重度哮喘患儿Mepolizumab的适格性——我们是否需要儿科特定标准?

B. Davies, Deevena Chinthala, S. Frost, Isobel Brookes, P. Kenia, S. Rao, P. Nagakumar
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引用次数: 2

摘要

背景:6岁以上的儿童,尽管有最大限度的处方治疗,但哮喘控制不良,有问题的严重哮喘。详细的多学科团队(MDT)评估是确定可改变因素和考虑许可生物疗法(Omalizumab, Mepolizumab)的关键。我们审查了英国一家地区儿科严重哮喘中心的生物制剂资格。方法:2015-2018年间,67例GINA指南治疗步骤4-5的患者接受了基于协议的医院、学校和家庭MDT哮喘评估。采用电子监测装置(EMD)评估药物依从性。进行肺活量测定、呼气一氧化氮分数(FeNO)、全血细胞计数、IgE和气敏原致敏试验。结果:评估时的中位年龄为10.9岁(范围6.0-15.8岁)。男:女:40:27。中位吸入类固醇剂量为1000mcg倍氯米松当量(范围400-2000mcg)。44例(66%)长期口服类固醇。预测FEV1中位数为81%(范围45-115%)。中位FeNO为37ppb(范围7-210ppb)。42例(63%)接受了EMD依从性评估。35例(52%)患者符合使用Omalizumab的条件;54例(80%)符合Mepolizumab治疗条件;27例(40%)同时符合这两种条件。在符合Mepolizumab治疗条件的患者中,FeNO与外周嗜酸性粒细胞计数没有相关性(p=0.23)。结论:令人惊讶的是,我们的队列中80%的儿童符合Mepolizumab的标准。包括改善药物依从性在内的干预措施对外周嗜酸性粒细胞计数的影响尚不清楚。由于资源的影响,应该探索确定儿科特异性资格标准和预测Mepolizumab反应的生物标志物的可靠研究。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Mepolizumab eligibility in children with severe asthma – do we need paediatric specific criteria?
Background: Children > 6yr with poor asthma control despite maximally prescribed treatment have Problematic Severe Asthma. Detailed multidisciplinary team (MDT) assessment is key to phenotype children for identification of modifiable factors and consideration of licensed biological therapies (Omalizumab, Mepolizumab). We reviewed eligibility for biologics in a regional paediatric severe asthma centre in the UK. Methods: 67 patients on Steps 4-5 of GINA guideline therapies underwent protocol-based hospital, school and home MDT asthma assessment between 2015-2018. Medication adherence was assessed by electronic monitoring device (EMD). Spirometry, fractional exhaled nitric oxide (FeNO), blood tests for full blood count, IgE and aeroallergen sensitisation were performed. Results: Median age at assessment was 10.9 yr (range 6.0-15.8 yr). Male:Female - 40:27. Median inhaled steroid dose was 1000mcg Beclomethasone equivalent (range 400-2000mcg). 44 (66%) were on long-term oral steroids. Median FEV1 was 81% predicted (range 45-115%). Median FeNO was 37ppb (range 7-210ppb). 42 (63%) had adherence assessed by EMD. 35 (52%) were eligible for Omalizumab; 54 (80%) were eligible for Mepolizumab; 27 (40%) were eligible for both. There was no correlation between FeNO and peripheral eosinophil count in patients eligible for Mepolizumab (p=0.23). Conclusions: Surprisingly 80% children in our cohort fulfilled the criteria for Mepolizumab. The effect of interventions including improved medication adherence on peripheral eosinophil count is unknown. Due to resource implications, robust studies to identify paediatric-specific eligibility criteria and biomarkers predicting response to Mepolizumab should be explored.
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