Department of Pediatrics, The Hospital for Sick Children, Toronto, Ontario, Canada.
Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada.
JAMA Netw Open. 2022 Oct 3;5(10):e2234714. doi: 10.1001/jamanetworkopen.2022.34714.
Despite advances in asthma therapeutics, the burden remains highest in preschool children; therefore, it is critical to identify primary care tools that distinguish preschool children at high risk for burdensome disease for further evaluation. Current asthma prediction tools, such as the modified Asthma Predictive Index (mAPI), require invasive tests, limiting their applicability in primary care and low-resource settings.
To develop and evaluate the use of a symptom-based screening tool to detect children at high risk of asthma, persistent wheeze symptoms, and health care burden.
DESIGN, SETTING, AND PARTICIPANTS: The cohort for this diagnostic study included participants from the CHILD Study (n = 2511) from January 1, 2008, to December 31, 2012, the Raine Study from January 1, 1989, to December 31, 2012 (n = 2185), and the Canadian Asthma Primary Prevention Study (CAPPS) from January 1, 1989, to December 31, 1995 (n = 349), with active follow-up to date. Data analysis was performed from November 1, 2019, to May 31, 2022.
The CHILDhood Asthma Risk Tool (CHART) identified factors associated with asthma in patients at 3 years of age (timing and number of wheeze or cough episodes, use of asthma medications, and emergency department visits or hospitalizations for asthma or wheeze) to identify children with asthma or persistent symptoms at 5 years of age.
Within the CHILD Study cohort, CHART was evaluated against specialist clinician diagnosis and the mAPI. External validation was performed in both a general population cohort (Raine Study [Australia]) and a high-risk cohort (CAPPS [Canada]). Predictive accuracy was measured by sensitivity, specificity, area under the receiver operating characteristic curve (AUROC), and positive and negative predicted values.
Among 2511 children (mean [SD] age at 3-year clinic visit, 3.08 [0.17] years; 1324 [52.7%] male; 1608 of 2476 [64.9%] White) with sufficient questionnaire data to apply CHART at 3 years of age, 2354 (93.7%) had available outcome data at 5 years of age. CHART applied in the CHILD Study at 3 years of age outperformed physician assessments and the mAPI in predicting persistent wheeze (AUROC, 0.94; 95% CI, 0.90-0.97), asthma diagnosis (AUROC, 0.73; 95% CI, 0.69-0.77), and health care use (emergency department visits or hospitalization for wheeze or asthma) (AUROC, 0.70; 95% CI, 0.61-0.78). CHART had a similar predictive performance for persistent wheeze in the Raine Study (N = 2185) in children at 5 years of age (AUROC, 0.82; 95% CI, 0.79-0.86) and CAPPS (N = 349) at 7 years of age (AUROC, 0.87; 95% CI, 0.80-0.94).
In this diagnostic study, CHART was able to identify children at high risk of asthma at as early as 3 years of age. CHART could be easily incorporated as a routine screening tool in primary care to identify children who need monitoring, timely symptom control, and introduction of preventive therapies.
重要性：尽管哮喘治疗取得了进展，但学龄前儿童的负担仍然最高；因此，确定可用于识别负担沉重疾病风险较高的学龄前儿童的初级保健工具至关重要，以便进一步评估。目前的哮喘预测工具，如改良哮喘预测指数 (mAPI)，需要进行侵入性测试，限制了其在初级保健和资源匮乏环境中的适用性。
目的：开发并评估一种基于症状的筛查工具，以检测患有哮喘、持续性喘息症状和医疗负担高的儿童。
设计、地点和参与者：本诊断研究的队列纳入了 2008 年 1 月 1 日至 2012 年 12 月 31 日期间儿童研究 (n=2511)、2012 年 1 月 1 日至 2012 年 12 月 31 日期间的雷因研究 (n=2185) 和 1989 年 1 月 1 日至 1995 年 12 月 31 日期间的加拿大哮喘初级预防研究 (CAPPS) (n=349) 的参与者，目前仍在积极随访。数据分析于 2019 年 11 月 1 日至 2022 年 5 月 31 日进行。
暴露：在 3 岁时，儿童哮喘风险工具 (CHART) 确定了与哮喘相关的因素（喘息或咳嗽发作的时间和次数、哮喘药物的使用、因哮喘或喘息而就诊急诊或住院的次数），以识别出 5 岁时患有哮喘或持续性症状的儿童。
主要结果和措施：在儿童研究队列中，CHART 与专科临床医生的诊断和 mAPI 进行了评估。在澳大利亚雷因研究（一般人群队列）和加拿大 CAPPS（高风险队列）中进行了外部验证。通过敏感性、特异性、接受者操作特征曲线下面积 (AUROC)、阳性和阴性预测值来衡量预测准确性。
结果：在 2511 名（3 岁诊所就诊时的平均[标准差]年龄，3.08[0.17]岁；男性 1324 名[52.7%]；2476 名中有 1608 名[64.9%]为白人）有足够问卷数据可在 3 岁时应用 CHART 的儿童中，有 2354 名（93.7%）在 5 岁时有可用的结局数据。在儿童研究中，3 岁时应用的 CHART 在预测持续性喘息 (AUROC，0.94；95%CI，0.90-0.97)、哮喘诊断 (AUROC，0.73；95%CI，0.69-0.77) 和医疗保健使用（因喘息或哮喘就诊急诊或住院）(AUROC，0.70；95%CI，0.61-0.78) 方面优于医生评估和 mAPI。CHART 在雷因研究（5 岁儿童，N=2185）和 CAPPS（7 岁儿童，N=349）中对持续性喘息的预测性能相似。
结论和相关性：在这项诊断研究中，CHART 能够在儿童 3 岁时识别出患有哮喘高风险的儿童。CHART 可以很容易地纳入初级保健作为常规筛查工具，以识别需要监测、及时控制症状和引入预防治疗的儿童。
