Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo
Autor(a) principal: | |
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Data de Publicação: | 2021 |
Outros Autores: | , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , , |
Tipo de documento: | Artigo |
Idioma: | eng |
Título da fonte: | Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) |
Texto Completo: | https://hdl.handle.net/10216/149510 |
Resumo: | Introduction: 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 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 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 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. |
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Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quoIntroduction: 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 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 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 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.European Respiratory Society20212021-01-01T00:00:00Zinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/articleapplication/pdfhttps://hdl.handle.net/10216/149510eng2312-054110.1183/23120541.00143-2021Santos-Valente, EBuntrock-Döpke, HAbou Taam, RArasi, SBakirtas, ALozano Blasco, JBønnelykke, KCraiu, MCutrera, RDeschildre, AElnazir, BFleming, LFrey, UGappa, MNieto García, ASkamstrup Hansen, KHanssens, LJahnz-Rozyk, KJesenak, MKerzel, SKopp, MVKoppelman, GHKrivec, UMacLeod, KAMäkelä, MMelén, EMezei, GMoeller, AMoreira, APohunek, PMinić, PRutjes, NWPSammut, PSchwerk, NSzépfalusi, ZTurkalj, MTzotcheva, IUlmeanu, AVerhulst, SXepapadaki, PNiggel, JVijverberg, SMaitland-van der Zee, AHPotočnik, UReinartz, SMvan Drunen, CMKabesch, Minfo:eu-repo/semantics/openAccessreponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos)instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãoinstacron:RCAAP2023-11-29T14:56:14Zoai:repositorio-aberto.up.pt:10216/149510Portal AgregadorONGhttps://www.rcaap.pt/oai/openaireopendoar:71602024-03-20T00:11:58.771657Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informaçãofalse |
dc.title.none.fl_str_mv |
Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo |
title |
Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo |
spellingShingle |
Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo Santos-Valente, E |
title_short |
Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo |
title_full |
Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo |
title_fullStr |
Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo |
title_full_unstemmed |
Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo |
title_sort |
Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo |
author |
Santos-Valente, E |
author_facet |
Santos-Valente, E Buntrock-Döpke, H Abou Taam, R Arasi, S Bakirtas, A Lozano Blasco, J Bønnelykke, K Craiu, M Cutrera, R Deschildre, A Elnazir, B Fleming, L Frey, U Gappa, M Nieto García, A Skamstrup Hansen, K Hanssens, L Jahnz-Rozyk, K Jesenak, M Kerzel, S Kopp, MV Koppelman, GH Krivec, U MacLeod, KA Mäkelä, M Melén, E Mezei, G Moeller, A Moreira, A Pohunek, P Minić, P Rutjes, NWP Sammut, P Schwerk, N Szépfalusi, Z Turkalj, M Tzotcheva, I Ulmeanu, A Verhulst, S Xepapadaki, P Niggel, J Vijverberg, S Maitland-van der Zee, AH Potočnik, U Reinartz, SM van Drunen, CM Kabesch, M |
author_role |
author |
author2 |
Buntrock-Döpke, H Abou Taam, R Arasi, S Bakirtas, A Lozano Blasco, J Bønnelykke, K Craiu, M Cutrera, R Deschildre, A Elnazir, B Fleming, L Frey, U Gappa, M Nieto García, A Skamstrup Hansen, K Hanssens, L Jahnz-Rozyk, K Jesenak, M Kerzel, S Kopp, MV Koppelman, GH Krivec, U MacLeod, KA Mäkelä, M Melén, E Mezei, G Moeller, A Moreira, A Pohunek, P Minić, P Rutjes, NWP Sammut, P Schwerk, N Szépfalusi, Z Turkalj, M Tzotcheva, I Ulmeanu, A Verhulst, S Xepapadaki, P Niggel, J Vijverberg, S Maitland-van der Zee, AH Potočnik, U Reinartz, SM van Drunen, CM Kabesch, M |
author2_role |
author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author author |
dc.contributor.author.fl_str_mv |
Santos-Valente, E Buntrock-Döpke, H Abou Taam, R Arasi, S Bakirtas, A Lozano Blasco, J Bønnelykke, K Craiu, M Cutrera, R Deschildre, A Elnazir, B Fleming, L Frey, U Gappa, M Nieto García, A Skamstrup Hansen, K Hanssens, L Jahnz-Rozyk, K Jesenak, M Kerzel, S Kopp, MV Koppelman, GH Krivec, U MacLeod, KA Mäkelä, M Melén, E Mezei, G Moeller, A Moreira, A Pohunek, P Minić, P Rutjes, NWP Sammut, P Schwerk, N Szépfalusi, Z Turkalj, M Tzotcheva, I Ulmeanu, A Verhulst, S Xepapadaki, P Niggel, J Vijverberg, S Maitland-van der Zee, AH Potočnik, U Reinartz, SM van Drunen, CM Kabesch, M |
description |
Introduction: 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 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 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 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. |
publishDate |
2021 |
dc.date.none.fl_str_mv |
2021 2021-01-01T00:00:00Z |
dc.type.status.fl_str_mv |
info:eu-repo/semantics/publishedVersion |
dc.type.driver.fl_str_mv |
info:eu-repo/semantics/article |
format |
article |
status_str |
publishedVersion |
dc.identifier.uri.fl_str_mv |
https://hdl.handle.net/10216/149510 |
url |
https://hdl.handle.net/10216/149510 |
dc.language.iso.fl_str_mv |
eng |
language |
eng |
dc.relation.none.fl_str_mv |
2312-0541 10.1183/23120541.00143-2021 |
dc.rights.driver.fl_str_mv |
info:eu-repo/semantics/openAccess |
eu_rights_str_mv |
openAccess |
dc.format.none.fl_str_mv |
application/pdf |
dc.publisher.none.fl_str_mv |
European Respiratory Society |
publisher.none.fl_str_mv |
European Respiratory Society |
dc.source.none.fl_str_mv |
reponame:Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) instname:Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação instacron:RCAAP |
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Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação |
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RCAAP |
institution |
RCAAP |
reponame_str |
Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) |
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Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) |
repository.name.fl_str_mv |
Repositório Científico de Acesso Aberto de Portugal (Repositórios Cientìficos) - Agência para a Sociedade do Conhecimento (UMIC) - FCT - Sociedade da Informação |
repository.mail.fl_str_mv |
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1799136042585948161 |