及功能健康状况指标来表征。哮喘日记和肺功能测试似乎并未提供额外的益处,尽管它们可能在个体患者管理中发挥重要作用。我们的研究结果表明,一种简洁的评估策略将仅包括收集有关症状、利用情况和功能健康状况的关键数据元素,而不会丢失重要的反应信息。

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