TY - JOUR T1 - Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo JF - ERJ Open Research JO - erjor DO - 10.1183/23120541.00143-2021 SP - 00143-2021 AU - Elisangela Santos-Valente AU - Heike Buntrock-Döpke AU - Rola Abou Taam AU - Stefania Arasi AU - Arzu Bakirtas AU - Jaime Lozano Blasco AU - Klaus Bønnelykke AU - Mihai Craiu AU - Renato Cutrera AU - Antonine Deschildre AU - Basil Elnazir AU - Louise Fleming AU - Urs Frey AU - Monika Gappa AU - Antonio Nieto Garcia AU - Kirsten Skamstrup Hansen AU - Laurence Hanssens AU - Karina Jahnz-Rozyk AU - Milos Jesenak AU - Sebastian Kerzel AU - Matthias Kopp AU - Gerard H. Koppelman AU - Uros Krivec AU - Kenneth A. MacLeod AU - Mika Mäkelä AU - Erik Melén AU - Györgyi Mezei AU - Alexander Moeller AU - Andre Moreira AU - Petr Pohunek AU - Predrag Minić AU - Niels W. P. Rutjes AU - Patrick Sammut AU - Nicolaus Schwerk AU - Zsolt Szépfalusi AU - Mirjana Turkalj AU - Iren Tzotcheva AU - Alexandru Ulmeanu AU - Stijn Verhulst AU - Paraskevi Xepapadaki AU - Jakob Niggel AU - Susanne Vijverberg AU - Anke-Hilse Maitland van der Zee AU - Uros Potocnik AU - Susanne Reinartz AU - Kees van Drunen AU - Michael Kabesch Y1 - 2021/01/01 UR - http://openres.ersjournals.com/content/early/2021/04/15/23120541.00143-2021.abstract N2 - Introduction Severe asthma is a rare disease in children, for which three biologicals –anti-IgE, anti-IL-5 and anti-IL-4RA 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 status quo and identify open questions in biological therapy of childhood asthma across Europe.Methods 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.Results 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 timepoint when 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 a priori to available biologicals.Conclusion 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.FootnotesThis manuscript has recently been accepted for publication in the ERJ Open Research. It is published here in its accepted form prior to copyediting and typesetting by our production team. After these production processes are complete and the authors have approved the resulting proofs, the article will move to the latest issue of the ERJOR online. Please open or download the PDF to view this article.Conflict of interest: Dr. Santos-Valente has nothing to disclose.Conflict of interest: Dr. Buntrock-Döpke has nothing to disclose.Conflict of interest: Dr. Abou Taam has nothing to disclose.Conflict of interest: Dr. Arasi has nothing to disclose.Conflict of interest: Dr. BAKIRTAS has nothing to disclose.Conflict of interest: Dr. Lozano Blasco reports personal fees from Novartis, personal fees from GSK, outside the submitted work;.Conflict of interest: Dr. Bønnelykke has nothing to disclose.Conflict of interest: M. Craiu reports a speaker fee for a scientific meeting regarding 5 years of Xolair in Romania (9 May 2018) from Novartis Romania outside the submitted work.Conflict of interest: Dr. Cutrera has nothing to disclose.Conflict of interest: Dr. Deschildre reports personal fees and other from Novartis, GSK, Sanofi, outside the submitted work.Conflict of interest: Dr. Elnazir has nothing to disclose.Conflict of interest: Dr. Fleming reports grants from Asthma UK, and speakers fees or fees for expert consultation from Teva, Astra Zeneca, Sanofi, Respiri, Novartis; all fees paid direct to her institution and outside the submitted work.Conflict of interest: Dr. Frey has nothing to disclose.Conflict of interest: Dr. Gappa reports personal fees from Boehringer Ingelheim, personal fees from GSK, personal fees from Novartis, personal fees from Sanofi, outside the submitted work; .Conflict of interest: Dr. NIETO GARCÍA reports and I received grants for clinical studies and advisory boards membership, as well as lecture fees from Novartis, GSK and MSD.Conflict of interest: Dr. Skamstrup Hansen has nothing to disclose.Conflict of interest: Dr. Hanssens has nothing to disclose.Conflict of interest: Dr. Jahnz-Rozyk has nothing to disclose.Conflict of interest: Dr. Jesenak has nothing to disclose.Conflict of interest: S. Kerzel reports a speaker fee from Novartis for a lecture that has no topical overlap at all with the current manuscript.Conflict of interest: Dr. Kopp reports personal fees from ALK-Abello GmbH, grants and personal fees from Allergopharma GmbH, personal fees from Chiesi GmbH, personal fees from Infectopharm GmbH, personal fees from Novartis GmbH, personal fees from Sanofi Aventis GmbH, personal fees from Vertex GmbH, outside the submitted work;.Conflict of interest: Dr. Koppelman reports grants from Lung Foundation Netherlands, TETRI Foudation, Ubbo Emmius Foundation, Vertex, Teva the Netherlands, GSK, European Union, outside the submitted work; and he participated in advisory board meetings to GSK and Pure IMS (Money to institution).Conflict of interest: Dr. Krivec has nothing to disclose.Conflict of interest: Dr. MacLeod has nothing to disclose.Conflict of interest: Dr. Mäkelä has nothing to disclose.Conflict of interest: Dr. Melén reports personal fees from AstraZeneca, Chiesi, Novartis and Sanofi (Advisory board fees), outside the submitted work;.Conflict of interest: Dr. Mezei reports non financial support from LOFARMA, outside the submitted work; travel grant.Conflict of interest: Dr. Möller has nothing to disclose.Conflict of interest: Dr. Moreira has nothing to disclose.Conflict of interest: P. Pohunek reports personal fees for advisory board membership from Novartis and GlaxoSmithKline, and for consultation from Chiesi and AstraZeneca, outside the submitted work.Conflict of interest: Dr. Minić has nothing to disclose.Conflict of interest: Dr. Rutjes reports personal fees from GSK (Advisory board on mepolizumab), personal fees from Sanofi (Advisory board on dupilumab), outside the submitted work;.Conflict of interest: Dr. Sammut has nothing to disclose.Conflict of interest: N. Schwerk reports lecture and advisory board fees from Novartis, Sanofi and Allergopharma; advisory board fees from Boehringer Ingelheim; and lecture fees from Abvie and Infectopharm, all outside the submitted work.Conflict of interest: Dr. Szépfalusi has nothing to disclose.Conflict of interest: Dr. Turkalj has nothing to disclose.Conflict of interest: Dr. Tzotcheva has nothing to disclose.Conflict of interest: Dr. Ulmeanu has nothing to disclose.Conflict of interest: Dr. Verhulst reports grants from GSK, during the conduct of the study;.Conflict of interest: P. Xepapadaki reports personal fees for advisory services from Uriach, Novartis, Nestle and Nutricia outside the submitted work.Conflict of interest: Jakob Niggel has nothing to disclose.Conflict of interest: Dr. Vijverberg reports grants from ZonMW, during the conduct of the study;.Conflict of interest: AHM has received research grants outside the submitted work from GSK, Boehringer Íngelheim and Vertex, she is the PI of a P4O2 (Precision Medicine for more Oxygen) public private partnership sponsored by Health Holland involving many private partners that contribute in cash and/or in kind (Boehringer Ingelheim, Breathomix, Fluidda, Ortec Logiqcare, Philips, Quantib-U, Smartfish, SODAQ, Thirona, TopMD and Novartis), and she has served in advisory boards for AstraZeneca, GSK and Boehringer Ingelheim with money paid to her institution.Conflict of interest: U. Potocnik was funded by the Ministry of Education, Science and Sport of the Republic of Slovenia, grant PERMEABLE (contract number C3330-19-252012), during the conduct of the study.Conflict of interest: Dr. Reinartz has nothing to disclose.Conflict of interest: Dr. van Drunen has nothing to disclose.Conflict of interest: ER -