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Biologicals in childhood severe asthma: the European PERMEABLE survey on the status quo
Elisangela Santos-Valente1, Heike Buntrock-Döpke1,2, Rola Abou Taam3
1Dept 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.
Insights
Management of severe asthma in children varies significantly across Europe. Further research is needed on biomarkers for selecting biological therapies and criteria for assessing and discontinuing treatment in children with asthma.
Area of Science:
- Pediatric Pulmonology
- Clinical Immunology
- Pharmacotherapy
Background:
- Severe asthma in children is rare, with limited biological treatments available in Europe.
- Global guidelines for biologicals exist, but real-world implementation and unmet needs are unclear.
- This survey investigates the current status and challenges of biological therapy for childhood asthma in Europe.
Purpose of the Study:
- To investigate the current practices and identify unmet needs in the biological therapy of childhood severe asthma across Europe.
- To understand the implementation of existing guidelines for biological treatments in pediatric asthma.
- To explore variations in drug selection, access, and treatment outcomes.
Main Methods:
- Structured interviews with 37 experts from 25 European countries and Turkey.
- Content analysis of data on biological therapy experience, regulations, drug selection, success assessment, and discontinuation.
- Focus on real-world data regarding the management of severe asthma in children.
Main Results:
- Significant variation exists in the number of children treated with biologicals per center across Europe.
- Public access to at least one biological is available in all participating countries, with varied prescribing practices.
- Considerable differences were observed in assessing treatment success, therapy duration, and discontinuation rates.
Conclusions:
- Substantial disparities in managing childhood severe asthma across Europe necessitate further investigation.
- There is a clear need for research on biomarkers to guide biological selection in pediatric asthma.
- Further studies are required to establish criteria for assessing therapy response and determining when to discontinue treatment in stable patients.
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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