奥马珠单抗治疗重症持续性过敏性哮喘患儿长达6年的疗效
2021/03/22
摘要
背景：各种研究评估了奥马珠单抗在临床实践中的疗效，但随访和/或纳入的患者数量有限。我们的目的是描述在迄今为止报道的最大的接受奥马珠单抗治疗的真实队列中重症持续性过敏性哮喘患儿的长期结果。
方法：ANCHORS是一项多中心、观察性、回顾性队列研究，在西班牙的25个儿童过敏和肺科病房进行。我们收集了2006-2018年间，从奥马珠单抗开始治疗前一年到停止治疗或最后一次随访期间，年龄<18岁且开始使用奥马珠单抗的患者的数据。主要结果是与基线期相比，中重度急性加重的年次的演变。
结果：484例患者中，101例（20.9%）达6年治疗时长。在治疗的第一年，急性加重平均±标准差次数减少（7.9±6.6到1.1±2.0，p<0.001），并且在长达六年的时间里保持不变。评估的其他临床参数在第一年内也有显著改善，此后趋于稳定或继续改善。发生不良事件的患者比例一直很低，停药的主要原因是疾病进展良好。
结论：在这项大型、长期、观察性研究中，从奥马珠单抗治疗的第一年开始，中度至重度的病情恶化显著减少。长期疗效良好，安全性良好。我们的研究结果将奥马珠单抗定位为重症持续性过敏性哮喘患儿的有效长期的治疗方法。
Omalizumab outcomes for up to 6 years in pediatric patients with severe persistent allergic asthma
Antonio Nieto García, Teresa Garriga-Baraut, Ana Mª Plaza Martín, María Nieto Cid 1, Javier Torres Borrego, María Del Mar Folqué Giménez, Jaime Lozano Blasco, Montse Bosque García, Laura Moreno-Galarraga, Miguel Tortajada-Girbés, Cristina Rivas Juesas, María Penín Antón, María Araceli Caballero-Rabasco, Mirella Gaboli, Alejandro López Neyra, Juan Navarro Morón, Andrea Freixa, Laura Valdesoiro Navarrete, Esther Ballester Asensio, Verónica Sanz Santiago, Raquel Romero García, Álvaro Gimeno Díaz de Atauri, Alfredo Valenzuela Soria, Mercedes Sánchez Mateos, José Batlles Garrido, Anselmo Andrés Martín, Elena Campos Alonso, Carmen Aragón Fernández, Elena Vázquez Rodríguez, Luz Martínez Pardo, Genoveva Del Río Camacho, Ángel Mazón Ramos
Abstract
Background: Various studies have assessed omalizumab outcomes in the clinical practice setting but follow-up and/or number of patients included were limited. We aim to describe the long-term outcomes of pediatric patients with severe persistent allergic asthma receiving omalizumab in the largest real-life cohort reported to date.
Methods: ANCHORS was a multicenter, observational, retrospective cohort study conducted in 25 Pediatric Allergy and Pulmonology units in Spain. We collected data of patients <18 years and initiating omalizumab between 2006-2018, from the year prior to omalizumab initiation to discontinuation or last available follow-up. The primary outcome was the evolution of the annual number of moderate-to-severe exacerbations compared to the baseline period.
Results: Of the 484 patients included, 101 (20.9%) reached six years of treatment. The mean±standard deviation number of exacerbations decreased during the first year of treatment (7.9±6.6 to 1.1±2.0, p<0.001) and remained likewise for up to six years. The other clinical parameters assessed also improved significantly during the first year and stabilized or continued to improve thereafter. The percentage of patients experiencing adverse events was consistently low, and the main reason for discontinuation was good disease evolution.
Conclusion: In this large, long-term, observational study, moderate-to-severe exacerbations decreased significantly from the first year of treatment with omalizumab. The beneficial effect was maintained in the long-term, along with a good safety profile. Our results position omalizumab as an effective long-term treatment in pediatric patients with severe persistent allergic asthma.
背景：各种研究评估了奥马珠单抗在临床实践中的疗效，但随访和/或纳入的患者数量有限。我们的目的是描述在迄今为止报道的最大的接受奥马珠单抗治疗的真实队列中重症持续性过敏性哮喘患儿的长期结果。
方法：ANCHORS是一项多中心、观察性、回顾性队列研究，在西班牙的25个儿童过敏和肺科病房进行。我们收集了2006-2018年间，从奥马珠单抗开始治疗前一年到停止治疗或最后一次随访期间，年龄<18岁且开始使用奥马珠单抗的患者的数据。主要结果是与基线期相比，中重度急性加重的年次的演变。
结果：484例患者中，101例（20.9%）达6年治疗时长。在治疗的第一年，急性加重平均±标准差次数减少（7.9±6.6到1.1±2.0，p<0.001），并且在长达六年的时间里保持不变。评估的其他临床参数在第一年内也有显著改善，此后趋于稳定或继续改善。发生不良事件的患者比例一直很低，停药的主要原因是疾病进展良好。
结论：在这项大型、长期、观察性研究中，从奥马珠单抗治疗的第一年开始，中度至重度的病情恶化显著减少。长期疗效良好，安全性良好。我们的研究结果将奥马珠单抗定位为重症持续性过敏性哮喘患儿的有效长期的治疗方法。
(中日友好医院呼吸与危重症医学科 李红雯 摘译 林江涛 审校)
(Pediatr Allergy Immunol. 2021 Feb 22. doi: 10.1111/pai.13484.)
(Pediatr Allergy Immunol. 2021 Feb 22. doi: 10.1111/pai.13484.)
Omalizumab outcomes for up to 6 years in pediatric patients with severe persistent allergic asthma
Antonio Nieto García, Teresa Garriga-Baraut, Ana Mª Plaza Martín, María Nieto Cid 1, Javier Torres Borrego, María Del Mar Folqué Giménez, Jaime Lozano Blasco, Montse Bosque García, Laura Moreno-Galarraga, Miguel Tortajada-Girbés, Cristina Rivas Juesas, María Penín Antón, María Araceli Caballero-Rabasco, Mirella Gaboli, Alejandro López Neyra, Juan Navarro Morón, Andrea Freixa, Laura Valdesoiro Navarrete, Esther Ballester Asensio, Verónica Sanz Santiago, Raquel Romero García, Álvaro Gimeno Díaz de Atauri, Alfredo Valenzuela Soria, Mercedes Sánchez Mateos, José Batlles Garrido, Anselmo Andrés Martín, Elena Campos Alonso, Carmen Aragón Fernández, Elena Vázquez Rodríguez, Luz Martínez Pardo, Genoveva Del Río Camacho, Ángel Mazón Ramos
Abstract
Background: Various studies have assessed omalizumab outcomes in the clinical practice setting but follow-up and/or number of patients included were limited. We aim to describe the long-term outcomes of pediatric patients with severe persistent allergic asthma receiving omalizumab in the largest real-life cohort reported to date.
Methods: ANCHORS was a multicenter, observational, retrospective cohort study conducted in 25 Pediatric Allergy and Pulmonology units in Spain. We collected data of patients <18 years and initiating omalizumab between 2006-2018, from the year prior to omalizumab initiation to discontinuation or last available follow-up. The primary outcome was the evolution of the annual number of moderate-to-severe exacerbations compared to the baseline period.
Results: Of the 484 patients included, 101 (20.9%) reached six years of treatment. The mean±standard deviation number of exacerbations decreased during the first year of treatment (7.9±6.6 to 1.1±2.0, p<0.001) and remained likewise for up to six years. The other clinical parameters assessed also improved significantly during the first year and stabilized or continued to improve thereafter. The percentage of patients experiencing adverse events was consistently low, and the main reason for discontinuation was good disease evolution.
Conclusion: In this large, long-term, observational study, moderate-to-severe exacerbations decreased significantly from the first year of treatment with omalizumab. The beneficial effect was maintained in the long-term, along with a good safety profile. Our results position omalizumab as an effective long-term treatment in pediatric patients with severe persistent allergic asthma.
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医生诊断的哮喘和/或慢性阻塞性肺病之间的内在异质性：NOVELTY队列
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