Airway clearance management in people with bronchiectasis: data from the European Bronchiectasis Registry (EMBARC).

BACKGROUND: International guidelines recommend airway clearance management as one of the important pillars of bronchiectasis treatment. However, the extent to which airway clearance is used for people with bronchiectasis in Europe is unclear. The aim of the study was to identify the use of airway clearance management in patients with bronchiectasis across different countries and factors influencing airway clearance use. METHODS: This was a prospective observational study using data from the European Multicentre Bronchiectasis Audit and Research Collaboration (EMBARC) Registry between January 2015 and April 2022. Prespecified options for airway clearance management were recorded, including airway clearance techniques, devices and use of mucoactive drugs. RESULTS: 16 723 people with bronchiectasis from 28 countries were included in the study. The mean age was 67 years (interquartile range 57-74 years, range 18-100 years) and 61% were female. 72% of the participants reported daily sputum expectoration and 52% (95% CI 51-53%) of all participants reported using regular airway clearance management. Active cycle of breathing technique was used by 28% of the participants and airway clearance devices by 16% of participants. The frequency of airway clearance management and techniques used varied significantly between different countries. Participants who used airway clearance management had greater disease severity and worse symptoms, including a higher daily sputum volume, compared to those who did not use it regularly. Mucoactive drugs were also more likely to be used in participants with more severe disease. Access to specialist respiratory physiotherapy was low throughout Europe, but particularly low in Eastern Europe. CONCLUSIONS: Only a half of people with bronchiectasis in Europe use airway clearance management. Use of and access to devices, mucoactive drugs and specialist chest physiotherapy appears to be limited in many European countries.

Translation, reliability and validity of the Greek version of the Child Engagement in Daily Life in children with cerebral palsy.

BACKGROUND: Participation in family, recreational activities and self-care is an integral part of health. It is also a main outcome of rehabilitation services for children and adolescents with disabilities. However, there are currently no available tools in Greek to assess participation in young children. METHODS: The Child Engagement in Daily Life (CEDL) was cross-culturally translated into Greek using forward-backward translation, review by expert committee, pretest application and final review. Parents with children who have been diagnosed with cerebral palsy (CP) and were 18 months to 5 years old were recruited. Internal consistency was evaluated using the Cronbach alpha and test-retest reliability in 2 weeks using intra-class correlation coefficient (ICC) and Bland-Altman plot for the agreement of each domain score. Measurement error was assessed utilising the standard error of measurement (SEM) and the smallest detectable change (SDC) and interpretability was assessed using the floor and ceiling effects. Validity was evaluated using the 'known groups' method, that is, assessing parents of children with typical development (TD). RESULTS: One hundred and seven children with CP (mean age 43.63 ± 13.5 months), Gross Motor Function Classification System (GMFCS) levels I-V and 97 children with TD (mean age 43.63 ± 14.4 months) were included. Significant differences (p < 0.01) between children with CP and children with TD were recorded for all CEDL domains. Mean ± standard deviation of the CEDL domains 'frequency of participation', 'enjoyment of participation' and 'self-care' were 58.8 ± 14.5, 3.9 ± 0.9 and 49.7 ± 23.5, respectively for children with CP and 62.3 ± 9.1, 4.4 ± 0.9 and 74.2 ± 15 for children with TD. Internal consistency of all domains was high; Cronbach alpha for 'frequency of participation' was 0.83, for 'enjoyment of participation' was 0.76 and for 'self-care' was 0.92. Test-retest reliability (ICC) was excellent for the 'self-care' (0.95) and good for 'frequency of participation' and 'enjoyment of participation' domains (0.90 and 0.88, respectively) while Bland-Altman analysis revealed no systematic differences or bias between the two measurements. SEM was 0.8, 0.05 and 1.6 for frequency of participation, enjoyment and self-care with SDC of 2.4, 0.16 and 4.5, respectively. No relevant floor and ceiling effects were observed. CONCLUSION: The Greek CEDL has good reliability, validity and interpretability. It can be used to evaluate participation in Greek young children with CP. Future studies should investigate the validity of this tool in longer periods and its responsiveness to intervention.

Effectiveness of therapeutic interventions on participation in children with cerebral palsy: A systematic review and meta-analysis.

BACKGROUND: Participation in life activities is an integral part of health and a main outcome of rehabilitation services for children and adolescents with disabilities. However, there is still no consensus on the most effective way to improve participation. The aim of this systematic review is to determine the effectiveness of therapeutic interventions on participation outcomes of children with cerebral palsy (CP). METHODS: A systematic review was conducted, searching the databases PubMed, Cochrane Library, Science Direct, Web of Science and Scopus for randomized controlled trials (RCTs), between 2001 and 2023. Studies were eligible for inclusion if they evaluated children with CP undergoing any intervention and using any tool measuring participation as an outcome measure. A meta-analysis of treatment effect was conducted. A sensitivity analysis was conducted to identify the effect on participation when intervention targeted different International Classification of Functioning (ICF) domains. RESULTS: A total of 1572 records were identified. Eight RCTs including 384 children (195 in the intervention group and 189 in the control group) were included in the systematic review and in the meta-analysis. A sensitivity analysis showed that interventions focusing on participation significantly improved participation; standardized mean difference (1.83; 95% CI: 1.33-2.32; Z = 7.21; P < 0.00001). When other types of interventions, that is, focusing on body functions and structures or activities, were used, then participation was not favourably affected. INTERPRETATION: Interventions primarily targeting barriers to participation across several ICF domains have a greater influence on enhancing participation. Interventions aimed at enhancing specific motor skills, including gross and fine motor function or strength, do not necessarily have a positive impact on participation.

European Respiratory Society statement on airway clearance techniques in adults with bronchiectasis.

Airway clearance techniques (ACTs) are part of the main management strategy for patients with bronchiectasis. Despite being a priority for patients, accessibility, implementation and reporting of ACTs are variable in clinical settings and research studies. This European Respiratory Society statement summarises current knowledge about ACTs in adults with bronchiectasis and makes recommendations to improve the future evidence base. A task force of 14 experts and two patient representatives (10 countries) determined the scope of this statement through consensus and defined six questions. The questions were answered based on systematic searches of the literature. The statement provides a comprehensive review of the physiological rationale for ACTs in adults with bronchiectasis, and the mechanisms of action along with the advantages and disadvantages of each ACT. Evidence on ACTs in clinical practice indicates that the most frequently used techniques are active cycle of breathing techniques, positive expiratory pressure devices and gravity-assisted drainage, although there is limited evidence on the type of ACTs used in specific countries. A review of 30 randomised trials for the effectiveness of ACTs shows that these interventions increase sputum clearance during or after treatment, reduce the impact of cough and the risk of exacerbations, and improve health-related quality of life. Furthermore, strategies for reducing the risk of bias in future studies are proposed. Finally, an exploration of patients' perceptions, barriers and enablers related to this treatment is also included to facilitate implementation and adherence to ACTs.

Psychometrics of health-related quality of life questionnaires in bronchiectasis: a systematic review and meta-analysis.

INTRODUCTION: Understanding the psychometric properties of health-related quality of life (HRQoL) questionnaires can help inform selection in clinical trials. Our objective was to assess the psychometric properties of HRQoL questionnaires in bronchiectasis using a systematic review and meta-analysis of the literature. METHODS: A literature search was conducted. HRQoL questionnaires were assessed for psychometric properties (reliability, validity, minimal clinically important difference (MCID) and floor/ceiling effects). Meta-analyses assessed the associations of HRQoL with clinical measures and responsiveness of HRQoL in clinical trials. RESULTS: 166 studies and 12 HRQoL questionnaires were included. The Bronchiectasis Health Questionnaire (BHQ), Leicester Cough Questionnaire (LCQ), Chronic Obstructive Pulmonary Disease (COPD) Assessment Test (CAT) and Medical Outcomes Study 36-item Short-Form Health Survey (SF-36) had good internal consistency in all domains reported (Cronbach's α≥0.7) across all studies, and the Quality of Life-Bronchiectasis (QOL-B), St George's Respiratory Questionnaire (SGRQ), Chronic Respiratory Disease Questionnaire (CRDQ) and Seattle Obstructive Lung Disease Questionnaire (SOLQ) had good internal consistency in all domains in the majority of (but not all) studies. BHQ, SGRQ, LCQ and CAT had good test-retest reliability in all domains reported (intraclass correlation coefficient ≥0.7) across all studies, and QOL-B, CRDQ and SOLQ had good test-retest reliability in all domains in the majority of (but not all) studies. HRQoL questionnaires were able to discriminate between demographics, important markers of clinical status, disease severity, exacerbations and bacteriology. For HRQoL responsiveness, there was a difference between the treatment and placebo effect. CONCLUSIONS: SGRQ was the most widely used HRQoL questionnaire in bronchiectasis studies and it had good psychometric properties; however, good psychometric data are emerging on the bronchiectasis-specific HRQoL questionnaires QOL-B and BHQ. Future studies should focus on the medium- to long-term test-retest reliability, responsiveness and MCID in these HRQoL questionnaires which show potential in bronchiectasis.

Physical Inactivity in Pulmonary Sarcoidosis.

PURPOSE: Reduced physical activity in many chronic diseases is consistently associated with increased morbidity. Little is known about physical activity in sarcoidosis. The aim of this study was to objectively assess physical activity in patients with pulmonary sarcoidosis and investigate its relationship with lung function, exercise capacity, symptom burden, and health status. METHODS: Physical activity was assessed over one week in 15 patients with pulmonary sarcoidosis and 14 age-matched healthy controls with a tri-axial accelerometer (ActivPal™) and the International Physical Activity Questionnaire (IPAQ). All participants underwent pulmonary function tests, 6-min walk test (6MWT) and completed the Fatigue Assessment Scale (FAS), Medical Research Council (MRC) Dyspnoea Scale and the King's Sarcoidosis Questionnaire (KSQ). RESULTS: Patients with sarcoidosis had significantly lower daily step counts than healthy controls; mean (SD) 5624 (1875) versus 10,429 (2942) steps (p < 0.01) and a trend towards fewer sit-to-stand transitions each day (p = 0.095). Only two patients (13%) self-reported undertaking vigorous physical activity (IPAQ) compared to half of healthy individuals (p < 0.01). Daily step count was significantly associated with 6MWT distance in sarcoidosis (r = 0.634, p = 0.01), but not with forced vital capacity (r = 0.290), fatigue (r = 0.041), dyspnoea (r = -0.466) or KSQ health status (r = 0.099-0.484). Time spent upright was associated with fatigue (r = -0.630, p = 0.012) and health status (KSQ Lung scores r = 0.524, p = 0.045), and there was a significant correlation between the number of sit-to-stand transitions and MRC dyspnoea score (r = -0.527, p = 0.044). CONCLUSION: Physical activity is significantly reduced in sarcoidosis and is associated with reduced functional exercise capacity (6MWD). Fatigue, exertional symptoms and health status were more closely associated with time spent upright and the number of bouts of physical activity, as compared to step counts. Further studies are warranted to identify the factors that determine different physical activity profiles in sarcoidosis.

The development and validation of the Bronchiectasis Health Questionnaire.

Health-related quality of life or health status is significantly impaired in bronchiectasis. There is a paucity of brief, simple-to-use, disease-specific health status measures. The aim of this study was to develop and validate the Bronchiectasis Health Questionnaire (BHQ), a new health status measure that is brief and generates a single overall score.Patients with bronchiectasis were recruited from two outpatient clinics, during a clinically stable stage. The development of the questionnaire followed three phases: item generation and item reduction using Rasch analysis, validation, and repeatability testing. The BHQ was translated into 11 languages using standardised methodology.206 patients with bronchiectasis completed a preliminary 65-item questionnaire. 55 items were removed due to redundancy or poor fit to the Rasch model. The final version of the BHQ consisted of 10 items. Internal consistency was good (Cronbach's α=0.85). Convergent validity of the BHQ with the St George's Respiratory Questionnaire was high (r= -0.82; p<0.001) and moderate with lung function (forced expiratory volume in 1 s % predicted r= -0.27; p=0.001). There was a significant association between BHQ scores and number of exacerbations of bronchiectasis in the last 12 months (p<0.001), hospital admissions (p=0.001) and computed tomography scan bronchiectasis pulmonary lobe counts (p<0.001). BHQ scores were significantly worse in patients with sputum bacterial colonisation versus no colonisation (p=0.048). The BHQ was highly repeatable after 2 weeks (intraclass correlation coefficient 0.89).The BHQ is a brief, valid and repeatable, self-completed health status questionnaire for bronchiectasis that generates a single total score. It can be used in the clinic to assess bronchiectasis from the patient's perspective.

The Objective Assessment of Cough Frequency in Bronchiectasis.

INTRODUCTION: Cough in bronchiectasis is associated with significant impairment in health status. This study aimed to quantify cough frequency objectively with a cough monitor and investigate its relationship with health status. A secondary aim was to identify clinical predictors of cough frequency. METHODS: Fifty-four patients with bronchiectasis were compared with thirty-five healthy controls. Objective 24-h cough, health status (cough-specific: Leicester Cough Questionnaire LCQ and bronchiectasis specific: Bronchiectasis Health Questionnaire BHQ), cough severity and lung function were measured. The clinical predictors of cough frequency in bronchiectasis were determined in a multivariate analysis. RESULTS: Objective cough frequency was significantly raised in patients with bronchiectasis compared to healthy controls [geometric mean (standard deviation)] 184.5 (4.0) vs. 20.6 (3.2) coughs/24-h; mean fold-difference (95% confidence interval) 8.9 (5.2, 15.2); p < 0.001 and they had impaired health status. There was a significant correlation between objective cough frequency and subjective measures; LCQ r = -0.52 and BHQ r = -0.62, both p < 0.001. Sputum production, exacerbations (between past 2 weeks to 12 months) and age were significantly associated with objective cough frequency in multivariate analysis, explaining 52% of the variance (p < 0.001). There was no statistically significant association between cough frequency and lung function. CONCLUSIONS: Cough is a common and significant symptom in patients with bronchiectasis. Sputum production, exacerbations and age, but not lung function, were independent predictors of cough frequency. Ambulatory objective cough monitoring provides novel insights and should be further investigated as an outcome measure in bronchiectasis.

The validity of health-related quality of life questionnaires in bronchiectasis: a systematic review and meta-analysis.

BACKGROUND: A range of questionnaires have been used to assess health-related quality of life (HRQOL) in bronchiectasis. A systematic review was conducted to evaluate their psychometric properties and assess associations between HRQOL and clinical measures. METHODS: Five electronic databases were searched. Studies eligible for inclusion were those that investigated the validity of HRQOL questionnaires and/or their association with other outcomes in adults with bronchiectasis. Patients with cystic fibrosis were excluded. The identified questionnaires were assessed for convergent, discriminant and cross-cultural translation validity; missing data, floor and ceiling effects, internal consistency, responsiveness and test-retest reliability. A meta-analysis was conducted to estimate the strength of associations between HRQOL and clinical measures. RESULTS: From 1918 studies identified, 43 studies were included in the systematic review, of which 38 were suitable for the meta-analysis. Nine HRQOL questionnaires were identified, with the most widely used being: St George's Respiratory Questionnaire, Leicester Cough Questionnaire, Quality of Life-Bronchiectasis and Short Form-36. HRQOL questionnaires had moderate to good internal consistency and good test-retest reliability. Only 8 of 18 studies that used translated HRQOL questionnaires reported or referred to the validity of the translated questionnaire. There was a stronger correlation (mean r (95% CI)) between HRQOL and subjective outcome measures, such as dyspnoea (0.55 (0.41 to 0.68)) and fatigue (0.42 (0.23 to 0.58)) compared with objective measures; exercise capacity (-0.41 (-0.54 to -0.24)), FEV1% predicted (-0.31 (-0.40 to -0.23)) and extent of bronchiectasis on CT scan (0.35 (0.03 to 0.61)); all p<0.001. CONCLUSIONS: This review supports most HRQOL questionnaires used in bronchiectasis have good psychometric properties. There was a weak to moderate association between HRQOL and objective outcome measures. This suggests that HRQOL questionnaires assess a unique aspect of health not captured by objective measures.

Prescribing preferences and availability of nebulisers and inhalers for inhaled medications in bronchiectasis: results of a specialist survey.

Specialists caring for people with bronchiectasis recommend specialised nebulisers for inhaled antibiotics, but are often limited by availability and cost of nebulisation devices https://bit.ly/40FvFdZ.

Development of a core outcome set for physiotherapy trials in adults with bronchiectasis (the COS-PHyBE study): A Delphi study and consensus meeting.

Background: Physiotherapy is recommended for bronchiectasis management, but there is disparity in evidence supporting its use. This is partly because of inconsistency and poor reporting of outcomes in available studies. A Core Outcome Set (COS) may improve trial consistency and decrease reporting bias. This study aimed to develop a COS for physiotherapy clinical trials in adults with bronchiectasis. Methods: A comprehensive list of outcomes was developed using a systematic review and qualitative semi-structured interviews with patients with bronchiectasis and physiotherapists.An international two-round online Delphi survey was conducted. Outcomes scored 7-9 (crucial) by ≥ 70 % of participants and 1-3 (not that important) by ≤ 15 % of participants from each stakeholder in the Likert scale were nominated for inclusion in the COS. Nominated outcomes and those considered crucial by only one of the stakeholders' groups were further discussed and voted in an international consensus meeting. Results: A list of 137 outcomes was generated; 104 from literature and 33 from interviews. A Delphi survey containing 59 outcomes was completed by 171 participants from 20 countries. After the consensus meeting, representatives agreed on seven outcomes: health-related quality of life, respiratory symptoms, physical functioning, emotional and psychological functioning, fatigue, adherence to treatment, and functional exercise capacity. Conclusion: A minimum set of seven outcomes are recommended to be included as measurements in future trials evaluating physiotherapy interventions for bronchiectasis.

The IMPROVE trial: study protocol for a pragmatic cluster randomised controlled trial to assess the effectiveness of using lay health workers to improve uptake and completion of pulmonary rehabilitation in patients with chronic obstructive pulmonary disease.

BACKGROUND: Pulmonary rehabilitation (PR) is a programme of exercise and education and the most effective treatment for the symptoms and disability associated with chronic obstructive pulmonary disease. However, the benefits of PR are limited by poor uptake and completion. This trial will determine whether using trained volunteer lay health workers, called "PR buddies," improves uptake and completion of PR and is cost-effective. This trial protocol outlines the methods for evaluating effectiveness, cost-effectiveness, and acceptability. METHODS: The IMPROVE trial is a pragmatic, open, cluster randomised controlled trial planned in 38 PR services across England and Wales. PR services will be randomised to either intervention arm-offering support from PR buddies to patients with chronic obstructive pulmonary disease-or to usual care as the control arm. PR staff in trial sites randomised to the intervention arm will receive training in recruiting and training PR buddies. They will deliver training to volunteers, recruited from among people who have recently completed PR in their service. The 3-day PR-buddy training programme covers communication skills, confidentiality, boundaries of the PR-buddy role and behaviour change techniques to help patients overcome obstacles to attending PR. An internal pilot will test the implementation of the trial in eight sites (four intervention sites and four in control arm). The primary outcome of the trial is the uptake and completion of PR. A process evaluation will investigate the acceptability of the intervention to patients, PR staff and the volunteer PR buddies, and intervention fidelity. We will also conduct a cost-effectiveness analysis. DISCUSSION: Improving outcomes for chronic obstructive pulmonary disease and access to PR are priorities for the UK National Health Service (NHS) in its long-term plan. The trial hypothesis is that volunteer PR buddies, who are recruited and trained by local PR teams, are an effective and cost-effective way to improve the uptake and completion rates of PR. The trial is pragmatic, since it will test whether the intervention can be incorporated into NHS PR services. Information obtained in this trial may be used to influence policy on the use of PR buddies in PR and other similar services in the NHS. TRIAL REGISTRATION: ISRCTN12658458. Registered on 23/01/2023.

Kinematic and Temporospatial Changes in Children with Cerebral Palsy during the Initial Stages of Gait Development.

PURPOSE: To identify changes in the gait kinematics and temporospatial parameters of children with bilateral Cerebral Palsy (CP) at 8 months after the onset of independent walking and identify differences to Typical Development (TD) children at the onset of independent walking and at 8 months follow up. METHOD: Sixteen children with bilateral CP, GMFCS levels I and II, and 15 TD children were recruited. Gait kinematics and temporospatial parameters were recorded using a 3-D gait analysis system; the sagittal plane of the lower limb joints was analyzed. Baseline measurements were recorded at the individual's onset of independent walking and follow up was after 8 months. RESULTS: Compared to baseline, children with bilateral CP demonstrated increased (mean difference ± SE) plantar flexion (11.79 ± 2.96), single support (0.04 ± 0.01), step length (0.2 ± 0.05) and stride length (0.4 ± 0.09), at follow up; all p < .05. Compared to TD children, they also had lower gait speed (0.16 ± 0.05), higher single support (0.02 ± 0.01) and lower maximum knee extension (9.14 ± 4.49) during the swing phase, at baseline and follow up (0.1 ± 0.04, 0.05 ± 0.01, 23.04 ± 4.17, respectively); all p < .05. CONCLUSION: There are changes in the sagittal plane kinematics and temporospatial parameters of the gait during the first 8 months of independent walking. These indicate gait maturation changes and highlight the impact of walking experience on the gait characteristics of children with bilateral CP.

Translation and linguistic validation of the Bronchiectasis Health Questionnaire (BHQ) into Danish.

INTRODUCTION: Patients with bronchiectasis suffer from breathlessness, cough and sputum production, which impairs quality of life. The Bronchiectasis Health Questionnaire (BHQ), a short and disease-specific questionnaire, has previously been developed and validated in English. The aim of this study was to translate and validate the BHQ in Danish, using established cross-cultural validation methods. METHODS: To create a Danish version of the questionnaire, this study used linguistic validation, a pilot study in 17 patients and forward-backward translation. Thereafter, 111 patients with bronchiectasis were asked to complete the BHQ (BHQ1) as well as the St George Respiratory Questionnaire (SGRQ), and one in three patients were asked to repeat the BHQ after two weeks had passed (BHQ2). RESULTS: There was a significant convergent validity between the BHQ1 and the SGRQ (ρ = -0,826, p = 0.0001), a satisfactory correlation coefficient between the BHQ1 and the BHQ2 (0.739) and a lower limit of agreement of -15.96 and 20.56 in the Bland Altmann plot. CONCLUSIONS: The BHQ is translated and validated in Danish and retains good validity properties. This questionnaire is ready for use in daily clinical practice among Danish-speaking patients. FUNDING: none. TRIAL REGISTRATION: not relevant.

Passive range of motion changes in young children with spastic diplegia. A study during the initial stages of independent walking.

PURPOSE: To investigate how the onset of independent walking in children with Cerebral Palsy (CP) influences the Passive Range of Motion (PROM) of lower limb joints. METHOD: Sixteen children with CP, GMFCS levels I and II, and 16 Typical Development (TD) children who had just begun independent walking participated in the study. The PROM of the hip abduction and external rotation, knee extension, popliteal angle, and ankle dorsiflexion was recorded with a goniometer at the onset of independent walking and 8 months later. A repeated-measures two-way ANOVA was conducted to compare the main effects of "walking experience" and "group of children" and the interaction effect between them on PROM of lower extremities' joints. RESULTS: The effect of "group of children" in the PROM was significant; differences were observed between children with CP and TD children for all joints (p< 0.05). The effect of "walking experience" did not have a significant impact on PROM changes and the interaction effect of "group of children" and "walking experience" was also not significant for all the variables. CONCLUSIONS: The alternative gait pattern spontaneously adopted by children with CP does not significantly impact their PROM during the initial stages of walking development. The lower PROM in highly functional children with CP compared to TD children cannot be attributed to gait initiation with an "atypical" pattern, but possibly to other factors such as reduced voluntary movement and age.