Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo
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 guidelin...
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| Format: | Article |
| Language: | English |
| Published: |
European Respiratory Society
2021-08-01
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| Series: | ERJ Open Research |
| Online Access: | http://openres.ersjournals.com/content/7/3/00143-2021.full |
| _version_ | 1818935453842145280 |
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| author | Elisangela Santos-Valente Heike Buntrock-Döpke Rola Abou Taam Stefania Arasi Arzu Bakirtas Jaime Lozano Blasco Klaus Bønnelykke Mihai Craiu Renato Cutrera Antoine Deschildre Basil Elnazir Louise Fleming Urs Frey Monika Gappa Antonio Nieto García Kirsten Skamstrup Hansen Laurence Hanssens Karina Jahnz-Rozyk Milos Jesenak Sebastian Kerzel Matthias V. Kopp Gerard H. Koppelman Uros Krivec Kenneth A. MacLeod Mika Mäkelä Erik Melén Györgyi Mezei Alexander Moeller Andre Moreira Petr Pohunek Predrag Minić Niels W.P. Rutjes Patrick Sammut Nicolaus Schwerk Zsolt Szépfalusi Mirjana Turkalj Iren Tzotcheva Alexandru Ulmeanu Stijn Verhulst Paraskevi Xepapadaki Jakob Niggel Susanne Vijverberg Anke H. Maitland-van der Zee Uroš Potočnik Susanne M. Reinartz Cornelis M. van Drunen Michael Kabesch |
| author_facet | Elisangela Santos-Valente Heike Buntrock-Döpke Rola Abou Taam Stefania Arasi Arzu Bakirtas Jaime Lozano Blasco Klaus Bønnelykke Mihai Craiu Renato Cutrera Antoine Deschildre Basil Elnazir Louise Fleming Urs Frey Monika Gappa Antonio Nieto García Kirsten Skamstrup Hansen Laurence Hanssens Karina Jahnz-Rozyk Milos Jesenak Sebastian Kerzel Matthias V. Kopp Gerard H. Koppelman Uros Krivec Kenneth A. MacLeod Mika Mäkelä Erik Melén Györgyi Mezei Alexander Moeller Andre Moreira Petr Pohunek Predrag Minić Niels W.P. Rutjes Patrick Sammut Nicolaus Schwerk Zsolt Szépfalusi Mirjana Turkalj Iren Tzotcheva Alexandru Ulmeanu Stijn Verhulst Paraskevi Xepapadaki Jakob Niggel Susanne Vijverberg Anke H. Maitland-van der Zee Uroš Potočnik Susanne M. Reinartz Cornelis M. van Drunen Michael Kabesch |
| author_sort | Elisangela Santos-Valente |
| collection | DOAJ |
| 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. |
| first_indexed | 2024-12-20T05:20:25Z |
| format | Article |
| id | doaj.art-2af4140fcd2f4150b9d8f84724496c4a |
| institution | Directory Open Access Journal |
| issn | 2312-0541 |
| language | English |
| last_indexed | 2024-12-20T05:20:25Z |
| publishDate | 2021-08-01 |
| publisher | European Respiratory Society |
| record_format | Article |
| series | ERJ Open Research |
| spelling | doaj.art-2af4140fcd2f4150b9d8f84724496c4a2022-12-21T19:52:02ZengEuropean Respiratory SocietyERJ Open Research2312-05412021-08-017310.1183/23120541.00143-202100143-2021Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quoElisangela Santos-Valente0Heike Buntrock-Döpke1Rola Abou Taam2Stefania Arasi3Arzu Bakirtas4Jaime Lozano Blasco5Klaus Bønnelykke6Mihai Craiu7Renato Cutrera8Antoine Deschildre9Basil Elnazir10Louise Fleming11Urs Frey12Monika Gappa13Antonio Nieto García14Kirsten Skamstrup Hansen15Laurence Hanssens16Karina Jahnz-Rozyk17Milos Jesenak18Sebastian Kerzel19Matthias V. Kopp20Gerard H. Koppelman21Uros Krivec22Kenneth A. MacLeod23Mika Mäkelä24Erik Melén25Györgyi Mezei26Alexander Moeller27Andre Moreira28Petr Pohunek29Predrag Minić30Niels W.P. Rutjes31Patrick Sammut32Nicolaus Schwerk33Zsolt Szépfalusi34Mirjana Turkalj35Iren Tzotcheva36Alexandru Ulmeanu37Stijn Verhulst38Paraskevi Xepapadaki39Jakob Niggel40Susanne Vijverberg41Anke H. Maitland-van der Zee42Uroš Potočnik43Susanne M. Reinartz44Cornelis M. van Drunen45Michael Kabesch46 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 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 Service de pneumologie pédiatrique, AP-HP, Hôpital Necker Enfants-Malades, Paris, France Translational Research in Pediatric Specialities Area, Division of Allergy, Bambino Gesù Children’s Research Hospital, IRCCS, Rome, Italy Dept of Pediatrics, Division of Pediatric Allergy and Asthma, Gazi University School of Medicine, Ankara, Turkey Dept of Pediatric Allergy and Clinical Immunology, Hospital Sant Joan de Déu, Universitat de Barcelona, Barcelona, Spain Dept of Pediatrics, Herlev and Gentofte Hospital, University Hospital Copenhagen, Denmark Respiratory Dept, National Institute for Mother and Child Health “Alessandrescu-Rusescu”, Bucharest, Romania Pediatric Pulmonology and Respiratory Intermediate Care Unit, Sleep and Long-Term Ventilation Unit, Academic Dept of Pediatrics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy University of Lille, CHU Lille, Paediatric Pulmonology and Allergy Unit, Hôpital Jeanne de Flandre, Lille, France Children's Health Ireland (CHI) at Tallaght University Hospital, Dublin, Ireland Respiratory Paediatrics, National Heart and Lung Institute, Imperial College London, London, UK University Children's Hospital Basel, University of Basel, Basel, Switzerland Evangelisches Krankenhaus Düsseldorf, Children's Hospital, Düsseldorf, Germany Pediatric Allergy and Pneumology Unit, Children's Hospital La Fe and Health Research Institute La Fe, Valencia, Spain Dept of Pediatrics, Herlev and Gentofte Hospital, University Hospital Copenhagen, Denmark Service de Pneumologie-Allergologie, Centre de reference de Mucoviscidose de l'ULB, Hôpital Universitaire des Enfants Reine Fabiola, Brussels, Belgium Dept of Internal Medicine, Pneumonology, Allergology and Clinical Immunology, Central Clinical Hospital of the Ministry of National Defense, Military Institute of Medicine, Warsaw, Poland Dept of Paediatrics, Jessenius Faculty of Medicine, Comenius University in Bratislava, Martin, Slovakia 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 University Children's Hospital Bern, Inselspital, University of Bern, Switzerland Dept of Pediatric Pulmonology and Pediatric Allergology, University Medical Center Groningen, University of Groningen, Beatrix Children's Hospital, Groningen, The Netherlands Dept of Paediatric Pulmology, University Children's Hospital Ljubljana, University Medical Centre Ljubljana, Ljubljana, Slovenia Royal Hospital for Children and Young People, Edinburgh, UK Skin and Allergy Hospital, Helsinki University Hospital and University of Helsinki, Helsinki, Finland Sachs’ Children and Youth Hospital, Södersjukhuset, Stockholm, Sweden Allergy Unit, First Dept of Pediatrics, Faculty of Medicine, Semmelweis University, Budapest, Hungary Division of Respiratory Medicine, University Children's Hospital Zurich and Childhood Research Center, Zurich, Switzerland Serviço de Imunoalergologia, Centro Hospitalar Universitário de São João, Porto, Portugal Pediatric Pulmonology, Pediatric Dept, 2nd Faculty of Medicine, Charles University, Prague, University Hospital Motol, Prague, Czech Republic Mother and Child Health Institute of Serbia, Belgrade, Serbia Dept of Paediatric Pulmonology, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands Dept of Paediatrics, Respiratory Medicine and Allergy, Mater Dei Hospital, Malta Dept of Pediatric Pneumology, Allergology and Neonatology, Hannover Medical School, Hannover, Germany Division of Pediatric Pulmonology, Allergology and Endocrinology, Dept of Pediatrics and Adolescent Medicine, Comprehensive Center Pediatrics, Medical University of Vienna, Vienna, Austria Srebrnjak Children's Hospital, Zagreb, and School of Medicine, University of Osijek “Josip Juraj Strossmayer”, Osijek, and Catholic University of Croatia, Zagreb, Croatia Dept of Pediatrics, University Hospital for Emergency Medicine “N. I. Pirogov”, Medical University, Sofia, Bulgaria “Grigore Alexandrescu” Emergency Hospital for Children, Bucharest, Romania Dept of Pediatrics, Antwerp University Hospital, Edegem, Belgium Allergy Dept, 2nd Pediatric Clinic, National and Kapodistrian University of Athens, Athens, Greece Datadesk Limited, Regensburg, Germany Dept of Respiratory Medicine, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands Dept of Respiratory Medicine, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands Laboratory for Biochemistry, Molecular Biology and Genomics, Faculty of Chemistry and Chemical Engineering, University of Maribor, Maribor, Slovenia Dept of Otorhinolaryngology, Tergooi Hospitals, Hilversum, the Netherlands Dept of Otorhinolaryngology, Amsterdam UMC, University of Amsterdam, Amsterdam, the Netherlands 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 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.http://openres.ersjournals.com/content/7/3/00143-2021.full |
| spellingShingle | Elisangela Santos-Valente Heike Buntrock-Döpke Rola Abou Taam Stefania Arasi Arzu Bakirtas Jaime Lozano Blasco Klaus Bønnelykke Mihai Craiu Renato Cutrera Antoine Deschildre Basil Elnazir Louise Fleming Urs Frey Monika Gappa Antonio Nieto García Kirsten Skamstrup Hansen Laurence Hanssens Karina Jahnz-Rozyk Milos Jesenak Sebastian Kerzel Matthias V. Kopp Gerard H. Koppelman Uros Krivec Kenneth A. MacLeod Mika Mäkelä Erik Melén Györgyi Mezei Alexander Moeller Andre Moreira Petr Pohunek Predrag Minić Niels W.P. Rutjes Patrick Sammut Nicolaus Schwerk Zsolt Szépfalusi Mirjana Turkalj Iren Tzotcheva Alexandru Ulmeanu Stijn Verhulst Paraskevi Xepapadaki Jakob Niggel Susanne Vijverberg Anke H. Maitland-van der Zee Uroš Potočnik Susanne M. Reinartz Cornelis M. van Drunen Michael Kabesch Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo ERJ Open Research |
| title | 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_short | 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 |
| url | http://openres.ersjournals.com/content/7/3/00143-2021.full |
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