Optimizing care for children with difficult-to-treat and severe asthma through specialist paediatric asthma centres: expert practical experience and advice

Severe asthma in children carries an unacceptable treatment burden, yet its rarity means clinical experience in treating it is limited, even among specialists. Practical guidance is needed to support clinical decision-making to optimize treatment for children with this condition. This modified Delphi convened 16 paediatric pulmonologists and allergologists from northern Europe, all experienced in treating children with severe asthma. Informed by interviews with stakeholders involved in the care of children with severe asthma (including paediatricians, nurses and carers), and an analysis of European guidelines, the experts built a consensus focused on the gaps in existing guidance. Explored were considerations for optimizing care for patients needing biologic treatment, and for selecting home or hospital delivery of biologics. This consensus is aimed at clinicians in specialist centres, as well as general paediatricians, paediatric allergologists and paediatric pulmonologists who refer children with the most severe asthma to specialist care. Consensus is based on expert opinion and is intended for use alongside published guidelines. Our discussions revealed three key facets to optimizing care. Firstly, early asthma detection in children presenting with wheezing and/or dyspnoea is vital, with a low threshold for referral from primary to specialist care. Secondly, children who may need biologics should be referred to and managed by specialist paediatric asthma centres; we define principles for the specialist team members, tests, and expertise necessary at such centres, as well as guidance on when homecare biologics delivery is and is not appropriate. Thirdly, shared decision-making is essential at all stages of the patient’s journey: clear, concise treatment plans are vital for patient/carer self-management, and structured processes for transition from paediatric to adult services are valuable. The experts identified the potential for specialist paediatric asthma nurses to play a significant role in facilitating multidisciplinary working. Through this project is agreed a framework of practical advice to optimize the care of children with severe asthma. We encourage clinicians and policymakers to implement this practical advice to enhance patient care. Supplementary Information The online version contains supplementary material available at 10.1186/s12887-024-04707-0.


Initial asthma presentation and diagnosis
Although no formal evaluation of suspected childhood asthma currently exists, the following assessments should be conducted as a minimum diagnostic workup: As part of the evaluation of suspected asthma in children, primary care practitioners should perform the diagnostic tests available to them and refer patients to specialist settings for remaining tests and confirmation of an asthma diagnosis.
Strongly disagree Disagree Neither agree nor disagree Agree Strongly agree • Agree when child is to be treated for longer time with ICS • over 12 years yes, 6-12 mainly tertiary care • spirometry in children needs experience and level of expertise to be performed correctly; atopy work-up can be performed in primary care, but not necessary for referral • pediatric pulmonary function is not available in primary care in Belgium, it can be available in secondary care.If this is meant by 'specialist settings', then I agree.Not all patients with asthma symptoms need to be referred to tertiary care/asthma specialist center.• in primary care mainly good clinical assessment a to e • IF they have spirometry available then it should be used to evaluate presence of reversible air flow limitation.However in our country most primary care practitioners do not have spirometry available or are able to use it for children in the lower age ranges.It has already been demonstrated that if spirometry is not used there is a huge overdiagnosis and over treatment of children with respiratory symptoms.So I do strongly agree spirometry is used, but in daily practice I think availability is lacking.In our medical care system in Belgium, all children with asthma should see a specialist at least once a year, and at diagnosis, to confirm diagnosis and follow the treatment, with in-between visits that can be done by primary care or specialist, according to the ability of the primary care, accessibility of the specialist, and severity of the disease • for children under 6 years with suspected asthma with intermittent wheeze, there is less added value for a specialist center, as they can not perform a reliable pulmonary function.Viral induced wheeze/preschool wheeze is so common that they can not all be followed by specialist center.I think all children above 6 with suspected asthma should be referred to a specialist (secondary care -pediatrician with access to pediatric PFT) for at least one PFT • This really depends on how primary care is arranged.In my experience asthma is overdiagnosed in younger children in the primary care setting and there usually is not enough time for a full evaluation that also includes evaluation and treatment of co-morbidities.To me there should be a low threshold for referral to a general pediatrician unless primary care is organized differently.In addition I have seen problems with follow up of patients in the primary care setting.
Experts rating agree or strongly agree 56% 88% 100% 100% Children with difficult-to-treat and severe asthma     Usually these children would primarily contact and visit their pediatric pulmonologist or perhaps a general paediatrician from a local hospital.So I would say that all treatment providers that are involved in shared care should be updated, but often a primary care clinician is not involved as a treatment provider in this population • Justn ot possible.They have access to electronic patient report • GP's are not very permanent in our system.They do not do follow-ups from their side but only based on the patient's activity to book an appointment Children with difficult-to-treat and severe asthma: Item 16 R1 Shared decision making and communication For complex cases, it is appropriate to work collaboratively with healthcare providers managing the patient in other settings, for example by occasionally including them in virtual MDT meetings.The following factors are essential to achieve effective transition from paediatric to adult centres: This question was originally posed as part of the previous question (Item R1-20).Several comments related to the possibility of nurses assistance with home delivery of biologics, so this was pulled out as a separate question to explore this possibility further.
Care related to biologic therapy: Item 18 R2 (Item 21 R1) of exercise-induced symptoms c) Assessment of the number of exacerbations in the last year d) Evaluation of risk factors e) Assessment of medication compliance and technique f) Lung function spirometry with reversibility g) Fractional exhaled nitric oxide (FeNO) test h) Atopic sensitization i) Blood eosinophil level as part of standard blood count

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a) Very young children < 2 years should be referred not all < 6 yrs.b) Depending on what morbidities are • Managing children with difficult to treat or severe asthma in specialist paediatric asthma centres: TO HELP EXPLAIN YOUR ANSWER • The strict interpretation of GDPR regulation in my country prohibits us to take part in the SPACE registry trial • d) due to interpretation of GDPR-law this is difficult pulmonologist or paediatric allergologist or paediatricians b) Paediatric pulmonary nurse or specialist asthma nurse c) Pulmonary function analyst or similar d) Medical social worker or paediatric psychologist e) Physiotherapist (paediatric or with experience in children) f) Dietician The multidisciplinary team members who are essential for specialist paediatric asthma centres are: TO HELP EXPLAIN YOUR ANSWER • f) Dietician on indication: food-allergies, under-or overweight • for a specialised paediatric asthma center a paediatric pulmonologist is essential.It is not or an allergologist or paediatrican • It is useful to have a medical social worker or psychologist to help children and families, but not mandatory.The same for dietician (if not available in the paediatric asthma centre, visits with dietician can be organised outside the center) • a) only pediatrician with pulmonology/allergology subspecialty b) agree but unfortunately financially difficult • It depends how a "Specialist asthma centre" is defined.Under (a) pediatric pulmonologist, allergologist and pediatrician are individual options.If a specialist paediatric asthma center is regarded as a center where children with severe asthma are treated and evaluated I think you would need somebody in your team who is able to perform bronchoscopy and has experience with the prescription of biologics of all medications including their purpose, dosing, administration, possible adverse events, etc c) The individual's role in self-management of their disease d) Triggers for intensifying, stopping or switching therapy e) Emergency contact information f) Advice on when to contact the emergency team The essential components of written (or digital) treatment plans are: TO HELP EXPLAIN YOUR ANSWER • b) except for side effects.c) triggers for intensifying: agree, stopping and switching: depends on what is meant.If it means switch from e.g.budesonide to prednisolone: agree, if it means change, or reducing salbutamol: agree • dosage of treatment should be in the written treatment plan, the possible adverse events is not mandatory • b) potential medication side effects should not be prominent in the treatment plan, more as extra info leaflet.the asthma plan should concise and clear c) what is meant by this? • written plan should not be too complex ; simple plan is important • On D I of course agree with intensifying the use of for instance SABA, but I don't think I would include something like "switching therapy".• written treatment plans should be short and concise and easy to understand TO HELP EXPLAIN YOUR ANSWER •In an optimal system yes but we do not have resources for thisChildren with difficult-to-treat and severe asthma: Item 13 R2 (Item 17 is a structured process rather than a single 'handover' event b) Transition timelines are tailored to the needs of the patient rather than dictated by their age c) Patient preparedness for transition is assessed by exploring their illness insight and ability to self-manage their disease d) The selection of the adult centre for referral is decided in consultation with patients and carers e) Detailed handover notes are provided to the adult centre and patient that include the details of any other specialists treating the patient in adult care settings of effectiveness of different biologics in children b) Collection and analysis of registry data on real-world asthma management c) How to select the most appropriate biologic for each patient d) When and how to start biologic treatment e) When and how to switch biologic treatment f) When and how to stop biologic treatment g) Biomarkers to support clinical decision making h) Effectiveness of home spirometry in enhancing disease control in children with difficult to treat or severe asthma Research priorities for difficult to treat and severe childhood asthma: TO HELP EXPLAIN YOUR ANSWER • h) I see effect of this in my daily practice in children with poor perception of asthma symptoms • first priority would be to have more data in the individual biological in childhood asthma • Regarding point h -there is already a lot of studies on this -see for example this paper published in ERJ from 2019: Clinical effect on uncontrolled asthma using a novel digital automated self-management solution: a physician-blinded randomised controlled crossover trial: https://erj.ersjournals.com/content/54/5ofhome spirometry in enhancing disease control in children with difficult-to-treat or severe asthma 69 This item was created from free text responses in R1 Children with suspected or confirmed asthma should be referred from primary care to a specialist in any of the following situations: • But if the GP has provided positive diagnostic tests for asthma, he/she does not have to refer for further confirmation, but may set the diagnosis him-/herself.Only in doubt, the child should be referred.But it must be totally clear to the GP, what is needed to set the diagnosis -• most GPs will not have availability of F and G. they should refer if diagnostic uncertainty or uncontrolled asthma despite step 2 treatment.• in small children yes.Adolescent, not complicated cases can be diagnosed in primary care b) The patient has multimorbidities in addition to asthma c) There is difficulty confirming the diagnosis of asthma d) Asthma is not adequately controlled on optimised therapy Access to the following facilities is essential for specialist paediatric asthma centres: . In my experience a general pediatrician usually does not have this expertise so I wonder if a pediatrician should be included in this description.• f) for food allergies yes, for pure, uncomplicated asthma no need PLEASE ADD INFORMATION TO HELP EXPLAIN YOUR ANSWER • d can be done by nurses in the daycare unit f) can be done by administrative team • administering biologics, and leading written treatment plan can be done either by physician or nurse according to each center habits • Regarding (D) it really depends on the local situation.You don't need a specialist pediatric asthma nurse to administer a biologic, depending on local situations this could be done by any nurse.PLEASE ADD INFORMATION TO HELP EXPLAIN YOUR ANSWER • a+b+c) or a referral center where these tests can be performed.Not necessarily available in the center • bronchoscopy and BAL can be referred in another center if needed, and children with severe asthma attacks can be referred to another center with ICU PLEASE ADD INFORMATION TO HELP EXPLAIN YOUR ANSWER • d) Not for every center • c) eucapnic hyperventilation test could be removed from the list, d) maybe rephrase the question into lung function tests may include?g) we use FeNo for follow up, not for differential diagnosis • Continuous laryngoscopy during exercise, and FeNO tests, are useful in some children, but not in all of them.Most centres don't use provocation testing in children.The other tests (spirometry, body plethysmography, and FeNO), are essential in evaluation of severe asthma in children.• We do methacholine challenge for 12 years or older and EVH for 9-10 years or older Some patients don't have the ability to self manage the disease even at adult age, but that should not prevent the transition • d) resources are also a factor, options might be limited Ideally so, sometimes when patient moves to different city for study eg is hard in daily practice • depends on the severity of asthma • the joint consultation helps a lot in lost of care; many children transitioning stop their follow up • in contrast to complexer chronic diseases, I find transition for asthma not that difficult.Selection of the biologic treatment should take into consideration clinical factors, practical factors (e.g.family circumstances, etc) and patient/carer preference.If the child has very poor symptom perception, home spirometry may enable effective homecare delivery of biologic treatment.If the carer or patient does not feel confident or capable of giving injections, a nurse may give injections in the home situation.
PLEASE ADD INFORMATION TO HELP EXPLAIN YOUR ANSWER • c) Consider administration by nurse at home or at GP • d) depends on type of reaction, frequency and whether the reaction has stopped to occur • c) can be given by homecare • d) depends on the reaction and the drug • in a and c a home nurse can be organised • c) with education they may fell more confident and capable PLEASE ADD INFORMATION TO HELP EXPLAIN YOUR ANSWER • I don't get this item.In the phase before start of biologics home spirometry is very important to me in patients with very poor symptom perception for self-management.If despite of home spirometry use asthma is still poorly controlled I would start biologics.I don't understand in what way it may enable treatment with biologics • home spirometry may not be reliable in children due to lack of cooperation; if the child has very poor symptom perception, delivery of biologic treatment should be performed at the clinical center and not at home • Why not measure PEF at home? • I have lots of experience in this -and the home spirometry may both indicate the need for biologics, or that the child actually are totally controlled on their present medication and their perceived symptoms are not due to their lung function • not too much evidence but does make sense PLEASE ADD INFORMATION TO HELP EXPLAIN YOUR ANSWER • The injections can be given in their nearest health care center • or nurse can give it at school or primary care setting