Figures
- FIGURE 1
Overview of the project methodology. ELF: European Lung Foundation; ERS: European Respiratory Society.
- FIGURE 2
Results from the parent/patient survey relating to aspects of their child's bronchiectasis on their quality of life. a) Mean scores of items rated by parents affecting their child's quality of life. b) Mean scores of items rated by parents affecting their own quality of life. #: e.g. walking far, swimming.
Tables
- TABLE 1
Parent/patient survey: clinical needs themes and priorities
Theme Items (verbatim from survey questions) Mean score Awareness and diagnosis To find ways to diagnose bronchiectasis earlier, such as by local doctors# 3.86 To improve awareness of bronchiectasis in community care services, e.g. among community-based nurses and physiotherapists 3.82 To identify the cause(s) of bronchiectasis 3.80 Education and support for parents and families Knowing more about the role of physiotherapy and pulmonary rehabilitation (a short course of regular exercise sessions and education sessions) 3.80 Having access to reliable, easy to understand information about different aspects of living with bronchiectasis 3.78 Providing each person with copies of their test results so they can keep a useful history of the progress of their own condition 3.74 Develop better ways of teaching people to use their medicines 3.71 Improving access to quality care Having access to physiotherapy and being taught the techniques and how to use the equipment at home 3.94 Good communication between healthcare professionals and each person with bronchiectasis 3.91 Testing new techniques for managing bronchiectasis in real world environments, such as at home and community 3.86 Better access to tests and experts on bronchiectasis 3.80 Using peer support forums and social media to exchange information with others 3.52 Managing exacerbations Having an action management plan for flare-ups/exacerbations 3.94 Having a self-management programme and care plan designed with each person to help them have greater control over their condition and recognise/manage an exacerbation 3.92 Finding triggers of exacerbation 3.86 Educating primary care doctors to prescribe the same dose/length of antibiotic therapy for exacerbations as used in cystic fibrosis¶ 3.81 Improving treatment To improve how children with bronchiectasis are treated through using longer-term antibiotic therapy when a person's condition is stable+ 3.71 Being able to identify people at increased risk of poor outcomes or needing urgent treatment for their bronchiectasis 3.85 Being able to monitor and treat the coughing up of blood 3.77 Improving monitoring Having regular lung function testing to help notice changes or increased risk of an exacerbation 3.76 Being able to monitor cough 3.68 Regular sputum examinations when a person is stable and during an exacerbation to learn more about how the condition changes 3.64 Having the equipment at home to monitor symptoms 3.58 Ensuring each person has access to a home intravenous antibiotic service to avoid unnecessary hospital admissions 3.53 Having regular computed tomography scans to look for changes or increased risk of an exacerbation§ 3.45 #: refers to parent's/patient's experience of delayed referral from a lack of awareness of the symptoms of bronchiectasis and dismissing children's chronic wet cough; importantly, we do not expect primary care doctors to undertake computed tomography (CT) scans in young children. ¶: refers to parent's/patient's experience that they are often given a shorter antibiotic course (e.g. 3 days as opposed to current guideline recommendations of 14 days [12]) and/or lower doses than what is generally considered optimal; doses for children with CF are generally higher than for those without CF as children with CF have higher volume of distribution and renal clearance mechanisms; thus, we do not suggest prescribing antibiotics using CF-based dosing regimens, as it can be potentially dangerous for some agents. +: refers to identifying when and in whom long-term antibiotics should be used to induce clinical stability. §: the ERS Clinical Practice Guidelines [12] state that “In children/adolescents with bronchiectasis, we suggest the decision to repeat chest CT scans is individualised based on the clinical status and setting. Remarks: Repeat chest CT scans should be considered to answer a question which will change management.”
- TABLE 2
Parent/patient survey: research priorities and themes
Research theme Items (verbatim from survey questions) Mean score Understanding mechanisms and biology of bronchiectasis Finding ways to prevent bronchiectasis 3.89 Identifying how bronchiectasis develops and continues 3.87 Identifying the cause(s) of bronchiectasis 3.80 Identifying how often and why bronchiectasis occurs in certain groups of people across the world 3.74 Finding new ways to improve diagnosis and treatment Finding ways to diagnose bronchiectasis earlier, such as by local doctors# 3.86 Testing new techniques for managing bronchiectasis in real world environments, such as at home and community settings (not in the laboratory or in hospitals) 3.86 Finding new medicines to treat bronchiectasis 3.84 Finding new physiotherapy/airway clearance techniques 3.79 Using longer-term antibiotic therapy when a person's condition is stable¶ 3.71 Developing medicines that can be taken in different ways, such as for inhaled or nebulised 3.67 Improving knowledge and treatment of exacerbations Identifying triggers for an exacerbation 3.86 Identifying people at increased risk of poor outcomes or needing urgent treatment for their bronchiectasis 3.85 Using vaccines to prevent exacerbations 3.78 Exploring the link between getting a cold (for example rhinovirus) and having an exacerbation 3.74 Improving monitoring and how to identify predictors of disease progression Identifying what makes some patients’ bronchiectasis get worse 3.92 Understanding the relationship between bronchiectasis and other medical conditions, e.g. asthma, “acid” reflux 3.80 Being able to monitor and treat the coughing up of blood 3.77 Having regular lung function testing to help notice changes or increased risk of an exacerbation 3.76 Being able to monitor cough 3.68 #: refers to parent's/patient's experience of delayed referral due to lack of awareness of the symptoms of bronchiectasis and dismissing children's chronic wet cough; we do not expect primary care doctors to undertake computed tomography scans in young children. ¶: refers to identifying when and in whom long-term antibiotics should be used to induce clinical stability.
- TABLE 3
Health practitioner survey: research priorities and themes
Theme In children and young people with bronchiectasis (unrelated to cystic fibrosis) Mean score Finding new ways to improve treatment When and how (antibiotic, dose, regimen, route (intravenous, oral or inhaled/nebulised) and duration) should pathogens other than Pseudomonas aeruginosa be eradicated, and do patient outcomes improve afterwards? 4.27 What are the optimal and most cost-effective airway clearance techniques? 4.26 When and how (antibiotic, dose, regimen, route (intravenous, oral or inhaled/nebulised) and duration) should P. aeruginosa be eradicated, and do patient outcomes improve afterwards? 4.18 What is the best antibiotic, dose, regimen and duration for long-term oral antibiotic therapy in patients with bronchiectasis (according to the presence or absence of P. aeruginosa or other pathogens)? 4.17 What are the indications of oral versus inhaled/nebulised long-term suppressive antibiotic treatment? 4.14 When should airway clearance techniques be started in patients with bronchiectasis, and how often should it be done during the stable state and for exacerbations? 4.09 What is the impact of long-term antibiotic therapy on anti-microbial resistance? 4.03 What is the role of different mucoactive agents (e.g. inhaled hypertonic or isotonic saline, mannitol, oral erdosteine or N-acetyl cysteine)? 4.03 What are the simple, reliable microbiological tests for determining lower airway infection? 3.90 What are the most efficient clinical trial designs and measurable outcomes? 3.83 What are the key factors leading to P. aeruginosa acquisition and infection? 3.78 Which clinical and microbiological factors affect macrolide efficacy? 3.71 What is the role of surgery (segmentectomy, lobectomy or pneumonectomy) and when should it be undertaken? 3.62 What is the role of long-term inhaled corticosteroids? 3.53 What is the role of non-pharmacological, non-airway clearance technique-based therapeutics, such as singing exercise, wind instruments and yoga during stable states and acute exacerbations? 3.45 What is the best model/approach for transferring an adolescent with bronchiectasis to adult services? 3.42 Finding new ways to improve diagnosis What are the baseline investigations to identify underlying aetiologies of bronchiectasis? 3.98 What clinical factors should be present to trigger referring a child for a chest computed tomography scan to diagnose bronchiectasis? 3.87 Improving knowledge and treatment of exacerbations What is the optimal antibiotic therapy (dosage, how many antibiotics, type, oral versus intravenous versus inhaled/nebulised and length of therapy) for an exacerbation of bronchiectasis? 4.41 What are the most important factors to prevent acute exacerbations? 4.12 How to define acute exacerbations that require additional treatment? 4.05 What are the causes of an exacerbation of bronchiectasis? 3.84 Which are the best systemic (e.g. blood) or local (e.g. sputum) inflammatory markers for the diagnosis, management and follow-up for an exacerbation? 3.82 How should the severity of an exacerbation of bronchiectasis be assessed and what is its impact on long-term outcomes? 3.78 What types of biomarker(s) can be used for predicting bronchiectasis exacerbation? 3.68 Improving prevention and monitoring What are the risk factors and causes of rapid progression of lung disease and poor outcomes (e.g. hospitalisation, lung transplantation and mortality)? 4.17 How best to prevent development of bronchiectasis? 4.16 Should there be paediatric-focused patient registries? 4.06 What are the best and most pragmatic functional tests (such as carbon monoxide diffusing capacity, 6-min walk test, lung clearance index, endurance shuttle walk, incremental exercise tests or accelerometers) as markers for severity of the disease, outcomes and end-points for the clinic? 4.00 What are the risk or protective factors for lung function decline in patients with bronchiectasis? 3.91 What are the factors that predict radiographic reversibility (on a high-resolution computed tomography scan)? 3.84 What is the best approach/score to evaluate radiographic severity? 3.82 What types of specific patient education packages, self-management plans and patient support groups improve outcomes? 3.81 Should a severity and prognostic score for children that is useful in clinical practice be developed? 3.70 What comorbidities are present and how do they influence bronchiectasis severity? 3.59 Is cross-infection important, what are the best strategies and is strict patient segregation required? 3.59 What types of biomarker(s) can be used to monitor bronchiectasis severity during stable state, so as to define the subgroup who will benefit from more intensive treatment? 3.58 Can endo-phenotyping predict severity and outcomes in children? 3.54 Understanding mechanisms and biology of bronchiectasis Do different aetiologies and/or comorbidities of bronchiectasis predetermine microbiological characteristics and affect severity, patients’ quality of life and disease progression? 3.92 What is the role of viruses, fungi and anaerobes (as single agents and/or polymicrobial infections), during both the stable state and exacerbation, and what is their impact upon patient severity and outcomes? 3.85 Is there an increased rate of primary immune defects (e.g. mannose-binding lectin deficiency, common variable immunodeficiency, IgM or IgA deficiency, or complement deficiency)? 3.78 What is the incidence and prevalence of different aetiologies of bronchiectasis across the world? 3.55 What is the relationship between paediatric and adult bronchiectasis? 3.54 What is the importance of host–pathogen–environment interactions? 3.53 What are the molecular and cellular mechanisms and pathobiological pathways of bronchiectasis development, exacerbations and progression? 3.52 What is the composition and function of the host microbiome, both during the stable state and exacerbations, and does it impact directly disease severity and progression? 3.48 What are the genetic and epigenetic findings in patients with bronchiectasis compared to healthy controls, and what is their role in acquisition of specific pathogens and patients’ outcomes? 3.39 What is the best experimental model system of bronchiectasis? 3.24 Other What are the healthcare costs of bronchiectasis management across the world? 3.19 - TABLE 4
Derivation of the top 10 research priorities#
Consensus priorities (parent/patient and health practitioners) and confirmed with two parent advisory groups Parent/patient survey (verbatim from survey questions) Health practitioner survey (verbatim from survey questions) Theme: Understanding mechanisms and biology of bronchiectasis Identifying risk and protective factors for bronchiectasis Identifying what makes some patients’ bronchiectasis get worse How best to prevent development of bronchiectasis? Finding ways to prevent bronchiectasis Identifying the underlying aetiologies of bronchiectasis Identifying the cause(s) of bronchiectasis What are the baseline investigations to identify underlying aetiologies of bronchiectasis? Theme: Diagnosis Discovering ways to diagnose bronchiectasis earlier, including ways to increase health practitioner awareness and to facilitate earlier referrals Discovering ways to diagnose bronchiectasis earlier, such as by local doctors¶ Theme: Improving knowledge and treatment of exacerbations Identifying triggers/prevention factors and optimal antibiotic treatment for acute exacerbations Identifying triggers for an exacerbation What are the most important factors at preventing acute exacerbations? Exploring the link between getting a cold (for example rhinovirus) and having an exacerbation What is the optimal antibiotic therapy (dosage, how many antibiotics, type, oral versus intravenous versus inhaled/nebulised and length of therapy) for an exacerbation of bronchiectasis? Using vaccines to prevent exacerbations Theme: Finding new ways to improve treatment Finding new and optimal airway clearance techniques Finding new physiotherapy/airway clearance techniques What are the optimal and most cost-effective airway clearance techniques? When should airway clearance techniques be started in patients with bronchiectasis, and how often should it be done during the stable state and for exacerbations? Defining optimal antibiotic therapy for eradicating specific pathogens (e.g. Pseudomonas aeruginosa) and for suppressing bacteria once chronic infection is established Using longer-term antibiotic therapy when a person's condition is stable+ When and how (antibiotic, dose, regimen, route (intravenous, oral or inhaled/nebulised) and duration) should P. aeruginosa be eradicated, and do patient outcomes improve afterwards? When and how (antibiotic, dose, regimen, route (intravenous, oral or inhaled/nebulised) and duration) should pathogens other than P. aeruginosa be eradicated, and do patient outcomes improve afterwards? What are the indications of oral versus inhaled/nebulised long-term suppressive antibiotic treatment? What is the best antibiotic, dose, regimen and duration for long-term oral antibiotic therapy in patients with bronchiectasis (according to the presence or absence of P. aeruginosa or other pathogens)? Finding new medications and/or techniques for managing bronchiectasis Testing new techniques for managing bronchiectasis in real world environments, such as at home and community What is the role of different mucoactive agents (e.g. inhaled hypertonic or isotonic saline, mannitol, oral erdosteine or N-acetyl cysteine)? Finding new medicines to treat bronchiectasis Theme: Improving monitoring and how to identify predictors of disease progression Identifying lung function tests/indices that predict outcomes Having regular lung function testing to help notice changes or increased risk of an exacerbation What are the best and most pragmatic functional tests (such as carbon monoxide diffusing capacity, 6-min walk test, lung clearance index, endurance shuttle walk, incremental exercise tests or accelerometers) as markers for severity of the disease, outcomes and end-points for the clinic? Understanding the relationship between causes and comorbidities of bronchiectasis with clinical outcomes To know how bronchiectasis affects other body parts/organs in addition to the lung Do different aetiologies and/or comorbidities of bronchiectasis predetermine microbiological characteristics, and affect severity, patients’ quality of life and disease progression? To understand the relationship between bronchiectasis and other medical conditions e.g. asthma, “acid” reflux Identifying factors associated with worse bronchiectasis outcomes Identifying what makes some patients’ bronchiectasis get worse What are the risk factors and causes of rapid progression of lung disease and poor outcomes (e.g. hospitalisation, lung transplantation and mortality)? Identifying people at increased risk of poor outcomes or needing urgent treatment for their bronchiectasis What are the risk or protective factors for lung function decline in patients with bronchiectasis? #: the list is not in order of priority, i.e. all are considered equal. ¶: refers to parent's/patient's experience of delayed referral due to lack of awareness of the symptoms of bronchiectasis and dismissing children's chronic wet cough; we do not expect primary care doctors to undertake computed tomography scans in young children. +: refers to identifying when and in whom long-term antibiotics should be used to induce clinical stability.
Supplementary Material
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Supplementary material 00122-2021.SUPPLEMENT
Vol 7 Issue 3
Table of Contents
Clinical and research priorities for children and young people with bronchiectasis: an international roadmap
