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Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo

E. Santos-Valente, H. Buntrock-Döpke, R. Abou Taam, S. Arasi, A. Bakirtas, J. Lozano Blasco, K. Bønnelykke, M. Craiu, R. Cutrera, A. Deschildre, B. Elnazir, L. Fleming, U. Frey, M. Gappa, A. Nieto García, K. Skamstrup Hansen, L. Hanssens, K....

. 2021 ; 7 (3) : . [pub] 20210816

Jazyk angličtina Země Velká Británie

Typ dokumentu časopisecké články

Perzistentní odkaz   https://www.medvik.cz/link/bmc21024146

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.

Allergy Dept 2nd Pediatric Clinic National and Kapodistrian University of Athens Athens Greece

Allergy Unit 1st Dept of Pediatrics Faculty of Medicine Semmelweis University Budapest Hungary

Centre for Human Molecular Genetics and Pharmacogenomics Medical Faculty University of Maribor Maribor Slovenia

Children's Health Ireland at Tallaght University Hospital Dublin Ireland

Datadesk Limited Regensburg Germany

Dept of Clinical Immunology and Allergology University Teaching Hospital in Martin Martin Slovakia

Dept of Clinical Science and Education Södersjukhuset Karolinska Institutet Stockholm Sweden

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 Otorhinolaryngology Amsterdam UMC University of Amsterdam Amsterdam the Netherlands

Dept of Otorhinolaryngology Tergooi Hospitals Hilversum the Netherlands

Dept of Paediatric Pulmology University Children's Hospital Ljubljana University Medical Centre Ljubljana Ljubljana Slovenia

Dept of Paediatric Pulmonology Amsterdam UMC University of Amsterdam Amsterdam The Netherlands

Dept of Paediatric Respiratory Medicine and Allergy Emma Children's Hospital Amsterdam UMC University of Amsterdam Amsterdam the Netherlands

Dept of Paediatrics Jessenius Faculty of Medicine Comenius University in Bratislava Martin Slovakia

Dept of Paediatrics Respiratory Medicine and Allergy Mater Dei Hospital Malta

Dept of Pediatric Allergy and Clinical Immunology Hospital Sant Joan de Déu Universitat de Barcelona Barcelona Spain

Dept of Pediatric Pneumology Allergology and Neonatology Hannover Medical School Hannover Germany

Dept of Pediatric Pneumology and Allergy University Children's Hospital Regensburg at the Hospital St Hedwig of the Order of St John University of Regensburg Regensburg Germany

Dept of Pediatric Pulmonology and Pediatric Allergology University Medical Center Groningen University of Groningen Beatrix Children's Hospital Groningen The Netherlands

Dept of Pediatrics Antwerp University Hospital Edegem Belgium

Dept of Pediatrics Division of Pediatric Allergy and Asthma Gazi University School of Medicine Ankara Turkey

Dept of Pediatrics Herlev and Gentofte Hospital University Hospital Copenhagen Denmark

Dept of Pediatrics University Hospital for Emergency Medicine N 1 Pirogov Medical University Sofia Bulgaria

Dept of Respiratory Medicine Amsterdam UMC University of Amsterdam Amsterdam the Netherlands

Division of Pediatric Pulmonology Allergology and Endocrinology Dept of Pediatrics and Adolescent Medicine Comprehensive Center Pediatrics Medical University of Vienna Vienna Austria

Division of Respiratory Medicine University Children's Hospital Zurich and Childhood Research Center Zurich Switzerland

EPIUnit Instituto de Saúde Pública Universidade do Porto Porto Portugal

Evangelisches Krankenhaus Düsseldorf Children's Hospital Düsseldorf Germany

German Center for Lung Research Biomedical Research in End Stage and Obstructive Lung Disease BREATH German Center for Lung Research Hannover Germany

German Center for Lung Research DZL Airway Center North Hannover Germany

Grigore Alexandrescu Emergency Hospital for Children Bucharest Romania

Groningen Research Institute for Asthma and COPD University Medical Center Groningen University of Groningen Groningen the Netherlands

Laboratory for Biochemistry Molecular Biology and Genomics Faculty of Chemistry and Chemical Engineering University of Maribor Maribor Slovenia

Laboratory of Experimental Medicine and Pediatrics Faculty of Medicine and Health Sciences University of Antwerp Antwerp Belgium

Member of the Research and Development Campus Regensburg at the Hospital St Hedwig of the Order of St John Regensburg Germany

Mother and Child Health Institute of Serbia Belgrade Serbia

Paediatric Respiratory Medicine Royal Brompton and Harefield NHS Foundation Trust London UK

Pediatric Allergy and Pneumology Unit Children's Hospital La Fe and Health Research Institute La Fe Valencia Spain

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

Pediatric Pulmonology Pediatric Dept 2nd Faculty of Medicine Charles University Prague University Hospital Motol Prague Czech Republic

Respiratory Dept National Institute for Mother and Child Health Alessandrescu Rusescu Bucharest Romania

Respiratory Paediatrics National Heart and Lung Institute Imperial College London London UK

Royal Hospital for Children and Young People Edinburgh UK

Sachs' Children and Youth Hospital Södersjukhuset Stockholm Sweden

Service de Pneumologie Allergologie Centre de reference de Mucoviscidose de l'ULB Hôpital Universitaire des Enfants Reine Fabiola Brussels Belgium

Service de pneumologie pédiatrique AP HP Hôpital Necker Enfants Malades Paris France

Serviço de Imunoalergologia Centro Hospitalar Universitário de São João Porto Portugal

Skin and Allergy Hospital Helsinki University Hospital and University of Helsinki Helsinki Finland

Srebrnjak Children's Hospital Zagreb and School of Medicine University of Osijek Josip Juraj Strossmayer Osijek and Catholic University of Croatia Zagreb Croatia

Translational Research in Pediatric Specialities Area Division of Allergy Bambino Gesù Children's Research Hospital IRCCS Rome Italy

Trinity College Dublin Ireland

University Children's Hospital Basel University of Basel Basel Switzerland

University Children's Hospital Bern Inselspital University of Bern Switzerland

University of Lille CHU Lille Paediatric Pulmonology and Allergy Unit Hôpital Jeanne de Flandre Lille France

Citace poskytuje Crossref.org

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$a Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo / $c E. Santos-Valente, H. Buntrock-Döpke, R. Abou Taam, S. Arasi, A. Bakirtas, J. Lozano Blasco, K. Bønnelykke, M. Craiu, R. Cutrera, A. Deschildre, B. Elnazir, L. Fleming, U. Frey, M. Gappa, A. Nieto García, K. Skamstrup Hansen, L. Hanssens, K. Jahnz-Rozyk, M. Jesenak, S. Kerzel, MV. Kopp, GH. Koppelman, U. Krivec, KA. MacLeod, M. Mäkelä, E. Melén, G. Mezei, A. Moeller, A. Moreira, P. Pohunek, P. Minić, NWP. Rutjes, P. Sammut, N. Schwerk, Z. Szépfalusi, M. Turkalj, I. Tzotcheva, A. Ulmeanu, S. Verhulst, P. Xepapadaki, J. Niggel, S. Vijverberg, AH. Maitland-van der Zee, U. Potočnik, SM. Reinartz, CM. van Drunen, M. Kabesch
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$a 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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