Santos-Valente Elisangela, Buntrock-Döpke Heike, Abou Taam Rola, Arasi Stefania, Bakirtas Arzu, Lozano Blasco Jaime, Bønnelykke Klaus, Craiu Mihai, Cutrera Renato, Deschildre Antoine, Elnazir Basil, Fleming Louise, Frey Urs, Gappa Monika, Nieto García Antonio, Skamstrup Hansen Kirsten, Hanssens Laurence, Jahnz-Rozyk Karina, Jesenak Milos, Kerzel Sebastian, Kopp Matthias V, Koppelman Gerard H, Krivec Uros, MacLeod Kenneth A, Mäkelä Mika, Melén Erik, Mezei Györgyi, Moeller Alexander, Moreira Andre, Pohunek Petr, Minić Predrag, Rutjes Niels W P, Sammut Patrick, Schwerk Nicolaus, Szépfalusi Zsolt, Turkalj Mirjana, Tzotcheva Iren, Ulmeanu Alexandru, Verhulst Stijn, Xepapadaki Paraskevi, Niggel Jakob, Vijverberg Susanne, Maitland-van der Zee Anke H, Potočnik Uroš, Reinartz Susanne M, van Drunen Cornelis M, Kabesch Michael
Dept of Pediatric Pneumology and Allergy, University Children's Hospital Regensburg (KUNO) at the Hospital St Hedwig of the Order of St John, University of Regensburg, Regensburg, Germany.
Member of the Research and Development Campus Regensburg (WECARE) at the Hospital St Hedwig of the Order of St John, Regensburg, Germany.
ERJ Open Res. 2021 Aug 16;7(3). doi: 10.1183/23120541.00143-2021. eCollection 2021 Jul.
Severe asthma is a rare disease in children, for which three biologicals, anti-immunoglobulin E, anti-interleukin-5 and anti-IL4RA antibodies, are available in European countries. While global guidelines exist on who should receive biologicals, knowledge is lacking on how those guidelines are implemented in real life and which unmet needs exist in the field. In this survey, we aimed to investigate the and identify open questions in biological therapy of childhood asthma across Europe.
Structured interviews regarding experience with biologicals, regulations on access to the different treatment options, drug selection, therapy success and discontinuation of therapy were performed. Content analysis was used to analyse data.
We interviewed 37 experts from 25 European countries and Turkey and found a considerable range in the number of children treated with biologicals per centre. All participating countries provide public access to at least one biological. Most countries allow different medical disciplines to prescribe biologicals to children with asthma, and only a few restrict therapy to specialised centres. We observed significant variation in the time point at which treatment success is assessed, in therapy duration and in the success rate of discontinuation. Most participating centres intend to apply a personalised medicine approach in the future to match patients to available biologicals.
Substantial differences exist in the management of childhood severe asthma across Europe, and the need for further studies on biomarkers supporting selection of biologicals, on criteria to assess therapy response and on how/when to end therapy in stable patients is evident.
重度哮喘在儿童中是一种罕见疾病，在欧洲国家有三种生物制剂可供使用，即抗免疫球蛋白E、抗白细胞介素-5和抗IL4RA抗体。虽然有关于哪些人应接受生物制剂治疗的全球指南，但对于这些指南在现实生活中如何实施以及该领域存在哪些未满足的需求，人们却缺乏了解。在这项调查中，我们旨在研究欧洲儿童哮喘生物治疗的现状并确定其中存在的未决问题。
就生物制剂的使用经验、不同治疗选择的准入规定、药物选择、治疗效果及治疗中断情况进行了结构化访谈。采用内容分析法对数据进行分析。
我们采访了来自25个欧洲国家和土耳其的37位专家，发现每个中心接受生物制剂治疗的儿童数量差异很大。所有参与国家都为至少一种生物制剂提供了公共准入途径。大多数国家允许不同医学学科为哮喘儿童开具生物制剂，只有少数国家将治疗限制在专科中心。我们观察到在评估治疗效果的时间点、治疗持续时间以及治疗中断成功率方面存在显著差异。大多数参与中心打算在未来采用个性化医疗方法，以使患者与可用的生物制剂相匹配。
欧洲儿童重度哮喘的管理存在很大差异，显然需要进一步研究支持生物制剂选择的生物标志物、评估治疗反应的标准以及如何/何时终止稳定患者的治疗。
