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Little is known about early predictors of later cystic fibrosis (CF) structural lung disease. This study examined early predictors of progressive structural lung abnormalities in children who completed the Australasian CF Bronchoalveolar Lavage (ACFBAL) clinical trial at age 5-years and participated in an observational follow-up study (CF-FAB).Eight Australian and New Zealand CF centres participated in CF-FAB and provided follow-up chest computed-tomography (CT) scans for children who had completed the ACFBAL study with baseline scans at age 5-years. CT scans were annotated using PRAGMA-CF scoring. Ordinal regression analysis and linear regression were used to investigate associations between PRAGMA-CF (Perth-Rotterdam Annotated Grid Morphometric Analysis for CF) outcomes at follow-up and variables measured during the ACFBAL study.99 out of 157 ACFBAL children (mean±sd age 13±1.5 years) participated in the CF-FAB study. The probability of bronchiectasis at follow-up increased with airway disease severity on the baseline CT scan. In multiple regression (retaining factors at p<0.05) the extent of bronchiectasis at follow-up was associated with baseline atelectasis (OR 7.2, 95% CI 2.4-22; p≤ 0.001), bronchoalveolar lavage (BAL) log2 interleukin (IL)-8 (OR 1.2, 95% CI 1.05-1.5; p=0.010) and body mass index z-score (OR 0.49, 95% CI 0.24-1.00; p=0.05) at age 5 years. Percentage trapped air at follow-up was associated with BAL log2 IL-8 (coefficient 1.3, 95% CI 0.57-2.1; p<0.001) at age 5 years.The extent of airway disease, atelectasis, airway inflammation and poor nutritional status in early childhood are risk factors for progressive structural lung disease in adolescence.
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Fibrosis Quística , Adolescente , Australia , Niño , Preescolar , Progresión de la Enfermedad , Estudios de Seguimiento , Humanos , Pulmón/diagnóstico por imagen , Nueva ZelandaRESUMEN
AIM: The overall aim was to investigate the feasibility and utility of linking a cerebral palsy (CP) register to an administrative data set for health services research purposes. We sought to compare CP hospital admissions to general childhood population admissions, and identify factors associated with type and frequency of admissions in a CP cohort. METHOD: The CP register for Victoria, Australia was linked to the state's hospital admissions database. Data pertaining to the admissions of a CP cohort (n=1748) that took place between 2007 and 2014 were extracted. Population data were also obtained. RESULTS: Overall, 80% of the CP cohort (n=1401) had at least admission between 2007 and 2014, accounting for 11 012 admissions or 1.5% of all admissions in their age group. Compared to general population admissions, CP admissions were more costly and more likely to be elective (66% vs 57%; p<0.001), medical (71% vs 57%; p<0.001), and to take place in metropolitan hospitals (92% vs 78%; p<0.001). Increased CP severity and complexity were associated with having more admissions and a higher proportion of admissions attributable to respiratory illness. INTERPRETATION: By linking with administrative data sets, CP registers may be useful for health services research and inform health service delivery.
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Parálisis Cerebral/epidemiología , Parálisis Cerebral/terapia , Administración Hospitalaria/estadística & datos numéricos , Adolescente , Distribución por Edad , Australia , Parálisis Cerebral/complicaciones , Niño , Preescolar , Estudios de Cohortes , Enfermedades del Sistema Digestivo/etiología , Femenino , Humanos , Lactante , Recién Nacido , Masculino , Enfermedades Musculoesqueléticas/etiología , Enfermedades del Sistema Nervioso/etiología , Sistema de Registros , Trastornos Respiratorios/etiologíaRESUMEN
AIM: The aims of this study were to estimate the proportion of emergency department presentations attributable to children with cerebral palsy (CP), investigate the frequency of emergency department presentations in a CP cohort, and compare emergency department presentations among children with CP with those of other children. METHOD: This was a retrospective cohort study. The Victorian Cerebral Palsy Register was linked to the Victorian Emergency Minimum Dataset. Data on emergency department presentations for the CP cohort occurring between 2007 and 2014 and population control data were obtained. RESULTS: The CP cohort (n=1748) had 7015 emergency department presentations during the 7-year period, accounting for 0.4% of the 1.69 million age-specific presentations during that time. The number of annual presentations per 1000 children rose with increasing CP severity. Compared with presentations among the general population, higher proportions of presentations among the CP cohort were preceded by ambulance arrivals (27% vs 8%), triaged as urgent (66% vs 32%), and required hospital admission (38% vs 12%). INTERPRETATION: The marked differences in presentations between the CP cohort and the general population in the proportions that were urgent and required ambulance arrivals and hospital admissions was an important finding. Strategies to ensure appropriate use of services, including encouragement to seek earlier assistance from primary care providers, may prevent problems escalating to the need for urgent care. WHAT THIS PAPER ADDS: Children with cerebral palsy (CP) account for 0.4% of childhood emergency department presentations. More emergency department presentations among children with CP require ambulance arrival. More CP emergency department presentations are urgent and require hospital admission. Traditional emergency department triage scales seem less accurate for this group.
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Parálisis Cerebral/epidemiología , Parálisis Cerebral/terapia , Servicio de Urgencia en Hospital/estadística & datos numéricos , Hospitales Pediátricos/estadística & datos numéricos , Adolescente , Parálisis Cerebral/complicaciones , Niño , Preescolar , Estudios de Cohortes , Enfermedades del Sistema Digestivo/etiología , Femenino , Humanos , Masculino , Enfermedades Musculoesqueléticas/etiología , Enfermedades del Sistema Nervioso/etiología , Nueva Zelanda/epidemiología , Sistema de Registros , Trastornos Respiratorios/etiologíaRESUMEN
AIM: To evaluate the reliability, validity and responsiveness of the High-level Mobility Assessment Tool (HiMAT) in children and adolescents with traumatic brain injury (TBI) and to compare the mobility skills of children with TBI to those of healthy peers. METHOD: The mobility skills of 52 children with moderate and severe TBI (36 males; mean age = 12 years, range = 6-17) were assessed using the HiMAT and the Pediatric Evaluation of Disability Inventory (PEDI). Inter-rater reliability, re-test reliability and responsiveness of the HiMAT were evaluated in sub-groups by comparing results scored at several time-points. The HiMAT scores of children with TBI were compared with those of a healthy comparative cohort. RESULTS: The HiMAT demonstrated excellent inter-rater reliability (ICC = 0.93), re-test reliability (ICC = 0.98) and responsiveness to change (p = 0.002). The PEDI demonstrated a ceiling effect in mobility assessment of ambulant children with TBI. The HiMAT scores of children with TBI were lower than those of their healthy peers (p < 0.001). INTERPRETATION: The HiMAT is a reliable, valid and sensitive measure of high-level mobility skills following childhood TBI. The high-level mobility skills of children with TBI are less proficient than their peers.
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Lesiones Encefálicas/diagnóstico , Lesiones Encefálicas/rehabilitación , Evaluación de la Discapacidad , Limitación de la Movilidad , Adolescente , Lesiones Encefálicas/fisiopatología , Estudios de Casos y Controles , Niño , Femenino , Humanos , Masculino , Recuperación de la Función/fisiología , Reproducibilidad de los ResultadosRESUMEN
BACKGROUND: In 1964, The Melbourne Asthma Study was established to describe the spectrum and natural history of childhood asthma. OBJECTIVE: To describe the clinical and lung function outcome of childhood asthma to the age of 50 years. METHOD: Subjects were invited to complete an interviewer-administered questionnaire, skin prick testing, and measurement of lung function from the age of 7 years to the age of 50 years at 7-year intervals. RESULTS: Of 458 survivors (from the original 484 subjects at recruitment), 346 subjects (76%) participated, of whom, 197 completed lung function measurement. Asthma remission at the age of 50 years was 64% in those with wheezy bronchitis, 47% for those with persistent asthma, and 15% for those with severe asthma in childhood. Multivariable analysis identified severe asthma in childhood (odds ratio [OR] 11.9 [95% CI, 3.4-41.8]), female sex (OR 2.0 [95% CI, 1.1-3.6]), and childhood hay fever (OR 2.0 [95% CI, 1.0-4.0]) as risk factors for "current asthma" at age 50 years. There was no evidence of a difference in the rate of decline in FEV1 (mL/y, 95% CI) between the severe asthma group (15 mL/y [95% CI, 9-22 mL/y]) and all the other recruitment groups: control (16 mL/y [95% CI, 12-20 mL/y]), mild wheezy bronchitis (14 mL/y [95% CI, 8-19 mL/y]), wheezy bronchitis (16 mL/y [95% CI, 11-20 mL/y]), and persistent asthma (19 mL/y [95% CI, 13-24 mL/y]). CONCLUSION: The clinical and lung function outcome in adult life is strongly determined by asthma severity in childhood. The reduced lung function seen in adults is established in childhood and does not appear to decline more rapidly in adult years despite continuing symptoms.
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Asma/epidemiología , Adolescente , Adulto , Asma/fisiopatología , Niño , Femenino , Humanos , Estudios Longitudinales , Masculino , Persona de Mediana Edad , Evaluación de Resultado en la Atención de Salud , Pruebas de Función Respiratoria , Pruebas Cutáneas , Fumar , Encuestas y Cuestionarios , Adulto JovenRESUMEN
OBJECTIVES: To determine whether bronchoalveolar lavage (BAL)-directed therapy for infants and young children with cystic fibrosis (CF), rather than standard therapy, was justified on the grounds of a decrease in average costs and whether the use of BAL reduced treatment costs associated with hospital admissions. STUDY DESIGN: Costs were assessed in a randomized controlled trial conducted in Australia and New Zealand on infants diagnosed with CF after newborn screening and assigned to receive either BAL-directed or standard therapy until they reached 5 years of age. A health care funder perspective was adopted. Resource use measurement was based on standardized data collection forms administered for patients across all sites. Unit costs were obtained primarily from government schedules. RESULTS: Mean costs per child during the study period were Australian dollars (AUD)92â860 in BAL-directed therapy group and AUD90â958 in standard therapy group (mean difference AUD1902, 95% CI AUD-27â782 to 31â586, P = .90). Mean hospital costs per child during the study period were AUD57â302 in the BAL-directed therapy group and AUD66â590 in the standard therapy group (mean difference AUD-9288; 95% CI AUD-35â252 to 16â676, P = .48). CONCLUSIONS: BAL-directed therapy did not result in either lower mean hospital admission costs or mean costs overall compared with managing patients with CF by a standard protocol based upon clinical features and oropharyngeal culture results alone. Following on our previous findings that BAL-directed treatment offers no clinical advantage over standard therapy at age 5 years, flexible bronchoscopy with BAL cannot be recommended for the routine management of preschool children with CF on the basis of overall cost savings.
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Lavado Broncoalveolar/economía , Fibrosis Quística/economía , Fibrosis Quística/terapia , Preescolar , Costos y Análisis de Costo , Humanos , Lactante , Admisión del Paciente/economía , Admisión del Paciente/estadística & datos numéricosRESUMEN
BACKGROUND: Long-term effects of early, recurrent human exposure to general anaesthesia remain unknown. The Australasian Cystic Fibrosis Bronchoalveolar Lavage (ACFBAL) trial provided an opportunity to examine this issue in children randomly assigned in infancy to either repeated bronchoalveolar-lavage (BAL)-directed therapy with general anaesthesia or standard care with no planned lavages up to 5 years of age when all children received BAL-directed therapy under general anaesthesia. METHODS: This multicentre, randomised, open-label phase 4 trial (CF-GAIN) used the original ACFBAL trial randomisation at 3·6 months (SD 1·6) to BAL-directed therapy or standard-care groups to assess the impact of general anaesthesia exposures over early childhood. Children who completed the ACFBAL trial, with a mean age of 5·1 (SD 0·18) years, received standardised neurobehavioural and health-related-quality-of-life assessment and brain MRI scans between Oct 8, 2013, and June 30, 2017, at a mean age of 12·8 (SD 1·7) years at three hospitals in Australia and one hospital in New Zealand. The primary outcome was a composite score of performance on a standardised, computer-based assessment of child attention, processing speed, and response inhibition skills (Conners Continuous Performance test, second edition). Secondary outcomes included intellectual function, other neurobehavioural measures, and brain imaging as an exploratory outcome. The trial was registered with the Australian New Zealand Clinical Trials Registry (ACTRN 12613000057785) and is completed. FINDINGS: At 2 years, the BAL-directed therapy group (n=52) and standard-care group (n=45) had a median of 2·0 (IQR 1·0-3·0) and 0·0 (0·0-0·0) exposures, respectively. At completion of the ACFBAL trial, the BAL-directed therapy group had a median of 6·0 (4·0-9·5) exposures and the standard-care group 2·0 (1·0-4·0) exposures. At CF-GAIN completion, the BAL-directed therapy group had a median of 10·0 (IQR 6·5-14·5) exposures and the standard-care group 4·0 (3·0-7·0) exposures. Cumulative general anaesthesia exposure time was not prospectively collected but, for those with complete cumulative exposure time data to the end of the ACFBAL trial, the median cumulative exposure time for the BAL-directed therapy group (n=29) was 180 (IQR 140-285) min and for the standard-care group (n=32) was 48 (30-122) min. The mean Conners Continuous Performance test, second edition composite score was 51 (SD 8·1) in BAL-directed therapy group and 53 (8·8) in the standard-care group; difference -1·7 (95% CI -5·2 to 1·7; p=0·32) with similar performance on other neurobehavioural measures, including measures of executive function, intellectual quotient scores, and brain imaging. INTERPRETATION: Our findings suggest that repeated general anaesthesia exposure in young children with cystic fibrosis is not related to functional impairment in attention, intellectual quotient, executive function, or brain structure compared with a group with fewer and shorter cumulative anaesthesia durations. FUNDING: National Health and Medical Research Council Australia, Queensland Government Health Service and Clinical Innovation Fellowship, and the Children's Hospital Foundation Queensland.
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BACKGROUND: Newborn screening allows novel treatments for cystic fibrosis (CF) to be trialled in early childhood before irreversible lung injury occurs. As respiratory exacerbations are a potential trial outcome variable, we determined their rate, duration and clinical features in preschool children with CF; and whether they were associated with growth, lung structure and function at age 5 years. METHODS: Respiratory exacerbations were recorded prospectively in Australasian CF Bronchoalveolar Lavage trial subjects from enrolment after newborn screening to age 5 years, when all participants underwent clinical assessment, chest CT scans and spirometry. RESULTS: 168 children (88 boys) experienced 2080 exacerbations, at an average rate of 3.66 exacerbations per person-year; 80.1% were community managed and 19.9% required hospital admission. There was an average increase in exacerbation rate of 9% (95% CI 4% to 14%; p<0.001) per year of age. Exacerbation rate differed by site (p<0.001) and was 26% lower (95% CI 12% to 38%) in children receiving 12 months of prophylactic antibiotics. The rate of exacerbations in the first 2 years was associated with reduced forced expiratory volume in 1 s z scores. Ever having a hospital-managed exacerbation was associated with bronchiectasis (OR 2.67, 95% CI 1.13 to 6.31) in chest CT scans, and lower weight z scores at 5 years of age (coefficient -0.39, 95% CI -0.74 to -0.05). CONCLUSIONS: Respiratory exacerbations in young children are markers for progressive CF lung disease and are potential trial outcome measures for novel treatments in this age group.
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Antibacterianos/uso terapéutico , Profilaxis Antibiótica/métodos , Lavado Broncoalveolar/métodos , Fibrosis Quística/complicaciones , Hospitalización/tendencias , Enfermedades Pulmonares/epidemiología , Pulmón/fisiopatología , Preescolar , Fibrosis Quística/diagnóstico , Fibrosis Quística/terapia , Femenino , Estudios de Seguimiento , Volumen Espiratorio Forzado , Humanos , Incidencia , Lactante , Recién Nacido , Pulmón/diagnóstico por imagen , Enfermedades Pulmonares/etiología , Enfermedades Pulmonares/prevención & control , Masculino , Nueva Zelanda/epidemiología , Pronóstico , Estudios Prospectivos , Radiografía Torácica , Espirometría , Tomografía Computarizada por Rayos XRESUMEN
BACKGROUND: There is no data exclusively on the relationship between health-related quality-of-life (HRQOL) and lung disease severity in early school-aged children with cystic fibrosis (CF). Using data from the Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF) we assessed the relationships between HRQOL, lung function and structure. METHODS: 125 children aged 6.5-10 years enrolled in the AREST CF program were included from CF clinics at Royal Children's Hospital (RCH), Melbourne (n = 66) and Perth Children's Hospital (PCH), Perth (n = 59), Australia. Demographics, HRQOL measured by Cystic Fibrosis Questionnaire-Revised (CFQ-R), spirometry, multiple-breath washout (MBW) and chest CT were collected across two years. Correlation between CFQ-R scores and lung structure/function parameters and agreement between parent-proxy and child-reported HRQOL were evaluated. RESULTS: No correlation was observed between most CFQ-R domain scores and FEV1 z-scores, excepting weak-positive correlation with parent CFQ-R Physical (rho = 0.21, CI 0.02-0.37), and Weight (rho = 0.21, CI 0.03-0.38) domain and child Body domain (rho = 0.26, CI 0.00-0.48). No correlation between most CFQ-R domain scores and LCI values was noted excepting weak-negative correlation with parent Respiratory (rho = -0.23, CI -0.41--0.05), Emotional (rho = -0.24, CI -0.43--0.04), and Physical (-0.21, CI -0.39--0.02) domains. Furthermore, structural lung disease on CT data demonstrated little to no association with CFQ-R parent and child domain scores. Additionally, no agreement between child self-report and parent-proxy CFQ-R scores was observed across the majority of domains and visits. CONCLUSION: HRQOL correlated poorly with lung function and structure in early school-aged children with CF, hence clinical trials should consider these outcomes independently when determining study end-points.
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Fibrosis Quística , Calidad de Vida , Australia/epidemiología , Niño , Estado de Salud , Humanos , Pulmón/diagnóstico por imagen , Índice de Severidad de la EnfermedadRESUMEN
CONTEXT: Early pulmonary infection in children with cystic fibrosis leads to increased morbidity and mortality. Despite wide use of oropharyngeal cultures to identify pulmonary infection, concerns remain over their diagnostic accuracy. While bronchoalveolar lavage (BAL) is an alternative diagnostic tool, evidence for its clinical benefit is lacking. OBJECTIVE: To determine if BAL-directed therapy for pulmonary exacerbations during the first 5 years of life provides better outcomes than current standard practice relying on clinical features and oropharyngeal cultures. DESIGN, SETTING, AND PARTICIPANTS: The Australasian Cystic Fibrosis Bronchoalveolar Lavage (ACFBAL) randomized controlled trial, recruiting infants diagnosed with cystic fibrosis through newborn screening programs in 8 Australasian cystic fibrosis centers. Recruitment occurred between June 1, 1999, and April 30, 2005, with the study ending on December 31, 2009. INTERVENTIONS: BAL-directed (n = 84) or standard (n = 86) therapy until age 5 years. The BAL-directed therapy group underwent BAL before age 6 months when well, when hospitalized for pulmonary exacerbations, if Pseudomonas aeruginosa was detected in oropharyngeal specimens, and after P. aeruginosa eradication therapy. Treatment was prescribed according to BAL or oropharyngeal culture results. MAIN OUTCOME MEASURES: Primary outcomes at age 5 years were prevalence of P. aeruginosa on BAL cultures and total cystic fibrosis computed tomography (CF-CT) score (as a percentage of the maximum score) on high-resolution chest CT scan. RESULTS: Of 267 infants diagnosed with cystic fibrosis following newborn screening, 170 were enrolled and randomized, and 157 completed the study. At age 5 years, 8 of 79 children (10%) in the BAL-directed therapy group and 9 of 76 (12%) in the standard therapy group had P. aeruginosa in final BAL cultures (risk difference, -1.7% [95% confidence interval, -11.6% to 8.1%]; P = .73). Mean total CF-CT scores for the BAL-directed therapy and standard therapy groups were 3.0% and 2.8%, respectively (mean difference, 0.19% [95% confidence interval, -0.94% to 1.33%]; P = .74). CONCLUSION: Among infants diagnosed with cystic fibrosis, BAL-directed therapy did not result in a lower prevalence of P. aeruginosa infection or lower total CF-CT score when compared with standard therapy at age 5 years. TRIAL REGISTRATION: anzctr.org.au Identifier: ACTRN12605000665639.
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Antibacterianos/uso terapéutico , Fibrosis Quística/complicaciones , Fibrosis Quística/patología , Lesión Pulmonar/etiología , Infecciones por Pseudomonas/diagnóstico , Infecciones por Pseudomonas/tratamiento farmacológico , Australia/epidemiología , Lavado Broncoalveolar , Preescolar , Fibrosis Quística/diagnóstico por imagen , Hospitalización , Humanos , Lactante , Recién Nacido , Pulmón/fisiopatología , Lesión Pulmonar/prevención & control , Nueva Zelanda/epidemiología , Prevalencia , Infecciones por Pseudomonas/epidemiología , Pruebas de Función Respiratoria , Tomografía Computarizada por Rayos XRESUMEN
Objectives: To describe characteristics and outcomes of children requiring intensive care therapy (ICT) within 12 hours following a medical emergency team (MET) event. Design: Retrospective cohort study. Setting: Quaternary paediatric hospital. Patients: Children experiencing a MET event. Measurements and main results: Between July 2017 and March 2019, 890 MET events occurred in 566 patients over 631 admissions. Admission to intensive care followed 183/890 (21%) MET events. 76/183 (42%) patients required ICT, defined as positive pressure ventilation or vasoactive support in intensive care, within 12 hours. Older children had a lower risk of requiring ICT than infants aged < 1 year (age 1-5 years [risk difference, -6.4%; 95% CI, -11% to -1.6%; P = 0.01] v age > 5 years [risk difference, -8.0%; 95% CI, -12% to -3.8%; P < 0.001]), while experiencing a critical event increased this risk (risk difference, 16%; 95% CI, 3.3-29%; P = 0.01). The duration of respiratory support and intensive care length of stay was approximately double in patients requiring ICT (ratio of geometric means, 2.0 [95% CI, 1.4-3.0] v 2.1 [95% CI, 1.5-2.8]; P < 0.001) and the intensive care mortality increased (risk difference, 9.6%; 95% CI, 2.4-17%; P = 0.01). Heart rate, oxygen saturation and respiratory rate were the most commonly measured vital signs in the 6 hours before the MET event. Conclusions: Approximately one-fifth of MET events resulted in intensive care admission and nearly half of these required ICT within 12 hours. This group had greater duration of respiratory support, intensive care and hospital length of stay, and higher mortality. Age < 1 year and a critical event increased the risk of ICT.
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BACKGROUND: For Australians living with cystic fibrosis (CF), increased longevity means greater consideration needs to be given to long-term endocrine sequelae such as CF-related bone disease. Deficits in bone mass accrual are most likely to occur during childhood and adolescence. Current guidelines in Australia suggest repeat dual-energy X-ray absorptiometry (DXA) scans every 2 years. This study aims to stratify clinical factors that determine future bone health in the Australian CF population and use this to guide a more streamlined approach to bone health screening. METHODS: This study was a retrospective audit of all patients diagnosed with CF who were treated at the Royal Children's Hospital Melbourne, Australia from 2000 to 2016 (n = 453). Two hundred and two patients had a DXA scan in the study period (191 with height-adjusted data) and 111 patients had more than one scan (108 with height-adjusted data). An investigation into the associations between bone mineral density (BMD) Z score and potential risk factors was conducted using DXA and historical data. RESULTS: The main predictor of future BMD was the previous BMD Z score (p < .001). Other factors found to be determinants of BMD included nutritional status, lung function (FEV1 ), age, history of previous fracture, oral corticosteroid use, and the number of hospital admissions. However, after adjusting for previous BMD, evidence of an association remained only with nutritional status, FEV1 , and number of hospital admissions. CONCLUSION: Second yearly scans may be unnecessary in children with an adequate DXA score on the initial scan who remain clinically stable. However, clinical deterioration in those whose BMD was previously normal, may require closer monitoring of bone health. We propose a guideline for the frequency of DXA monitoring in relation to clinical risk factors.
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Densidad Ósea , Fibrosis Quística , Absorciometría de Fotón , Adolescente , Australia/epidemiología , Niño , Fibrosis Quística/complicaciones , Fibrosis Quística/diagnóstico por imagen , Fibrosis Quística/epidemiología , Humanos , Estudios RetrospectivosRESUMEN
BACKGROUND: Exercise echocardiography reveals abnormalities in asymptomatic childhood cancer survivors who previously have been treated with anthracyclines. We determined the added value of monitoring childhood cancer survivors with exercise echocardiography compared to monitoring with resting echocardiography alone to predict anthracycline-induced cardiotoxicity. Secondary aims were to evaluate change in resting cardiac function over 10 years and to determine risk factors for late cardiotoxicity. PROCEDURE: We invited a cohort of 110 originally asymptomatic anthracycline-treated childhood cancer survivors, who had undergone cardiac tests including exercise echocardiography 10.5 years earlier, for new cardiac evaluation. Each subject underwent a resting echocardiogram at both evaluations. At first evaluation a repeat echocardiogram was performed following peak exercise. Resting echocardiographic parameters were converted to z-character. RESULTS: Ninety-two of 110 survivors (mean anthracycline dose 307 mg/m(2), mean follow-up time from start of treatment 8.2 years at first and 18.8 years at second evaluation) were evaluated prospectively. Mean resting fractional shortening z-character (RFSz) decreased from -0.18 to -0.93. Higher cumulative anthracycline dose was a risk factor for a lower RFSz at late follow-up (P = 0.0002). Adding exercise fractional shortening (XFS) to a model containing RFSz did not improve prediction of abnormal RFSz at late follow-up. CONCLUSIONS: Monitoring with exercise echocardiography has no added value to monitoring with resting echocardiography alone in predicting late anthracycline-induced cardiotoxicity in childhood cancer survivors. RFSz deteriorates over time, even in originally asymptomatic patients. Previous treatment with higher cumulative anthracycline dose is the main risk factor for a lower RFSz at late follow-up.
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Antraciclinas/efectos adversos , Antineoplásicos/efectos adversos , Ecocardiografía/métodos , Cardiopatías/diagnóstico , Neoplasias/tratamiento farmacológico , Adolescente , Niño , Preescolar , Ejercicio Físico , Femenino , Estudios de Seguimiento , Cardiopatías/inducido químicamente , Humanos , Lactante , Masculino , Curva ROC , SobrevivientesRESUMEN
BACKGROUND: Serum Glycoprotein A (GlycA) levels are increased in a variety of inflammatory disease states. However, GlycA has not been previously evaluated in children with cystic fibrosis (CF). We assessed the relationship between GlycA and pulmonary infection, inflammation, bronchial wall thickening (BWT) and bronchiectasis in young children with CF. METHODS: From 95 patients, we obtained 311 paired serum and bronchoalveolar lavage (BAL) samples at multiple timepoints, with concurrent chest computed tomography on 168 occasions. Quantitative GlycA was determined using high-throughput nuclear magnetic resonance metabolomic testing. Participants were considered to be infected if ≥1 significant proinflammatory organism was isolated from their BAL. The presence of free neutrophil elastase (NE) above the limit of detection was considered evidence of inflammation. The relationships between GlycA levels and infection state, inflammation, and bronchiectasis were examined using a generalized estimating equation approach. RESULTS: There was a positive relationship between GlycA (mean 1.01 mmol/L, range 0.68-1.92 mmol/L) and being infected with one or more proinflammatory organisms, even after adjusting for age and gender (odds ratio [OR], 1.2 per 0.1 mmol/L, 95% confidence interval [CI], 1.02, 1.4, P = .03). There was also a positive relationship between GlycA and NE (unadjusted OR, 1.2 95% CI, 1.01, 1.4, P = .04), not significant after adjustment. GlycA concentration was associated with BWT but not bronchiectasis. CONCLUSIONS: Although GlycA levels were higher on average in those who had an infection or neutrophilic inflammation, there was also considerable variability, limiting the clinical utility of this biomarker alone in determining early disease status in CF.
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Fibrosis Quística/sangre , Glicoproteínas/sangre , Biomarcadores/sangre , Bronquiectasia/sangre , Lavado Broncoalveolar , Preescolar , Femenino , Humanos , Lactante , Inflamación/sangre , Elastasa de Leucocito/sangre , Masculino , Infecciones del Sistema Respiratorio/complicacionesRESUMEN
BACKGROUND: The impact of early cystic fibrosis (CF) on health-related quality-of-life (HRQOL) in preschool children is poorly characterised, and data on relationships between HRQOL and health outcomes in young children with CF are limited. We aimed to characterise and compare parent-proxy and child-reported HRQOL and evaluate relationships with clinical outcomes at age 5-years. METHODS: Subjects were participating in the multi-centre Australasian Cystic Fibrosis Bronchoalveolar Lavage (ACFBAL) trial investigating BAL-directed versus standard CF therapy. Children aged 5-years and their parents rated HRQOL using the Pediatric Quality of Life Inventory (PedsQL™) and Cystic Fibrosis Questionnaire-Revised (CFQ-R) questionnaires. RESULTS: PedsQL and CFQ-R questionnaires were completed by 141 primary caregivers and 135 and 130 children, respectively. There were no differences in HRQOL between children randomised to BAL-directed versus standard CF therapy. Children with CF rated worse HRQOL than healthy children and there was poor parent-child concordance across HRQOL domains. Nutritional status, CF-CT scan score, forced expiratory volume in 1-second (FEV1), and pulmonary exacerbations correlated with HRQOL at age 5-years. FEV1 z-scores positively correlated with parent-proxy HRQOL in CFQ-R Respiratory (p = 0.018), Physical (<0.001), Emotional (p = 0.007) subscales and PedsQL Total-score (p = 0.021), Physical (p = 0.019) domains. Pulmonary exacerbation rates were inversely associated with parent-proxy CFQ-R Respiratory (p = 0.004), Physical (p = 0.022), PedsQL Total (p = 0.009) and Physical (p = 0.009) scores. CONCLUSION: Parent-reported HRQOL is a meaningful clinical endpoint to evaluate interventions in young children. Parent and child HRQOL reports provide different, complementary information. A preschool version of the CFQ-R is needed to assess relationships between HRQOL and clinical outcomes in young children.
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Fibrosis Quística/psicología , Manejo de la Enfermedad , Estado de Salud , Padres/psicología , Calidad de Vida , Preescolar , Fibrosis Quística/fisiopatología , Fibrosis Quística/terapia , Femenino , Volumen Espiratorio Forzado , Humanos , Masculino , Estado Nutricional , Psicología Infantil/métodos , Pruebas de Función Respiratoria/estadística & datos numéricos , Índice de Severidad de la Enfermedad , Encuestas y Cuestionarios , Resultado del TratamientoRESUMEN
BACKGROUND: Both infection and inflammation are critical to the progression of cystic fibrosis (CF) lung disease. Potential anatomical differences in lower airway infection, inflammation and bronchiectasis in young children with CF raise questions regarding the pathogenesis of early structural lung disease. METHODS: A longitudinal multi-centre birth cohort study of infants newly diagnosed with CF was conducted. Paired bronchoalveolar lavage (BAL) samples were obtained from the right middle lobe (RML) and lingula bronchi. Chest computed tomography (CT) was performed biennially and analysed using the modified CF-CT scoring system. RESULTS: One hundred and twenty-four children (0.11 - 7.0 years) contributed 527 BAL samples and underwent 388 CT chest scans. Pro-inflammatory microbes were detected in 279 BAL samples (53%), either in both lingula and RML samples (69%), in the lingula alone (24%), or in the RML alone in only 7% of samples. Overall, the prevalence of structural lung disease was greater in the setting of pro-inflammatory microbes. Although infection was less commonly isolated in the right lung, bronchiectasis was more commonly detected in the right lung compared with the left. No anatomical differences in the presence of air trapping were detected. CONCLUSION: Overall, the detection of pro-inflammatory microbes in the lower airways was associated with increased risk of both air trapping and bronchiectasis. However, the apparent discordance between commonest sites of isolation of pro-inflammatory microbes and the anatomical site of early bronchiectasis warrants further exploration.
Asunto(s)
Bronquiectasia/microbiología , Fibrosis Quística/fisiopatología , Infecciones del Sistema Respiratorio/complicaciones , Lavado Broncoalveolar , Niño , Preescolar , Progresión de la Enfermedad , Femenino , Humanos , Lactante , Recién Nacido , Inflamación/microbiología , Estudios Longitudinales , Masculino , Tomografía Computarizada por Rayos XRESUMEN
OBJECTIVES: To determine the association between residence and climate with risk of Pseudomonas aeruginosa (Pa) and other respiratory outcomes. METHODS: We performed regular bronchoalveolar lavage and upper airway cultures in young children with CF to identify Pa infection. Children were classified for residence as regional or metropolitan. Bronchiectasis was detected on periodic chest computed tomography scans. Multilocus sequence typing determined Pa genotype. Lung function was assessed using Multiple Breath Washout. RESULTS: Of infants diagnosed with CF between 2006 and 2017, 129 were included in the study. Seven patients moved between metropolitan and regional Victoria and were excluded from analysis. Of the remaining 122 subjects, seventy-four (61%) children resided in metropolitan areas and over half (54%) were male. There were 83â¯Pa episodes in the 122 children who lived consistently in a geographical location. The incidence rate was 0.15 episodes per person-years. We found weak evidence of a 15% increase in the rate of Pa episodes with increasing average annual maximum temperature (95%CI (0.98, 1.36); pâ¯=â¯.086), while the rate of Pa acquision decreased with average annual 3â¯pm humidity (IRRâ¯=â¯0.96; 95%CI(0.92, 1.0008); pâ¯=â¯.054). The rate of Pa episodes was 2.1 times higher in regional participants (95%CI (1.4, 3.1); pâ¯=â¯.001) and risk of second episode was more than five times greater (HR 5.7; 95%CI 1.9, 17); pâ¯=â¯.002). No difference between regions in lung clearance index and presence of bronchiectasis was detected. CONCLUSION: Regional residence is associated with risk of acquiring recurrent infection with Pseudomonas aeruginosa in young children with CF.
Asunto(s)
Clima , Fibrosis Quística , Infecciones por Pseudomonas , Pseudomonas aeruginosa/aislamiento & purificación , Características de la Residencia/estadística & datos numéricos , Medición de Riesgo/métodos , Antibacterianos/uso terapéutico , Australia/epidemiología , Bronquiectasia/diagnóstico , Bronquiectasia/etiología , Lavado Broncoalveolar/métodos , Preescolar , Fibrosis Quística/epidemiología , Fibrosis Quística/fisiopatología , Fibrosis Quística/terapia , Femenino , Humanos , Lactante , Pulmón/diagnóstico por imagen , Pulmón/microbiología , Masculino , Infecciones por Pseudomonas/epidemiología , Infecciones por Pseudomonas/fisiopatología , Infecciones por Pseudomonas/terapia , Recurrencia , Pruebas de Función Respiratoria/métodos , Factores de Riesgo , Tomografía Computarizada por Rayos X/métodosRESUMEN
BACKGROUND AND AIM: Polyethylene glycol (PEG) is the gold standard for fecal disimpaction in constipation. A regimen of PEG combined with the stimulant laxative sodium picosulphate (SPS) produced fecal disimpaction in chronically constipated children in the community, but it is unknown if it is effective for more severe constipation. To determine the stool output and effect of a combined PEG and SPS regimen on fecaloma in children with severe constipation and impaction. METHODS: Children with symptoms for a duration of ≥2 years, a palpable fecaloma, and enlarged rectum on X-ray (rectal: pelvic ratio > 0.6) were recruited from a tertiary hospital. Daily diaries recorded laxative dose, stool frequency, volume, and consistency (Bristol stool scale, BSS). Abdominal X-rays were taken on day 1 and day 8, and stool loading was assessed using the Leech score. Laxative doses were based on the child's age. The dose of PEG with electrolytes taken was 2-8 sachets (14.7 g/sachet) on days 1-2, reducing to 2-6 sachets on day 3. The SPS dose was 15-20 drops on days 2-3. RESULTS: Eighty-nine children (4-18 years) produced a large volume of soft stool (median/inter-quartile-range: 2.2/1.6-3.1 L) over 7 days. Stool volume on X-rays decreased significantly in the colon (P < 0.001). Fecalomas resolved in 40 of 89 children, while 49 needed a second high dose. Rectal:pelvic ratios did not change. CONCLUSIONS: A combined high dose of PEG and SPS on days 1 and 2 was effective in removing the fecaloma in half of the children. Administering high doses for a longer period should be tested to provide outpatient disimpaction for severe fecalomas. Rectums remained flaccid after emptying.
RESUMEN
BACKGROUND/AIM: International criteria currently suggest orchidopexy at 6-12months for congenital undescended testis (UDT). Some children require repeat orchidopexy for recurrent UDT. This study aimed to assess practice in Australia over a 20-year period. METHODS: We examined 20years of Australian orchidopexy data (1995-2014) from the Department of Human Services to explore the national revision orchidopexy rates over time. RESULTS: The total number of orchidopexy revisions was 890 over 20years compared with 25,984 primary operations. More than 50% of all primary and revision orchidopexies in 0-14year-old boys were performed in major population centers of NSW and Victoria (which hold 52% male population of same age), with a small number of revisions on 15-24year-old males. The incidence of revision orchidopexy significantly decreased over the 20-year period in boys ages 0-14years old, from 276 operations between 1995 and 1999 decreasing to 165 operations between 2010 and 2014 (-53%), compared to a population increase of +15% (p<0.05). CONCLUSION: These data demonstrate a decrease in revision orchidopexy since 1995, which may be related to change in referral practice with more children undergoing orchidopexy (primary and revision) by pediatric surgeons over the 20-year period. LEVEL OF EVIDENCE: Level IV. TYPE OF STUDY: Therapeutic Case Series with no Comparison Group.