Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo
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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.
| Originalsprog | Engelsk |
|---|---|
| Artikelnummer | 00143-2021 |
| Tidsskrift | ERJ Open Research |
| Vol/bind | 7 |
| Udgave nummer | 3 |
| Antal sider | 9 |
| ISSN | 2312-0541 |
| DOI | |
| Status | Udgivet - 2021 |
Bibliografisk note
Funding Information:
Support statement: The PERMEABLE consortium is supported by ZonMW (project number 456008004), the Swedish Research Council (project number 2018-05619), the Ministry of Education, Science and Sport of the Republic of Slovenia (contract number C3330-19-252012), and the German Ministry of Education and Research (BMBF) (project number FKZ01KU1909A), under the frame of the ERA PerMed JTC 2018 Call. Funding information for this article has been deposited with the Crossref Funder Registry.
Funding Information:
Conflict of interest: E. Santos-Valente has nothing to disclose. H. Buntrock-Döpke has nothing to disclose. R. Abou Taam has nothing to disclose. S. Arasi has nothing to disclose. A. Bakirtas has nothing to disclose. J. Lozano Blasco reports personal fees from Novartis and GSK outside the submitted work. K. Bønnelykke has nothing to disclose. 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. R. Cutrera has nothing to disclose. A. Deschildre reports personal fees and other support from Novartis, GSK and Sanofi outside the submitted work. B. Elnazir has nothing to disclose. L. 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. U. Frey has nothing to disclose. M. Gappa reports personal fees from Boehringer Ingelheim, GSK, Novartis and Sanofi outside the submitted work. A. Nieto García reports grants for clinical studies and advisory boards membership, as well as lecture fees from Novartis, GSK and MSD. K. Skamstrup Hansen has nothing to disclose. L. Hanssens has nothing to disclose. K. Jahnz-Rozyk received lecture and advisory board fees from AstraZeneca, Novartis, GSK, and Sanofi Aventis, outside the submitted work. M. Jesenak has nothing to disclose. S. Kerzel reports a speaker fee from Novartis for a lecture that has no topical overlap at all with the current manuscript. M. V. Kopp reports personal fees from ALK-Abello GmbH, Chiesi GmbH, Infectopharm GmbH, Novartis GmbH, Sanofi Aventis HmbH and Vertex GmbH, and grants and personal fees from Allergopharma GmbH, outside the submitted work. G.H. 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). U. Krivec has nothing to disclose. K.A. MacLeod has nothing to disclose. M. Mäkelä has nothing to disclose. E. Melén reports personal fees from AstraZeneca, Chiesi, Novartis and Sanofi (advisory board fees), outside the submitted work. G. Mezei reports a travel grant from LOFARMA outside the submitted work. A. Moeller has nothing to disclose. A. Moreira has nothing to disclose. P. Pohunek reports personal fees for advisory board membership from Novartis and GlaxoSmithKline, and for consultation from Chiesi and AstraZeneca, outside the submitted work. P. Minić has nothing to disclose. N.W.P. Rutjes reports personal fees from GSK (advisory board on mepolizumab) and Sanofi (advisory board on dupilumab) outside the submitted work. P. Sammut has nothing to disclose. 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. Z. Szépfalusi has nothing to disclose. M. Turkalj has nothing to disclose. I. Tzotcheva has nothing to disclose. A. Ulmeanu has nothing to disclose. S. Verhulst reports grants from GSK during the conduct of the study. P. Xepapadaki reports personal fees for advisory services from Uriach, Novartis, Nestle and Nutricia outside the submitted work. J. Niggel has nothing to disclose. S. Vijverberg reports grants from ZonMW during the conduct of the study. A-H. Maitland van der Zee has received research grants outside the submitted work from GSK, Boehringer Ingelheim and Vertex; she is the principal investigator 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. 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. S. M. Reinartz has nothing to disclose. C. M. van Drunen has nothing to disclose. M. Kabesch reports grants to his institution from the European Union, the German Ministry of Education and Research, and the German Research Foundation, during the conduct of the study; and consultancy fees from Bionorica, Sanofi, Novartis and Bencard, payments for lectures form the ERS, EAACI, ATS, Novartis, Glaxo, Nutricia, Hipp and Allergopharma, and a patent for a method for testing a subject thought to have or be disposed to asthma (European patent application 5 EP070301 135.5), outside the submitted work.
Funding Information:
The PERMEABLE consortium wishes to acknowledge the help and expertise of the following individuals, without whom this work would not have been possible: Gabriel Zink (Byteschmiede UG, Parsberg, Germany); Boris Gole (Molecular Biology and Genomics, Faculty for Chemistry and Chemical Engineering, University of Maribor, Maribor, Slovenia); Korneliusz Golebski, Paul Brinkman and Simone Hashimoto (Respiratory Medicine, Amsterdam UMC, University of Amsterdam, the Netherlands); Jon Konradsen (Dept of Women?s and Children?s Health, Karolinska Institutet, Stockholm, Sweden); and Caroline Nilsson (Dept of Clinical Sciences and Education, S?dersjukhuset, Karolinska Institutet, Stockholm, Sweden).Support statement: The PERMEABLE consortium is supported by ZonMW ( project number 456008004), the Swedish Research Council ( project number 2018-05619), the Ministry of Education, Science and Sport of the Republic of Slovenia (contract number C3330-19-252012), and the German Ministry of Education and Research (BMBF) ( project number FKZ01KU1909A), under the frame of the ERA PerMed JTC 2018 Call. Funding information for this article has been deposited with the Crossref Funder Registry.
Publisher Copyright:
© The authors 2021.
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