ERJ Open Research (Aug 2021)

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

  • 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

DOI
https://doi.org/10.1183/23120541.00143-2021
Journal volume & issue
Vol. 7, no. 3

Abstract

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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.