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采用多领域评估方法评估儿童重症哮喘患者的皮质类固醇反应。

Assessment of corticosteroid response in pediatric patients with severe asthma by using a multidomain approach.

机构信息

Respiratory Paediatrics, Royal Brompton Hospital and National Heart & Lung Institute, Imperial College London, London, United Kingdom; Respiratory Paediatrics, Kings College Hospital, London, United Kingdom.

Respiratory Paediatrics, Royal Brompton Hospital and National Heart & Lung Institute, Imperial College London, London, United Kingdom; Leukocyte Biology, National Heart & Lung Institute, Imperial College London, London, United Kingdom.

出版信息

J Allergy Clin Immunol. 2016 Aug;138(2):413-420.e6. doi: 10.1016/j.jaci.2015.12.1347. Epub 2016 Apr 6.

Abstract

BACKGROUND

There is no agreed upon definition of systemic corticosteroid response in asthmatic children. Moreover, pediatric severe therapy-resistant asthma (STRA) is heterogeneous, and thus response to steroids is unlikely to be uniform in all patients.

OBJECTIVE

We sought to evaluate the utility of a multidomain approach incorporating symptoms, lung function, and inflammation to determine steroid responsiveness in pediatric patients with STRA.

METHODS

Eighty-two children (median age, 12 years) with STRA received a clinically indicated dose of intramuscular steroid. Changes in 4 separate domains were assessed 4 weeks after intramuscular triamcinolone acetonide: normalization of (1) symptoms (Asthma Control Test score, >19/25 or 50% increase), (2) spirometric results (FEV1 ≥80% of predicted value or ≥15% increase), (3) fraction of exhaled nitric oxide levels (<24 ppb), and (4) sputum eosinophil counts (<2.5%). Fifty-four of 82 children had complete data in all 4 domains.

RESULTS

Twenty-three (43%) of 54 children had a symptom response, 29 (54%) of 54 had a lung function response, 28 (52%) of 54 had a fraction of exhaled nitric oxide response, and 29 (54%) of 54 had a sputum eosinophil response. Although a similar proportion of children responded to systemic corticosteroids in each domain, there were no reliable predictors of a response pattern. Seven (13%) of 54 were complete responders (response in all domains), 8 (15%) of 54 were nonresponders (no response in any domain), and 39 (72%) of 54 were partial responders (response in ≥1 domain).

CONCLUSIONS

A multidomain evaluation of systemic steroid responsiveness using pragmatic clinical assessments confirms childhood STRA is heterogeneous and that a complete response in symptoms and inflammatory and physiologic parameters is rare. Individual response patterns to systemic steroids might be useful in guiding the choice of add-on therapies in each child as a step toward achieving personalized medicine.

摘要

背景

目前尚无公认的儿童哮喘全身皮质激素反应定义。此外,儿科重症治疗抵抗性哮喘(STRA)具有异质性,因此皮质激素反应不太可能在所有患者中都一致。

目的

我们旨在评估一种多领域方法,该方法结合症状、肺功能和炎症,以确定患有 STRA 的儿科患者的皮质激素反应性。

方法

82 名(中位年龄,12 岁)STRA 患儿接受了临床推荐剂量的肌内注射类固醇。在肌内注射曲安奈德 4 周后评估 4 个独立领域的变化:(1)症状正常(哮喘控制测试评分,>19/25 或增加 50%);(2)肺活量结果(FEV1≥预测值的 80%或增加 15%);(3)呼气一氧化氮分数正常(<24 ppb);(4)痰嗜酸性粒细胞计数正常(<2.5%)。54 名 82 名患儿中有 4 个领域的完整数据。

结果

54 名患儿中有 23 名(43%)出现症状反应,54 名中有 29 名(54%)出现肺功能反应,54 名中有 28 名(52%)出现呼气一氧化氮分数反应,54 名中有 29 名(54%)出现痰嗜酸性粒细胞计数反应。尽管每个领域都有相似比例的儿童对全身皮质激素有反应,但没有可靠的反应模式预测指标。54 名患儿中有 7 名(13%)为完全反应者(所有领域均有反应),8 名(15%)为无反应者(任何领域均无反应),39 名(72%)为部分反应者(≥1 个领域有反应)。

结论

使用实用的临床评估对全身皮质激素反应性进行多领域评估,证实儿童 STRA 具有异质性,症状和炎症及生理参数的完全反应很少见。全身皮质激素的个体反应模式可能有助于指导每个患儿选择附加治疗,以迈向个体化医学。

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