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AIMS: The aim was to evaluate whether standardised exercise performance during the incremental shuttle walk test (ISWT) can be used to assess disease severity in children and young people (CYP) with chronic conditions, through (1) identifying the most appropriate paediatric normative reference equation for the ISWT, (2) assessing how well CYP with haemophilia and cystic fibrosis (CF) perform against the values predicted by the best fit reference equation and (3) evaluating the association between standardised ISWT performance and disease severity. METHODS: A cross-sectional analysis was carried out using existing data from two independent studies (2018-2019) at paediatric hospitals in London,UK. CYP with haemophilia (n=35) and CF (n=134) aged 5-18 years were included. Published reference equations for standardising ISWT were evaluated through a comparison of populations, and Bland-Altman analysis was used to assess the level of agreement between distances predicted by each equation. Associations between ISWT and disease severity were assessed with linear regression. RESULTS: Three relevant reference equations were identified for the ISWT that standardised performance based on age, sex and body mass index (Vardhan, Lanza, Pinho). A systematic proportional bias of standardised ISWT was observed in all equations, most pronounced with Vardhan and Lanza; the male Pinho equation was identified as most appropriate. On average, CYP with CF and haemophilia performed worse than predicted by the Pihno equation, although the range was wide. Standardised ISWT, and not ISWT distance alone, was significantly associated with forced expiratory volume in 1 s in CYP with CF. Standardised ISWT in CYP with haemophilia was slightly associated with haemophilia joint health score, but this was not significant. CONCLUSIONS: ISWT performance may be useful in a clinic to identify those with worsening disease, but only when performance is standardised against a healthy reference population. The development of validated global reference equations is necessary for more robust assessment.
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Fibrosis Quística , Hemofilia A , Humanos , Masculino , Niño , Adolescente , Prueba de Paso , Estudios Transversales , Tolerancia al Ejercicio , Enfermedad Crónica , Gravedad del Paciente , Prueba de Esfuerzo , CaminataRESUMEN
Sleep disordered breathing is commonly treated with positive airway pressure therapy. Positive airway pressure therapy is delivered via a tight-fitting mask with common side effects including: leak, ineffective treatment, residual sleep disordered breathing, eye irritation, nasal congestion, pressure ulcers and poor concordance with therapy. This systematic review and meta-analysis aimed to identify the effectiveness of current treatment strategies for managing side effects associated with positive airway pressure therapy. Five databases were searched and 10,809 articles were screened, with 36 articles included in the review. Studies investigated: dressings, nasal spray/douche, chin straps, heated humidification and interfaces. No intervention either improved or detrimentally affected: positive airway pressure concordance, Epworth Sleepiness Score, residual apnoea hypopnea index or interface leak. The review was limited by study heterogeneity, particularly for outcome measures. Additionally, patient demographics were not reported, making it difficult to apply the findings to a broad clinical population. This review highlights the paucity of evidence supporting treatment strategies to manage side effects of positive airway pressure therapy.
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Síndromes de la Apnea del Sueño , Humanos , Síndromes de la Apnea del Sueño/terapia , Evaluación de Resultado en la Atención de Salud , Calor , Presión de las Vías Aéreas Positiva Contínua/efectos adversosRESUMEN
INTRODUCTION: Boys with haemophilia (BwH) have improved health outcomes. Measures of physical function in haemophilia are not challenging or sensitive enough to reflect physical limitations or guide rehabilitation. To identify meaningful tests, we aimed to: evaluate the performance of BwH on two physical performance measures: iSTEP and 10 m-ISWT; identify factors which predict performance and compare BwH to their unaffected peers. METHODS: BwH completed both iSTEP and 10 m-ISWT. Disease severity, age, BMI, HJHS, lower limb muscle torque, time spent in moderate to vigorous physical activity, sedentary time, were included as factors to predict performance. Results were compared to unaffected peers. RESULTS: 43 boys median age 10 (10 mild/moderate, 26 severe, 7 inhibitors) were recruited. BwH were less likely to complete the iSTEP and performed less well on the 10 m-ISWT than age matched peers. Ceiling effects were apparent for iSTEP, but not the 10 m-ISWT test. Age was the only significant predictor for performance in the iSTEP, with older boys being more likely to achieve a higher level or complete the test. Greater age, lower BMI, milder disease severity and more time spent in MVPA all predicted better performance on the 10 m-ISWT, with BMI and habitual physical activity a potential rehabilitation focus for underperforming individuals. HJHS and muscle strength did not predict performance on either test. CONCLUSION: Despite the space need to conduct the 10 m-ISWT, it appears to be a superior performance measure than the iSTEP in BwH and provides clinically meaningful information, which can be interpreted using age-specific normative reference equations.
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Hemofilia A , Masculino , Humanos , Niño , Ejercicio Físico , Caminata/fisiologíaRESUMEN
BACKGROUND: Cystic fibrosis (CF) is commonly characterised by thick respiratory mucus. From diagnosis, people with CF are prescribed daily physiotherapy, including airway clearance techniques (ACTs). ACTs consume a large proportion of treatment time, yet the efficacy and effectiveness of ACTs are poorly understood. This study aimed to evaluate associations between the quality and quantity of ACTs and lung function in children and young people with CF. METHODS: Project Fizzyo, a longitudinal observational cohort study in the UK, used remote monitoring with electronic pressure sensors attached to four different commercial ACT devices to record real-time, breath-by-breath pressure data during usual ACTs undertaken at home over 16â months in 145 children. ACTs were categorised either as conformant or not with current ACT recommendations based on breath pressure and length measurements, or as missed treatments if not recorded. Daily, weekly and monthly associations between ACT category and lung function were investigated using linear mixed effects regression models adjusting for clinical confounders. RESULTS: After exclusions, 45 224 ACT treatments (135 individuals) and 21 069â days without treatments (141 individuals) were analysed. The mean±sd age of participants was 10.2±2.9â years. Conformant ACTs (21%) had significantly higher forced expiratory volume in 1â s (FEV1) (mean effect size 0.23 (95% CI 0.19-0.27) FEV1 % pred per treatment) than non-conformant (79%) or missed treatments. There was no benefit from non-conformant or missed treatments and no significant difference in FEV1 between them (mean effect size 0.02 (95% CI -0.01-0.05) FEV1 % pred per treatment). CONCLUSIONS: ACTs are beneficial when performed as recommended, but most people use techniques that do not improve lung function. Work is needed to monitor and improve ACT quality and to increase the proportion of people doing effective airway clearance at home.
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Fibrosis Quística , Humanos , Niño , Adolescente , Fibrosis Quística/terapia , Volumen Espiratorio Forzado , Modelos Lineales , Prednisona , EsputoRESUMEN
BACKGROUND: Clinical outcomes are normally captured less frequently than data from remote technologies, leaving a disparity in volumes of data from these different sources. To align these data, flexible polynomial regression was investigated to estimate personalised trends for a continuous outcome over time. METHODS: Using electronic health records, flexible polynomial regression models inclusive of a 1st up to a 4th order were calculated to predict forced expiratory volume in 1 s (FEV1) over time in children with cystic fibrosis. The model with the lowest AIC for each individual was selected as the best fit. The optimal parameters for using flexible polynomials were investigated by comparing the measured FEV1 values to the values given by the individualised polynomial. RESULTS: There were 8,549 FEV1 measurements from 267 individuals. For individuals with > 15 measurements (n = 178), the polynomial predictions worked well; however, with < 15 measurements (n = 89), the polynomial models were conditional on the number of measurements and time between measurements. The method was validated using BMI in the same population of children. CONCLUSION: Flexible polynomials can be used to extrapolate clinical outcome measures at frequent time intervals to align with daily data captured through remote technologies.
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Fibrosis Quística , Modelos Estadísticos , Niño , Humanos , Volumen Espiratorio Forzado , Fibrosis Quística/terapiaRESUMEN
BACKGROUND: Cystic fibrosis (CF) is an inherited life-limiting disorder. Over time persistent infection and inflammation within the lungs contribute to severe airway damage and loss of respiratory function. Chest physiotherapy, or airway clearance techniques (ACTs), are integral in removing airway secretions and initiated shortly after CF diagnosis. Conventional chest physiotherapy (CCPT) generally requires assistance, while alternative ACTs can be self-administered, facilitating independence and flexibility. This is an updated review. OBJECTIVES: To evaluate the effectiveness (in terms of respiratory function, respiratory exacerbations, exercise capacity) and acceptability (in terms of individual preference, adherence, quality of life) of CCPT for people with CF compared to alternative ACTs. SEARCH METHODS: We used standard, extensive Cochrane search methods. The latest search was 26 June 2022. SELECTION CRITERIA: We included randomised or quasi-randomised controlled trials (including cross-over design) lasting at least seven days and comparing CCPT with alternative ACTs in people with CF. DATA COLLECTION AND ANALYSIS: We used standard Cochrane methods. Our primary outcomes were 1. pulmonary function tests and 2. number of respiratory exacerbations per year. Our secondary outcomes were 3. quality of life, 4. adherence to therapy, 5. cost-benefit analysis, 6. objective change in exercise capacity, 7. additional lung function tests, 8. ventilation scanning, 9. blood oxygen levels, 10. nutritional status, 11. mortality, 12. mucus transport rate and 13. mucus wet or dry weight. We reported outcomes as short-term (seven to 20 days), medium-term (more than 20 days to up to one year) and long-term (over one year). MAIN RESULTS: We included 21 studies (778 participants) comprising seven short-term, eight medium-term and six long-term studies. Studies were conducted in the USA (10), Canada (five), Australia (two), the UK (two), Denmark (one) and Italy (one) with a median of 23 participants per study (range 13 to 166). Participant ages ranged from newborns to 45 years; most studies only recruited children and young people. Sixteen studies reported the sex of participants (375 males; 296 females). Most studies compared modifications of CCPT with a single comparator, but two studies compared three interventions and another compared four interventions. The interventions varied in the duration of treatments, times per day and periods of comparison making meta-analysis challenging. All evidence was very low certainty. Nineteen studies reported the primary outcomes forced expiratory volume in one second (FEV1)and forced vital capacity (FVC), and found no difference in change from baseline in FEV1 % predicted or rate of decline between groups for either measure. Most studies suggested equivalence between CCPT and alternative ACTs, including positive expiratory pressure (PEP), extrapulmonary mechanical percussion, active cycle of breathing technique (ACBT), oscillating PEP devices (O-PEP), autogenic drainage (AD) and exercise. Where single studies suggested superiority of one ACT, these findings were not corroborated in similar studies; pooled data generally concluded that effects of CCPT were comparable to those of alternative ACTs. CCPT versus PEP We are uncertain whether CCPT improves lung function or has an impact on the number of respiratory exacerbations per year compared with PEP (both very low-certainty evidence). There were no analysable data for our secondary outcomes, but many studies provided favourable narrative reports on the independence achieved with PEP mask therapy. CCPT versus extrapulmonary mechanical percussion We are uncertain whether CCPT improves lung function compared with extrapulmonary mechanical percussions (very low-certainty evidence). The annual rate of decline in average forced expiratory flow between 25% and 75% of FVC (FEF25-75) was greater with high-frequency chest compression compared to CCPT in medium- to long-term studies, but there was no difference in any other outcome. CCPT versus ACBT We are uncertain whether CCPT improves lung function compared to ACBT (very low-certainty evidence). Annual decline in FEF25-75 was worse in participants using the FET component of ACBT only (mean difference (MD) 6.00, 95% confidence interval (CI) 0.55 to 11.45; 1 study, 63 participants; very low-certainty evidence). One short-term study reported that directed coughing was as effective as CCPT for all lung function outcomes, but with no analysable data. One study found no difference in hospital admissions and days in hospital for exacerbations. CCPT versus O-PEP We are uncertain whether CCPT improves lung function compared to O-PEP devices (Flutter device and intrapulmonary percussive ventilation); however, only one study provided analysable data (very low-certainty evidence). No study reported data for number of exacerbations. There was no difference in results for number of days in hospital for an exacerbation, number of hospital admissions and number of days of intravenous antibiotics; this was also true for other secondary outcomes. CCPT versus AD We are uncertain whether CCPT improves lung function compared to AD (very low-certainty evidence). No studies reported the number of exacerbations per year; however, one study reported more hospital admissions for exacerbations in the CCPT group (MD 0.24, 95% CI 0.06 to 0.42; 33 participants). One study provided a narrative report of a preference for AD. CCPT versus exercise We are uncertain whether CCPT improves lung function compared to exercise (very low-certainty evidence). Analysis of original data from one study demonstrated a higher FEV1 % predicted (MD 7.05, 95% CI 3.15 to 10.95; P = 0.0004), FVC (MD 7.83, 95% CI 2.48 to 13.18; P = 0.004) and FEF25-75 (MD 7.05, 95% CI 3.15 to 10.95; P = 0.0004) in the CCPT group; however, the study reported no difference between groups (likely because the original analysis accounted for baseline differences). AUTHORS' CONCLUSIONS: We are uncertain whether CCPT has a more positive impact on respiratory function, respiratory exacerbations, individual preference, adherence, quality of life, exercise capacity and other outcomes when compared to alternative ACTs as the certainty of the evidence is very low. There was no advantage in respiratory function of CCPT over alternative ACTs, but this may reflect insufficient evidence rather than real equivalence. Narrative reports indicated that participants prefer self-administered ACTs. This review is limited by a paucity of well-designed, adequately powered, long-term studies. This review cannot yet recommend any single ACT above others; physiotherapists and people with CF may wish to try different ACTs until they find an ACT that suits them best.
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Fibrosis Quística , Adolescente , Niño , Humanos , Recién Nacido , Persona de Mediana Edad , Fibrosis Quística/complicaciones , Drenaje Postural/métodos , Modalidades de Fisioterapia , Calidad de Vida , Terapia Respiratoria/métodosRESUMEN
Background: Autism spectrum disorders (ASDs) are diagnosed by social communication difficulties strong, narrow interests, and repetitive stereotyped behavior. An apparently-elevated prevalence of ASD at a major UK hemophilia center warranted investigation. Objectives: To screen boys with hemophilia for difficulties in social communication and executive function and identify the prevalence and risk factors for ASD. Methods: Parents of boys with hemophilia aged 5 to 16 years completed the Social Communication Questionnaire, Children's Communication Checklist, and the Behavior Rating Inventory of executive function. Prevalence and potential risk factors for ASD were evaluated. Boys with an existing diagnosis of ASD did not complete questionnaires, but were included in the prevalence analysis. Results: Negative scores on all 3 questionnaires were observed for 60 of 79 boys. Positive scores on 1, 2, and 3 questionnaires were seen in 12 of 79, 3 of 79, and 4 of 79 boys, respectively. In addition to the 11 of 214 boys with a prior ASD diagnosis, 3 further boys were diagnosed with ASD, yielding a prevalence of 14 (6.5%) of 214, greater than that of boys in the UK general population. Premature birth was linked to having ASD, but did not fully explain the increased prevalence with more boys born <37 weeks scoring positively on the Social Communications Questionnaire and Children's Communication Checklist compared with those born at term. Conclusion: This study identified an increased prevalence of ASD at 1 UK hemophilia center. Prematurity was identified as a risk factor but did not fully explain the higher prevalence of ASD. Further investigation in the wider national/global hemophilia communities is warranted to determine whether this is an isolated finding.
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Replacing traditional airway clearance therapy (tACT) with exercise (ExACT) in people with cystic fibrosis (pwCF) is a top research priority. A UK-based e-Delphi consensus was performed to inform the type(s), duration and intensity of ExACT. The expert panel comprised CF physiotherapists, doctors, pwCF and parents/partners. Exercise ACT was considered to be aerobic activity, of at least 20 min duration and intense enough to elicit deep breathing. Consensus was reached that assessment breaths, coughs and huffs should accompany exercise to remove loose secretions, with support for trials to investigate ExACT versus tACT during times of stable disease but not pulmonary exacerbations.
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Fibrosis Quística , Terapia por Ejercicio , Humanos , Cuidadores , Fibrosis Quística/terapia , Terapia Respiratoria , Reino Unido , Técnica Delphi , Internet , Encuestas y Cuestionarios , Personal de Salud , Pacientes , ConsensoRESUMEN
Children and young people with CF (CYPwCF) get advice about using positive expiratory pressure (PEP) or oscillating PEP (OPEP) devices to clear sticky mucus from their lungs. However, little is known about the quantity (number of treatments, breaths, or sets) or quality (breath pressures and lengths) of these daily airway clearance techniques (ACTs) undertaken at home. This study used electronic pressure sensors to record real time breath-by-breath data from 145 CYPwCF (6-16y) during routine ACTs over 2 months. ACT quantity and quality were benchmarked against individual prescriptions and accepted recommendations for device use. In total 742,084 breaths from 9,081 treatments were recorded. Individual CYPwCF maintained consistent patterns of ACT quantity and quality over time. Overall, 60% of CYPwCF did at least half their prescribed treatments, while 27% did fewer than a quarter. About 77% of pre-teens did the right number of daily treatments compared with only 56% of teenagers. CYPwCF usually did the right number of breaths. ACT quality (recommended breath length and pressure) varied between participants and depended on device. Breath pressures, lengths and pressure-length relationships were significantly different between ACT devices. PEP devices encouraged longer breaths with lower pressures, while OPEP devices encouraged shorter breaths with higher pressures. More breaths per treatment were within advised ranges for both pressure and length using PEP (30-31%) than OPEP devices (1-3%). Objective measures of quantity and quality may help to optimise ACT device selection and support CYPwCF to do regular effective ACTs.
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Fibrosis Quística , Adolescente , Niño , Humanos , Fibrosis Quística/terapia , Volumen Espiratorio Forzado , Moco , Ejercicios RespiratoriosRESUMEN
Machine learning (ML) holds great potential for predicting clinical outcomes in heterogeneous chronic respiratory diseases (CRD) affecting children, where timely individualised treatments offer opportunities for health optimisation. This paper identifies rate-limiting steps in ML prediction model development that impair clinical translation and discusses regulatory, clinical and ethical considerations for ML implementation. A scoping review of ML prediction models in paediatric CRDs was undertaken using the PRISMA extension scoping review guidelines. From 1209 results, 25 articles published between 2013 and 2021 were evaluated for features of a good clinical prediction model using the Transparent Reporting of a multivariable prediction model for Individual Prognosis Or Diagnosis (TRIPOD) guidelines.Most of the studies were in asthma (80%), with few in cystic fibrosis (12%), bronchiolitis (4%) and childhood wheeze (4%). There were inconsistencies in model reporting and studies were limited by a lack of validation, and absence of equations or code for replication. Clinician involvement during ML model development is essential and diversity, equity and inclusion should be assessed at each step of the ML pipeline to ensure algorithms do not promote or amplify health disparities among marginalised groups. As ML prediction studies become more frequent, it is important that models are rigorously developed using published guidelines and take account of regulatory frameworks which depend on model complexity, patient safety, accountability and liability.
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Lista de Verificación , Modelos Estadísticos , Algoritmos , Niño , Humanos , Aprendizaje Automático , PronósticoRESUMEN
BACKGROUND: There is a lack of functional performance measures for children and young people with haemophilia (CYPwH) with associated control data from typically developing boys (TDB). The literature advocates development of a core set of outcome measures for different chronic conditions. As medical treatment improves, CYPwH are experiencing better outcomes; therefore, more challenging measures are required to monitor physical performance. Such testing is not performed routinely, due to practical and safety concerns. AIM: Evaluate the feasibility, safety and acceptability of select outcome measures as part of a study protocol testing CYPwH; including myometry, 10 metre incremental shuttle walk test (10-m ISWT), iSTEP (an incremental step test, with data from TDB), and 1 week of accelerometry-wear at home. METHODS: Sixty-six boys aged 6-15 years with mild, moderate or severe haemophilia A or B (including inhibitors) attending routine clinics at Great Ormond Street Hospital were approached to participate. Descriptive statistics and content analysis were used to assess outcomes of feasibility, safety and acceptability, which included recruitment/retention rates, protocol completion within routine appointment timeframes, performance testing without serious adverse events/reactions (SAE/SARs), and acceptability to CYPwH of high-level performance measures. RESULTS: Outcomes were met: 43 boys completed testing at clinic review (Jan-Nov 2018) within a 10-month timeframe, retention was 95% at completion of protocol and no SAE/SARs were reported throughout testing. CONCLUSION: Feasibility, safety and acceptability of the study protocol have been established in this population. Both high-level performance tests, iSTEP and 10-m ISWT, were an acceptable addition to boys' routine clinic appointments and could be safe, acceptable choices of outcome measure as part of a core set of tests for CYPwH. Further investigation of the psychometric properties for the iSTEP is now justified, in order for it to be used as a standardised, validated, reliable outcome measure in clinical or research settings. TRIAL REGISTRATION: Retrospectively registered on September 3, 2019, on ClinicalTrials.gov (ID: NCT04076306 ).
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INTRODUCTION: Botulinum neurotoxin-A (BoNT-A) is an accepted treatment modality for the management of hypertonia in children and young people with cerebral palsy (CYPwCP). Nevertheless, there are concerns about the long-term effects of BoNT-A, with a lack of consensus regarding the most meaningful outcome measures to guide its use. Most evidence to date is based on short-term outcomes, related to changes at impairment level (restrictions of body functions and structures), rather than changes in adaptive skills (enabling both activity and participation). The proposed study aims to evaluate clinical and patient reported outcomes in ambulant CYPwCP receiving lower limb BoNT-A injections over a 12-month period within all domains of the WHO's International Classification of Functioning, Disability and Health and health-related quality of life (HRQoL). METHODS AND ANALYSIS: This pragmatic prospective longitudinal observational study will use a one-group repeated measures design. Sixty CYPwCP, classified as Gross Motor Function Classification System (GMFCS) levels I-III, aged between 4 and 18 years, will be recruited from an established movement disorder service in London, UK. Standardised clinical and patient reported outcome measures within all ICF domains; body structures and function, activity (including quality of movement), goal attainment, participation and HRQoL, will be collected preinjection and at 6 weeks, 6 months and up to 12 months postinjection. A representative subgroup of children and carers will participate in a qualitative component of the study, exploring how their experience of BoNT-A treatment relates to clinical outcome measures. ETHICS AND DISSEMINATION: Central London Research Ethics Committee has granted ethics approval (#IRAS 211617 #REC 17/LO/0579). Findings will be disseminated in peer-reviewed publications, conferences and via networks to participants and relevant stakeholders using a variety of accessible formats including social media.
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Toxinas Botulínicas Tipo A , Parálisis Cerebral , Fármacos Neuromusculares , Adolescente , Toxinas Botulínicas Tipo A/uso terapéutico , Parálisis Cerebral/tratamiento farmacológico , Niño , Preescolar , Humanos , Inyecciones Intramusculares , Londres , Extremidad Inferior , Fármacos Neuromusculares/uso terapéutico , Estudios Observacionales como Asunto , Medición de Resultados Informados por el Paciente , Estudios Prospectivos , Calidad de VidaRESUMEN
BACKGROUND: Cystic fibrosis (CF) is a multisystem disease in which the assessment of disease severity based on lung function alone may not be appropriate. The aim of the study was to develop a comprehensive machine-learning algorithm to assess clinical status independent of lung function in children. METHODS: A comprehensive prospectively collected clinical database (Toronto, Canada) was used to apply unsupervised cluster analysis. The defined clusters were then compared by current and future lung function, risk of future hospitalisation, and risk of future pulmonary exacerbation treated with oral antibiotics. A k-nearest-neighbours (KNN) algorithm was used to prospectively assign clusters. The methods were validated in a paediatric clinical CF dataset from Great Ormond Street Hospital (GOSH). RESULTS: The optimal cluster model identified four (A-D) phenotypic clusters based on 12â200 encounters from 530 individuals. Two clusters (A and B) consistent with mild disease were identified with high forced expiratory volume in 1â s (FEV1), and low risk of both hospitalisation and pulmonary exacerbation treated with oral antibiotics. Two clusters (C and D) consistent with severe disease were also identified with low FEV1. Cluster D had the shortest time to both hospitalisation and pulmonary exacerbation treated with oral antibiotics. The outcomes were consistent in 3124 encounters from 171 children at GOSH. The KNN cluster allocation error rate was low, at 2.5% (Toronto) and 3.5% (GOSH). CONCLUSION: Machine learning derived phenotypic clusters can predict disease severity independent of lung function and could be used in conjunction with functional measures to predict future disease trajectories in CF patients.
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Fibrosis Quística , Niño , Análisis por Conglomerados , Fibrosis Quística/diagnóstico , Volumen Espiratorio Forzado , Humanos , Pulmón , Pruebas de Función RespiratoriaRESUMEN
INTRODUCTION: Daily physiotherapy is believed to mitigate the progression of cystic fibrosis (CF) lung disease. However, physiotherapy airway clearance techniques (ACTs) are burdensome and the evidence guiding practice remains weak. This paper describes the protocol for Project Fizzyo, which uses innovative technology and analysis methods to remotely capture longitudinal daily data from physiotherapy treatments to measure adherence and prospectively evaluate associations with clinical outcomes. METHODS AND ANALYSIS: A cohort of 145 children and young people with CF aged 6-16 years were recruited. Each participant will record their usual physiotherapy sessions daily for 16 months, using remote monitoring sensors: (1) a bespoke ACT sensor, inserted into their usual ACT device and (2) a Fitbit Alta HR activity tracker. Real-time breath pressure during ACTs, and heart rate and daily step counts (Fitbit) are synced using specific software applications. An interrupted time-series design will facilitate evaluation of ACT interventions (feedback and ACT-driven gaming). Baseline, mid and endpoint assessments of spirometry, exercise capacity and quality of life and longitudinal clinical record data will also be collected.This large dataset will be analysed in R using big data analytics approaches. Distinct ACT and physical activity adherence profiles will be identified, using cluster analysis to define groups of individuals based on measured characteristics and any relationships to clinical profiles assessed. Changes in adherence to physiotherapy over time or in relation to ACT interventions will be quantified and evaluated in relation to clinical outcomes. ETHICS AND DISSEMINATION: Ethical approval for this study (IRAS: 228625) was granted by the London-Brighton and Sussex NREC (18/LO/1038). Findings will be disseminated via peer-reviewed publications, at conferences and via CF clinical networks. The statistical code will be published in the Fizzyo GitHub repository and the dataset stored in the Great Ormond Street Hospital Digital Research Environment. TRIAL REGISTRATION NUMBER: ISRCTN51624752; Pre-results.
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Fibrosis Quística , Modalidades de Fisioterapia , Adolescente , Factores de Edad , Niño , Estudios de Cohortes , Fibrosis Quística/terapia , Humanos , Estudios Longitudinales , Estudios Observacionales como Asunto , Calidad de VidaRESUMEN
Building on the core syllabus for postgraduate training in respiratory physiotherapy, published in 2014, the European Respiratory Society (ERS) respiratory physiotherapy task force has developed a harmonised and structured postgraduate curriculum for respiratory physiotherapy training. The curriculum outlines the knowledge, skills and attitudes which must be mastered by a respiratory physiotherapist working with adult or paediatric patients, together with guidance for minimal clinical exposures, and forms of learning and assessment. This article presents the rationale, methodology and content of the ERS respiratory physiotherapy curriculum. The full curriculum can be found in the supplementary material.
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OBJECTIVE: To develop paediatric gait standards in healthy children and young people. METHODS: This observational study aims to address the lack of population standards for gait measurements in children. Analysing gait in children affected by neurological or musculoskeletal conditions is an important component of paediatric assessment but is often confounded by developmental changes. The standards presented here do not require clinician expertise to interpret and offer an alternative to developmental tables of normalised gait data. Healthy children aged 1-19 years were recruited from community settings in London and Hertfordshire, UK. The GAITRite walkway was used to record measurements for each child for velocity, cadence, step length, base of support and stance, single and double support (as percentage of gait cycle). We fitted generalised linear additive models for location, scale and shape (gamlss). RESULTS: We constructed percentile charts for seven gait variables measured on 624 (321 males) contemporary healthy children using a gamlss package in R. A clinical application of gait standards was explored. CONCLUSION: Age-related, gender-specific standards for seven gait variables were developed and are presented here. They have a familiar format and can be used clinically to aid diagnoses and to monitor change over time for both medical therapy and natural history of the condition. The clinical example demonstrates the potential of the Great Ormond Street Institute of Child Health Paediatric Gait Centiles to enable meaningful interpretation of change in an individual's performance and describes characteristic features of gait from a specific population throughout childhood.