难治性哮喘的特征概况:临床影响和对系统评估的反应
2023/09/21
摘要
背景：多学科系统评估可改善难治性哮喘的预后，但缺乏明确的反应预测因素。使用可治疗的特征框架，我们根据特征概况对患者进行分层，检查临床影响和对系统评估的治疗反应。
方法：我们对在我院接受系统评估的难治性哮喘患者进行了12个特征的潜在分类分析。我们在基线和系统评估后检查了哮喘控制问卷(ACQ-6)和哮喘生活质量问卷(AQLQ)评分、FEV1、恶化频率和维持口服皮质类固醇(mOCS)剂量。
结果：在241例患者中，两种以气道为中心的特征分别为早发性变应性鼻炎(n=46)和成年期嗜酸性粒细胞增加/慢性鼻窦炎(n=60)，并伴有最小的合并症或社会心理特征;三个非气道中心型表现出共病(肥胖、声带功能障碍、呼吸功能障碍)主导(n=51)、社会心理(焦虑、抑郁、吸烟、失业)主导(n=72)或多域损伤(n=12)。与气道中心型相比，非气道中心型的基线ACQ-6 (2.7 vs 2.2, p＜0.001)和AQLQ (3.8 vs 4.5, p＜0.001)分数更差。经过系统评估，该队列显示所有结果的总体改善。然而，以气道为中心的方案有更大的FEV1改善(5.6% vs.预计2.2%，p＜0.05)，而非以气道为中心的方案有更大的加重减少趋势(1.7 vs 1.0, p=0.07);mOCS减量相似(3.1 mg vs 3.5 mg, p=0.782)。
结论：难治性哮喘的不同特征与不同的临床结果和对系统评估的治疗反应有关。这些发现为难治性哮喘提供了临床和机制方面的见解，为解决疾病异质性提供了一个概念框架，并突出了对靶向干预有反应的领域。
Trait profiles in difficult-to-treat asthma: Clinical impact and response to systematic assessment
T. Lin, J. Pham, E. Denton, J. Lee, F. Hore-Lacy, A. Sverrild, et al.
Abstract
BACKGROUND: Multidisciplinary systematic assessment improves outcomes in difficult-to-treat asthma, but without clear response predictors. Using a treatable-traits framework, we stratified patients by trait profile, examining clinical impact and treatment responsiveness to systematic assessment.
METHODS：We performed latent class analysis using 12 traits on difficult-to-treat asthma patients undergoing systematic assessment at our institution. We examined Asthma Control Questionnaire (ACQ-6) and Asthma Quality of Life Questionnaire (AQLQ) scores, FEV1, exacerbation frequency, and maintenance oral corticosteroid (mOCS) dose, at baseline and following systematic assessment.
RESULTS:Among 241 patients, two airway-centric profiles were characterized by early-onset with allergic rhinitis (n=46) and adult onset with eosinophilia/chronic rhinosinusitis (n=60), respectively, with minimal comorbid or psychosocial traits; three non-airway-centric profiles exhibited either comorbid (obesity, vocal cord dysfunction, dysfunctional breathing) dominance(n=51), psychosocial (anxiety, depression, smoking, unemployment) dominance (n=72), or multi-domain impairment (n=12). Compared to airway-centric profiles, non-airway-centric profiles had worse baseline ACQ-6 (2.7 vs. 2.2, p < 0.001) and AQLQ (3.8 vs. 4.5, p < 0.001) scores. Following systematic assessment, the cohort showed overall improvements across all outcomes. However, airway-centric profiles had more FEV1 improvement (5.6% vs. 2.2% predicted, p < 0.05) while non-airway-centric profiles trended to greater exacerbation reduction (1.7 vs. 1.0, p = 0.07); mOCS dose reduction was similar (3.1 mg vs. 3.5 mg, p = 0.782).
CONCLUSIONS:Distinct trait profiles in difficult-to-treat asthma are associated with different clinical outcomes and treatment responsiveness to systematic assessment. These findings yield clinical and mechanistic insights into difficult-to-treat asthma, offer a conceptual framework to address disease heterogeneity, and highlight areas responsive to targeted intervention.
背景：多学科系统评估可改善难治性哮喘的预后，但缺乏明确的反应预测因素。使用可治疗的特征框架，我们根据特征概况对患者进行分层，检查临床影响和对系统评估的治疗反应。
方法：我们对在我院接受系统评估的难治性哮喘患者进行了12个特征的潜在分类分析。我们在基线和系统评估后检查了哮喘控制问卷(ACQ-6)和哮喘生活质量问卷(AQLQ)评分、FEV1、恶化频率和维持口服皮质类固醇(mOCS)剂量。
结果：在241例患者中，两种以气道为中心的特征分别为早发性变应性鼻炎(n=46)和成年期嗜酸性粒细胞增加/慢性鼻窦炎(n=60)，并伴有最小的合并症或社会心理特征;三个非气道中心型表现出共病(肥胖、声带功能障碍、呼吸功能障碍)主导(n=51)、社会心理(焦虑、抑郁、吸烟、失业)主导(n=72)或多域损伤(n=12)。与气道中心型相比，非气道中心型的基线ACQ-6 (2.7 vs 2.2, p＜0.001)和AQLQ (3.8 vs 4.5, p＜0.001)分数更差。经过系统评估，该队列显示所有结果的总体改善。然而，以气道为中心的方案有更大的FEV1改善(5.6% vs.预计2.2%，p＜0.05)，而非以气道为中心的方案有更大的加重减少趋势(1.7 vs 1.0, p=0.07);mOCS减量相似(3.1 mg vs 3.5 mg, p=0.782)。
结论：难治性哮喘的不同特征与不同的临床结果和对系统评估的治疗反应有关。这些发现为难治性哮喘提供了临床和机制方面的见解，为解决疾病异质性提供了一个概念框架，并突出了对靶向干预有反应的领域。
（中日友好医院呼吸与危重症医学科 李春晓 摘译 林江涛 审校）
（Allergy 2023 DOI: 10.1111/all.15719）
（Allergy 2023 DOI: 10.1111/all.15719）
Trait profiles in difficult-to-treat asthma: Clinical impact and response to systematic assessment
T. Lin, J. Pham, E. Denton, J. Lee, F. Hore-Lacy, A. Sverrild, et al.
Abstract
BACKGROUND: Multidisciplinary systematic assessment improves outcomes in difficult-to-treat asthma, but without clear response predictors. Using a treatable-traits framework, we stratified patients by trait profile, examining clinical impact and treatment responsiveness to systematic assessment.
METHODS：We performed latent class analysis using 12 traits on difficult-to-treat asthma patients undergoing systematic assessment at our institution. We examined Asthma Control Questionnaire (ACQ-6) and Asthma Quality of Life Questionnaire (AQLQ) scores, FEV1, exacerbation frequency, and maintenance oral corticosteroid (mOCS) dose, at baseline and following systematic assessment.
RESULTS:Among 241 patients, two airway-centric profiles were characterized by early-onset with allergic rhinitis (n=46) and adult onset with eosinophilia/chronic rhinosinusitis (n=60), respectively, with minimal comorbid or psychosocial traits; three non-airway-centric profiles exhibited either comorbid (obesity, vocal cord dysfunction, dysfunctional breathing) dominance(n=51), psychosocial (anxiety, depression, smoking, unemployment) dominance (n=72), or multi-domain impairment (n=12). Compared to airway-centric profiles, non-airway-centric profiles had worse baseline ACQ-6 (2.7 vs. 2.2, p < 0.001) and AQLQ (3.8 vs. 4.5, p < 0.001) scores. Following systematic assessment, the cohort showed overall improvements across all outcomes. However, airway-centric profiles had more FEV1 improvement (5.6% vs. 2.2% predicted, p < 0.05) while non-airway-centric profiles trended to greater exacerbation reduction (1.7 vs. 1.0, p = 0.07); mOCS dose reduction was similar (3.1 mg vs. 3.5 mg, p = 0.782).
CONCLUSIONS:Distinct trait profiles in difficult-to-treat asthma are associated with different clinical outcomes and treatment responsiveness to systematic assessment. These findings yield clinical and mechanistic insights into difficult-to-treat asthma, offer a conceptual framework to address disease heterogeneity, and highlight areas responsive to targeted intervention.
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