一项来自法国早期计划数据的研究-美伯利单抗在重症嗜酸性粒细胞型及激素依赖型哮喘患者中的应用
2020/07/13
摘要
背景：美伯利单抗在商业化前作为重症嗜酸性哮喘患者（nATU临时使用权）早期抢救计划的一部分已在法国上市。这项研究旨在描述在nATU中接受美泊利单抗治疗患者的临床特征。
方法：这项回顾性观察研究分析了患者初始治疗后长达24个月的医院病历数据。研究目的是描述患者的基线特征，疾病严重程度的变化以及随访期间的治疗方案的改变，同时进行安全性调查。
结果：研究共计146例接受≥1剂量美泊珠单抗治疗的患者入组。患者入组时平均年龄为58.2岁，重症哮喘平均病程为13.4年，37.0％患者有呼吸道过敏史。患者基线情况为平均每年每人经历5.8次急性发作，其中0.6和0.5次分别需要住院和急诊就诊。在随访的24个月中，每位患者每年的急性发作次数分别改善至0.6、0.1及0.1。大多数患者（92.8％）在基线时使用口服激素，而至随访24个月时为34.7％。此外，平均嗜酸粒细胞计数基线时为722细胞·μL-1，至随访24个月时为92细胞·μL-1，肺功能和哮喘控制情况也呈类似趋势。
解释：研究证实临床试验结果，表明美伯利单抗与重要的临床结局指标改善具有相关性，并且在临床试验可控的环境之外，对于重症嗜酸性粒细胞型哮喘人群具有良好的安全性。
Mepolizumab in a Population With Severe Eosinophilic Asthma and Corticosteroid Dependence: Results From a French Early Access Programme
Camille Taillé , Pascal Chanez , Gilles Devouassoux , Alain Didier , Christophe Pison , Gilles Garcia , Jeremy Charriot , Stéphane Bouée , Alina Gruber , Celine Pribil , Arnaud Bourdin , Marc Humbert
Abstract
Background:Mepolizumab was available in France as part of an early access programme for patients with severe eosinophilic asthma (nominative autorisation temporaire d'utilisation [temporary use authorisation] (nATU)) before its commercialisation. This study aimed to characterise patients who received mepolizumab in the nATU.
Methods: This retrospective, observational study analysed data from the hospital medical records of patients up to 24 months after treatment initiation. Study objectives were to describe patient baseline characteristics, the evolution of disease severity and treatment modifications during follow-up; safety was also investigated.
Findings: Overall, 146 patients who received ≥1 dose of mepolizumab were included. At inclusion, patients had a mean age of 58.2 years with a mean severe asthma duration of 13.4 years, and 37.0% had respiratory allergies. Patients experienced, on average, 5.8 exacerbations per patient per year at baseline, 0.6 and 0.5 of which required hospitalisation and emergency department visits, respectively. These values improved to 0.6, 0.1 and 0.1 exacerbations per patient per year, respectively, at 24 months of follow-up. Most patients (92.8%) were using oral corticosteroids at baseline, compared with 34.7% by 24 months of follow-up. Moreover, mean blood eosinophil counts improved from 722 cells·µL-1 at baseline to 92 cells·µL-1 at 24 months of follow-up; lung function and asthma control followed a similar trend.
Interpretation:Results confirm findings from clinical trials, demonstrating that mepolizumab is associated with important improvements in several clinically meaningful outcomes and has a favourable safety profile in a population with severe eosinophilic asthma, outside of the controlled environment of a clinical trial.
背景：美伯利单抗在商业化前作为重症嗜酸性哮喘患者（nATU临时使用权）早期抢救计划的一部分已在法国上市。这项研究旨在描述在nATU中接受美泊利单抗治疗患者的临床特征。
方法：这项回顾性观察研究分析了患者初始治疗后长达24个月的医院病历数据。研究目的是描述患者的基线特征，疾病严重程度的变化以及随访期间的治疗方案的改变，同时进行安全性调查。
结果：研究共计146例接受≥1剂量美泊珠单抗治疗的患者入组。患者入组时平均年龄为58.2岁，重症哮喘平均病程为13.4年，37.0％患者有呼吸道过敏史。患者基线情况为平均每年每人经历5.8次急性发作，其中0.6和0.5次分别需要住院和急诊就诊。在随访的24个月中，每位患者每年的急性发作次数分别改善至0.6、0.1及0.1。大多数患者（92.8％）在基线时使用口服激素，而至随访24个月时为34.7％。此外，平均嗜酸粒细胞计数基线时为722细胞·μL-1，至随访24个月时为92细胞·μL-1，肺功能和哮喘控制情况也呈类似趋势。
解释：研究证实临床试验结果，表明美伯利单抗与重要的临床结局指标改善具有相关性，并且在临床试验可控的环境之外，对于重症嗜酸性粒细胞型哮喘人群具有良好的安全性。
（中日友好医院呼吸与危重症医学科 张鑫 翻译 林江涛 审校）
（Eur Respir J. 2020 Jun 25;55(6):1902345.）
（Eur Respir J. 2020 Jun 25;55(6):1902345.）
Mepolizumab in a Population With Severe Eosinophilic Asthma and Corticosteroid Dependence: Results From a French Early Access Programme
Camille Taillé , Pascal Chanez , Gilles Devouassoux , Alain Didier , Christophe Pison , Gilles Garcia , Jeremy Charriot , Stéphane Bouée , Alina Gruber , Celine Pribil , Arnaud Bourdin , Marc Humbert
Abstract
Background:Mepolizumab was available in France as part of an early access programme for patients with severe eosinophilic asthma (nominative autorisation temporaire d'utilisation [temporary use authorisation] (nATU)) before its commercialisation. This study aimed to characterise patients who received mepolizumab in the nATU.
Methods: This retrospective, observational study analysed data from the hospital medical records of patients up to 24 months after treatment initiation. Study objectives were to describe patient baseline characteristics, the evolution of disease severity and treatment modifications during follow-up; safety was also investigated.
Findings: Overall, 146 patients who received ≥1 dose of mepolizumab were included. At inclusion, patients had a mean age of 58.2 years with a mean severe asthma duration of 13.4 years, and 37.0% had respiratory allergies. Patients experienced, on average, 5.8 exacerbations per patient per year at baseline, 0.6 and 0.5 of which required hospitalisation and emergency department visits, respectively. These values improved to 0.6, 0.1 and 0.1 exacerbations per patient per year, respectively, at 24 months of follow-up. Most patients (92.8%) were using oral corticosteroids at baseline, compared with 34.7% by 24 months of follow-up. Moreover, mean blood eosinophil counts improved from 722 cells·µL-1 at baseline to 92 cells·µL-1 at 24 months of follow-up; lung function and asthma control followed a similar trend.
Interpretation:Results confirm findings from clinical trials, demonstrating that mepolizumab is associated with important improvements in several clinically meaningful outcomes and has a favourable safety profile in a population with severe eosinophilic asthma, outside of the controlled environment of a clinical trial.
