Prioritizing family-centered developmental care: insights from parents of children with critical congenital heart disease: a qualitative study.

As survivors of early cardiac surgery are at high risk of neurodevelopmental impairments, systematic health observations of children with critical congenital heart disease (CCHD) throughout childhood are recommended to enable early diagnosis and offer interventions to optimize neurodevelopment. A qualitative study using thematic analysis was performed to explore parents' concerns, experiences, and needs regarding the development and received developmental care of their child (0-10 years) during hospital admission and beyond. Data were collected using semi-structured online interviews with 20 parents of children with CCHD. Four major themes were identified: (1) "impact of diagnosis and disease on the family-system," (2) "parental concerns from diagnoses and beyond," (3) "the need for information," and (4) "the need for individualized and family-centered care." The main themes can be divided into 13 sub-themes as impact, concerns, and needs are influenced by various impactful moments from diagnosis and afterwards.     Conclusion: This study confirms the importance of early identification of neurodevelopmental problems by experienced healthcare professionals, especially in the early years when parental expectations and concerns about their child's neurodevelopment are lower. A tailor-made family-centered follow-up program should be offered, which pays attention to both the neurodevelopment of patients with CCHD as well as the mental wellbeing of the entire family system. Furthermore, an online portal is recommended with a variety of reliable, controlled, understandable information from which parents can obtain the desired information to understand better the consequences of specific heart condition and to provide their child with the best possible guidance. What is Known: • Survivors of early cardiac surgery are at high risk of neurodevelopmental impairments; systematic health observations of children with CCHD throughout childhood are strongly recommended. What is New: • Parents need a tailor-made family-centered follow-up program, which pays attention to both the neurodevelopment of patients with CCHD as well as the mental wellbeing of the entire family system. • An online portal offering diverse, trustworthy information and sources would effectively meet parents' needs by providing accessible insights into the potential consequences of specific heart conditions and guiding them in supporting their child optimally.

Methotrexate for juvenile idiopathic arthritis.

BACKGROUND: Juvenile idiopathic arthritis (JIA) is the most common rheumatic disease in childhood. Methotrexate has broad immunomodulatory properties and is the most commonly used disease-modifying antirheumatic drug (DMARD). This is an update of a 2001 Cochrane review. It supports a living guideline for children and young people with JIA. OBJECTIVES: To assess the benefits and harms of methotrexate for children and young people with juvenile idiopathic arthritis. SEARCH METHODS: The Australian JIA Living Guideline Working Group created a registry of all randomised controlled trials (RCTs) of JIA by searching CENTRAL, MEDLINE, Embase, and trials registries. The date of the most recent search of online databases was 1 February 2023. SELECTION CRITERIA: We searched for RCTs that compared methotrexate with placebo, no treatment, or another DMARD (with or without concomitant therapies) in children and young people (aged up to 18 years) with JIA. DATA COLLECTION AND ANALYSIS: We used standard Cochrane methods. The main comparison was methotrexate versus placebo. Our outcomes were treatment response, sustained clinically inactive disease, function, pain, participant global assessment of well-being, serious adverse events, and withdrawals due to adverse events. We used GRADE to assess the certainty of evidence for each outcome. MAIN RESULTS: We identified three new trials in this update, bringing the total number of included RCTs to five (575 participants). Three trials evaluated oral methotrexate versus placebo, one evaluated methotrexate plus intra-articular glucocorticoid (IAGC) therapy versus IAGC therapy alone, and one evaluated methotrexate versus leflunomide. Doses of methotrexate ranged from 5 mg/m2/week to 15 mg/m2/week in four trials, and participants in the methotrexate group of the remaining trial received 0.5 mg/kg/week. Trial size varied from 31 to 226 participants. The average age of participants ranged from four to 10 years. Most participants were females and most had nonsystemic JIA. The study that evaluated methotrexate plus IAGC therapy versus IAGC therapy alone recruited children and young people with the oligoarticular disease subtype of JIA. Two placebo-controlled trials and the trial of methotrexate versus leflunomide were adequately randomised and blinded, and likely not susceptible to important biases. One placebo-controlled trial may have been susceptible to selection bias due to lack of adequate reporting of randomisation methods. The trial investigating the addition of methotrexate to IAGC therapy was susceptible to performance and detection biases. Methotrexate versus placebo Methotrexate compared with placebo may increase the number of children and young people who achieve treatment response up to six months (absolute difference of 163 more per 1000 people; risk ratio (RR) 1.67, 95% confidence interval (CI) 1.21 to 2.31; I2 = 0%; 3 trials, 328 participants; low-certainty evidence). However, methotrexate compared with placebo may have little or no effect on pain as measured on an increasing scale of 0 to 100 (mean difference (MD) -1.10 points, 95% CI -9.09 to 6.88; 1 trial, 114 participants), improvement in participant global assessment of well-being (absolute difference of 92 more per 1000 people; RR 1.23, 95% CI 0.88 to 1.72; 1 trial, 176 participants), occurrence of serious adverse events (absolute difference of 5 fewer per 1000 people; RR 0.63, 95% CI 0.04 to 8.97; 3 trials, 328 participants), and withdrawals due to adverse events (RR 3.46, 95% CI 0.60 to 19.79; 3 trials, 328 participants) up to six months. We could not estimate the absolute difference for withdrawals due to adverse events because there were no withdrawals in the placebo group. All outcomes were reported within six months of randomisation. We downgraded the certainty of the evidence to low for all outcomes due to indirectness (suboptimal dosing of methotrexate and diverse outcome measures) and imprecision (few participants and low event rates). No trials reported function or the number of participants with sustained clinically inactive disease. Serious adverse events included liver derangement, abdominal pain, and inadvertent overdose. Methotrexate plus intra-articular corticosteroid therapy versus intra-articular corticosteroid therapy alone Methotrexate plus IAGC therapy compared with IAGC therapy alone may have little or no effect on the probability of sustained clinically inactive disease or the rate of withdrawals due to adverse events up to 12 months in children and young people with the oligoarticular subtype of JIA (low-certainty evidence). We could not calculate the absolute difference in withdrawals due to adverse events because there were no withdrawals in the control group. We are uncertain if there is any difference between the interventions in the risk of severe adverse events, because none were reported. The study did not report treatment response, function, pain, or participant global assessment of well-being. Methotrexate versus an alternative disease-modifying antirheumatic drug Methotrexate compared with leflunomide may have little or no effect on the probability of treatment response or on function, participant global assessment of well-being, risk of serious adverse events, and rate of withdrawals due to adverse events up to four months. We downgraded the certainty of the evidence for all outcomes to low due to imprecision. The study did not report pain or sustained clinically inactive disease. AUTHORS' CONCLUSIONS: Oral methotrexate (5 mg/m2/week to 15 mg/m2/week) compared with placebo may increase the number of children and young people achieving treatment response but may have little or no effect on pain or participant global assessment of well-being. Oral methotrexate plus IAGC injections compared to IAGC injections alone may have little or no effect on the likelihood of sustained clinically inactive disease among children and young people with oligoarticular JIA. Similarly, methotrexate compared with leflunomide may have little or no effect on treatment response, function, and participant global assessment of well-being. Serious adverse events due to methotrexate appear to be rare. We will update this review as new evidence becomes available to inform the living guideline.

Changes in Self-Reported and Device-Measured Physical Activity Before Abdominal Resection Surgery: A Meta-Analysis.

OBJECTIVE: To determine the effect of interventions on physical activity levels of patients awaiting abdominal resection surgery using self-reported as well as device-measured outcome measures. DATA SOURCE: PubMed and EMBASE databases were searched on the 18th of April 2023 up to April 2023 for studies on interventions to promote physical activity during the preoperative phase. REVIEW METHODS: Studies were included if pre- and post-intervention physical activity was measured between diagnosis and abdominal surgery. Risk of bias was assessed by the Physiotherapy Evidence Database (PEDro) assessment tool for trials. Meta-analyses were performed to assess the effect of the pre-surgery activity promoting interventions on self-reported and device-measured physical activity. RESULTS: Seventeen studies were included in the analysis with 452 subjects in the intervention groups. The random-effect meta-analysis showed a moderate improvement in intervention groups measures in pre-surgery physical activity levels compared to the baseline (SMD = 0.67, [CI = 0.30;1.03], I2 = 79%). The self-reported subgroup meta-analysis showed the largest increase in performed physical activity, (SMD = 0.78, [CI = 0.4;1.15], I2 = 79%) whilst non-significant increase was shown in the device-measured subgroup (SMD = 0.16, [CI = -0.64;0.97], I2 = 58%). CONCLUSION: Increasing physical activity in the preoperative phase is feasible. Self-reported physical activity outcome measures show larger effects compared to device-measured outcome measures. More high-quality research should be performed utilizing objective measures.

Detecting Asymmetry of Upper Limb Activity with Accelerometry in Infants at Risk for Unilateral Spastic Cerebral Palsy.

AIMS: To examine whether accelerometry can quantitate asymmetry of upper limb activity in infants aged 3-12 months at risk for developing unilateral spastic cerebral palsy (USCP). METHOD: A prospective study was performed in 50 infants with unilateral perinatal brain injury at high risk of developing USCP. Triaxial accelerometers were worn on the ipsilateral and contralesional upper limb during the Hand Assessment for Infants (HAI). Infants were grouped in three age intervals (3-5 months, 5-7.5 months and 7.5 until 12 months). Each age interval group was divided in a group with and without asymmetrical hand function based on HAI cutoff values suggestive of USCP. RESULTS: In a total of 82 assessments, the asymmetry index for mean upper limb activity was higher in infants with asymmetrical hand function compared to infants with symmetrical hand function in all three age groups (ranging from 41 to 51% versus - 2-6%, p < 0.01), while the total activity of both upper limbs did not differ. CONCLUSIONS: Upper limb accelerometry can identify asymmetrical hand function in the upper limbs in infants with unilateral perinatal brain injury from 3 months onwards and is complementary to the Hand Assessment for Infants.

Physical Activity Levels, Cardiorespiratory Fitness, and Physical Literacy in Children With a Chronic Medical Condition.

PURPOSE: This study investigated differences in cardiorespiratory fitness (CRF) and other physical literacy domains between children with a chronic medical condition (CMC) who adhered to with the physical activity guideline and those who did not. METHOD: Forty children with a CMC (7-12 years) wore an accelerometer for 7 days to measure moderate-to-vigorous physical activity. CRF and muscle power were assessed with a maximal cardiopulmonary exercise test, and motor performance was measured with an exercise course. Motivation, confidence, knowledge, and understanding were assessed with a questionnaire. RESULTS: Children who adhered to the PA guideline (n = 13) scored significantly higher for CRF, muscle power, motivation and confidence than children who did not adhere to the PA guideline (n = 27). No significant differences were found for motor performance, knowledge and understanding. CONCLUSION: Future research should investigate the effect of paying specific attention to motivation and confidence in children with a CMC to stimulate their adherence to the PA guideline.

Exercise capacity in a cohort of children with congenital heart disease.

In patients with congenital heart disease (CHD), reduced exercise capacity can be a predictor for late complications and may be used to guide interventions. Yet, the interpretation of exercise capacity is challenged by changes in body composition during growth. Our aim was to create an overview of disease-specific exercise capacity in children with CHD. We performed a multicentre retrospective study of exercise capacity of CHD patients, aged 6-18 years, tested between January 2001 and October 2018. Sex-specific distribution graphs were made using the LMS method and height to relate to body size. We included all CHD with N > 50, including severe defects (e.g., univentricular heart, tetralogy of Fallot) and "simple" lesions as ventricular septum defect and atrial septum defect. We included 1383 tests of 1208 individual patients for analysis. The peak oxygen uptake (VO2peak, 37.3 ml/min/kg (25th-75th percentile 31.3-43.8)) varied between specific defects; patients with univentricular hearts had lower VO2peak compared with other CHD. All groups had lower VO2peak compared to healthy Dutch children. Males had higher VO2peak, Wpeak and O2pulsepeak than females. Sex- and disease-specific distribution graphs for VO2peak, Wpeak and O2pulsepeak showed increase in variation with increase in height.   Conclusion: Disease-specific distribution graphs for exercise capacity in children with CHD from a large multicentre cohort demonstrated varying degrees of reduced VO2peak and Wpeak. The distribution graphs can be used in the structured follow-up of patients with CHD to predict outcome and identify patients at risk. What is Known: • Children with congenital heart disease (COnHD) are at risk to develop heart failure, arrhytmia's and other complications. Exercise capacity may be an important predictor for outcome in children with ConHD. In children, the interpretation of exercise capacity poses an additional challenge related to physical changes during growth. What is New: • In this report of a multi-center cohort >1300 childrewn with ConHD, we related the changes in exercise capacity to length. We demonstrated that exercise capacity was reduced as compared with healthy children and we observed variation between disease groups. Patients with a univentricular circulation (Fontan) had worse exercise capacity. We constructed disease specific charts of development of exercise capacity throughout childhood, accessible via a web-site. These graphs may help practitioner to guide children with ConHD.

Practical Aspects of Cardiopulmonary Exercise Testing in Children.

The use of cardiopulmonary exercise testing (CPET) in pediatrics provides critical insights into potential physiological causes of unexplained exercise-related complaints or symptoms, as well as specific pathophysiological patterns based on physiological responses or abnormalities. Furthermore, CPET helps evaluate exercise performance in children with chronic (lung/heart) diseases. For instance, it can ascertain any adverse reaction to exercise and estimate the effects of specific treatment measures. It affords a global assessment of the pathophysiological patterns, responses and abnormalities to exercise that is inadequately reflected by resting lung function and/or cardiac function assessment. Clinical interpretation of the results of a CPET in pediatrics requires specific knowledge regarding pathophysiological responses and interpretative strategies that can be adapted to address concerns specific to the child's medical condition or disability.

Is Measuring Physical Literacy in School-Aged Children With Cystic Fibrosis or Congenital Heart Disease Needed?

PURPOSE: To explore the association between cardiorespiratory fitness and other physical literacy domains in children with cystic fibrosis (CF) or congenital heart disease (CHD). METHODS: In 28 children with CF (n = 10) or CHD (n = 18), aged 7 to 11 years, cardiorespiratory fitness and the following physical literacy domains were measured: ( a ) physical competence, ( b ) motivation and confidence, ( c ) knowledge and understanding, and ( d ) daily behavior (ie, self-perceived moderate-to-vigorous physical activity [MVPA]). RESULTS: Cardiorespiratory fitness was significantly associated with motivation and confidence and self-perceived MVPA. There were no other significant associations. CONCLUSIONS: Cardiorespiratory fitness is associated with self-perceived MVPA, motivation, and confidence in children with CF or CHD.

Effects of 12 weeks of recreational football (soccer) with caloric control on glycemia and cardiovascular health of adolescent boys with type 1 diabetes.

OBJECTIVE: To determine the effects of recreational football combined with caloric control on glycemia and cardiovascular health of adolescent boys with type 1 diabetes. BACKGROUND: Though 12 weeks of physical activity alone improves the health of people with type 1 diabetes, there is little evidence that physical activity alone can improve glycemia in 12 weeks. RESEARCH DESIGN AND METHODS: The participants were divided into four groups as follows: football with diet, football-only, diet-only, and the control groups. Each group consisted of 10 participants. The football with diet and the football-only groups had 1.5 h of football twice a week for 12 weeks. The following outcomes were measured before and after 12 weeks: Glycated hemoglobin, fasting blood glucose, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and resting blood pressures. Changes were considered significant when p ≤ 0.050 and common language effect size ≤42% or common language effect size ≥58%. RESULTS: Glycated hemoglobin decreased in the football with diet group (mean change (standard deviation) = -0.9 (1.0) %, p = 0.019, and common language effect size = 31.5%) and was different from the control group (p = 2.4 × 10-4 and common language effect size = 95.5%.). However, none of the intervention groups showed a clear change in blood lipids nor blood pressure. CONCLUSIONS: 12 weeks of combined football with diet intervention provides the greatest improvement in glycemia in adolescent boys with type 1 diabetes.

Reference values for maximum oxygen uptake relative to body mass in Dutch/Flemish subjects aged 6-65 years: the LowLands Fitness Registry.

BACKGROUND: The maximum oxygen uptake (VO2max) during cardiopulmonary exercise testing (CPET) is considered the best measure of cardiorespiratory fitness. AIM: To provide up-to-date reference values for the VO2max per kilogram of body mass (VO2max/kg) obtained by CPET in the Netherlands and Flanders. METHODS: The Lowlands Fitness Registry contains data from health checks among different professions and was used for this study. Data from 4612 apparently healthy subjects, 3671 males and 941 females, who performed maximum effort during cycle ergometry were analysed. Reference values for the VO2max/kg and corresponding centile curves were created according to the LMS method. RESULTS: Age had a negative significant effect (p < .001) and males had higher values of VO2max/kg with an overall difference of 18.0% compared to females. Formulas for reference values were developed: Males: VO2max/kg = - 0.0049 × age2 + 0.0884 × age + 48.263 (R2 = 0.9859; SEE = 1.4364) Females: VO2max/kg = - 0.0021 × age2 - 0.1407 × age + 43.066 (R2 = 0.9989; SEE = 0.5775). Cross-validation showed no relevant statistical mean difference between measured and predicted values for males and a small but significant mean difference for females. We found remarkable higher VO2max/kg values compared to previously published studies. CONCLUSIONS: This is the first study to provide reference values for the VO2max/kg based on a Dutch/Flemish cohort. Our reference values can be used for a more accurate interpretation of the VO2max in the West-European population.

Sports Participation, Physical Activity, and Health-Related Fitness in Youth With Chronic Diseases or Physical Disabilities: The Health in Adapted Youth Sports Study.

ABSTRACT: Lankhorst, K, Takken, T, Zwinkels, M, van Gaalen, L, Velde, St, Backx, F, Verschuren, O, Wittink, H, and de Groot, J. Sports participation, physical activity, and health-related fitness in youth with chronic diseases or physical disabilities: the health in adapted youth sports study. J Strength Cond Res 35(8): 2327-2337, 2021-Youth with chronic diseases or physical disabilities (CDPD) often show reduced fitness and physical activity (PA) levels and participate less in organized sports compared with healthy peers. The purpose of this study was to examine the associations between participation in sports and health-related fitness and PA in youth with CDPD. A total of 163 subjects (mean age 14 years; range 8-19 years) with CDPD were included in this cross-sectional study, with 81 participating in organized sports and 82 not. Subjects were recruited between October 2014 and November 2016. Aerobic and anaerobic fitness, agility, and muscle strength were assessed in the laboratory, whereas PA was monitored in daily life using accelerometry during 1 week. Linear regression analyses were used to assess the associations of sports participation (independent variable) with health-related fitness and PA (dependent variables). Results show that youth with CDPD participating in organized sports 2 times a week performed better on all outcome measures. They reached a higher peak oxygen uptake (difference of 4.9 ml O2·kg-1·min-1, P = 0.001) compared with their peers not participating in sports. Also, anaerobic fitness, agility, muscle strength, and PA were all positively associated with sports participation. Moreover, the association between sports participation and aerobic fitness was mediated by PA for 31% (P = 0.045). In conclusion, participation in sports is associated with both higher levels of PA and health-related fitness in youth with CDPD. Promotion and stimulation of participation in sports seems a good way to promote health-related fitness as well as a healthy active lifestyle in youth with CDPD.

Nutritional ketosis improves exercise metabolism in patients with very long-chain acyl-CoA dehydrogenase deficiency.

A maladaptive shift from fat to carbohydrate (CHO) oxidation during exercise is thought to underlie myopathy and exercise-induced rhabdomyolysis in patients with fatty acid oxidation (FAO) disorders. We hypothesised that ingestion of a ketone ester (KE) drink prior to exercise could serve as an alternative oxidative substrate supply to boost muscular ATP homeostasis. To establish a rational basis for therapeutic use of KE supplementation in FAO, we tested this hypothesis in patients deficient in Very Long-Chain acyl-CoA Dehydrogenase (VLCAD). Five patients (range 17-45 y; 4 M/1F) patients were included in an investigator-initiated, randomised, blinded, placebo-controlled, 2-way cross-over study. Patients drank either a KE + CHO mix or an isocaloric CHO equivalent and performed 35 minutes upright cycling followed by 10 minutes supine cycling inside a Magnetic Resonance scanner at individual maximal FAO work rate (fatmax; approximately 40% VO2 max). The protocol was repeated after a 1-week interval with the alternate drink. Primary outcome measures were quadriceps phosphocreatine (PCr), Pi and pH dynamics during exercise and recovery assayed by in vivo 31 P-MR spectroscopy. Secondary outcomes included plasma and muscle metabolites and respiratory gas exchange recordings. Ingestion of KE rapidly induced mild ketosis and increased muscle BHB content. During exercise at FATMAX, VLCADD-specific plasma acylcarnitine levels, quadriceps glycolytic intermediate levels and in vivo Pi/PCr ratio were all lower in KE + CHO than CHO. These results provide a rational basis for future clinical trials of synthetic ketone ester supplementation therapy in patients with FAO disorders. Trial registration: ClinicalTrials.gov. Protocol ID: NCT03531554; METC2014.492; ABR51222.042.14.

Instruments Measuring Physical Activity in Individuals Who Use a Wheelchair: A Systematic Review of Measurement Properties.

OBJECTIVE: To systematically review the evidence evaluating validity or reliability of self-reported and device-based instruments, to measure physical activity (PA) in individuals who use a wheelchair, and to make recommendations for the selection of PA outcomes tools. DATA SOURCES: PubMed, Embase, and CINAHL were systematically searched. STUDY SELECTION: Studies reporting measurement properties of instruments to assess PA in individuals who use a wheelchair. DATA EXTRACTION: The Consensus-Based Standards for the Selection of Health Status Measurement Instruments checklist was used to assess the methodological quality of the included studies. The measurement properties of instruments assessing PA were examined. DATA SYNTHESIS: The search yielded 5341 records, 61 were considered relevant, 21 articles were included. A best evidence synthesis was performed on 9 studies including 4 self-reported instruments and 13 studies including 8 device-based instruments. One study evaluated both self-reported and device-based instruments. The overall methodological quality of all studies ranged from poor to excellent. Variable levels of evidence were found for both the validity and reliability for self-reported instruments and for criterion validity for device-based instruments. CONCLUSIONS: The Physical Activity Scale for Individuals with Disabilities (PASIPD) and The Physical Activity Recall Assessment for People with Spinal Cord Injury (PARA-SCI) seem the most promising self-reported instruments for measuring the intensity of PA. Device-based instruments that can be used for measuring both the intensity and type of PA are the GENEActiv, Actigraph GT3X+, Actiheart, or the Physical Activity Monitor System (PAMS), showing moderate evidence for a positive rating of criterion validity. For measuring the type of PA, the PAMS and VitaMove are suitable, showing both good evidence for a positive rating of criterion validity.

Improvement of exercise capacity following neonatal respiratory failure: A randomized controlled trial.

Exercise capacity deteriorates in school-aged children born with major anatomical foregut anomalies and/or treated with extracorporeal membrane oxygenation. The aim of the present study was to evaluate whether exercise capacity can be improved in the short term and long term in children born with anatomical foregut anomalies and/or treated with extracorporeal membrane oxygenation. Therefore, we evaluated two different interventions in this single-blinded randomized controlled trial. Forty participants were randomly assigned to group A: standardized anaerobic high-intensity interval training plus online lifestyle coaching program, B: online lifestyle coaching program only, or C: standard of care. Inclusion criteria were as follows: score ≤-1 standard deviation (SD) on the Bruce protocol. Exercise capacity was assessed at baseline (T0), after 3 months (T1), and after 12 months (T2). Exercise capacity improved over time: mean (SD) standard deviation score (SDS) endurance time: T0 -1.91 (0.73); T1 -1.35 (0.94); T2 -1.20 (1.03): both P < .001. No significant differences in maximal endurance time were found at T1 (group A-C: estimated mean difference (SDS): 0.06 P = .802; group B-C: -0.17 P = .733) or T2 (group A-C: -0.13 P = .635; group B-C: -0.18 P = .587). Exercise capacity improved significantly over time, irrespective of the study arm. Not only residual morbidities may be responsible for reduced exercise capacity. Parental awareness of reduced exercise capacity rather than specific interventions may have contributed. Monitoring of exercise tolerance and providing counseling on lifestyle factors that improve physical activity should be part of routine care, and aftercare should be offered on an individual basis.

Cardiopulmonary Exercise Testing Provides Additional Prognostic Information in Cystic Fibrosis.

RATIONALE: The prognostic value of cardiopulmonary exercise testing (CPET) for survival in cystic fibrosis (CF) in the context of current clinical management, when controlling for other known prognostic factors, is unclear. OBJECTIVES: To determine the prognostic value of CPET-derived measures beyond peak oxygen uptake ( V. o2peak) following rigorous adjustment for other predictors. METHODS: Data from 10 CF centers in Australia, Europe, and North America were collected retrospectively. A total of 510 patients completed a cycle CPET between January 2000 and December 2007, of which 433 fulfilled the criteria for a maximal effort. Time to death/lung transplantation was analyzed using Cox proportional hazards regression. In addition, phenotyping using hierarchical Ward clustering was performed to characterize high-risk subgroups. MEASUREMENTS AND MAIN RESULTS: Cox regression showed, even after adjustment for sex, FEV1% predicted, body mass index (z-score), age at CPET, Pseudomonas aeruginosa status, and CF-related diabetes as covariates in the model, that V. o2peak in % predicted (hazard ratio [HR], 0.964; 95% confidence interval [CI], 0.944-0.986), peak work rate (% predicted; HR, 0.969; 95% CI, 0.951-0.988), ventilatory equivalent for oxygen (HR, 1.085; 95% CI, 1.041-1.132), and carbon dioxide (HR, 1.060; 95% CI, 1.007-1.115) (all P < 0.05) were significant predictors of death or lung transplantation at 10-year follow-up. Phenotyping revealed that CPET-derived measures were important for clustering. We identified a high-risk cluster characterized by poor lung function, nutritional status, and exercise capacity. CONCLUSIONS: CPET provides additional prognostic information to established predictors of death/lung transplantation in CF. High-risk patients may especially benefit from regular monitoring of exercise capacity and exercise counseling.

Sleep quantity and its relation with physical activity in children with cerebral palsy; insights using actigraphy.

AIM: To objectively assess the sleep quantity, and explore the relationships between sleep quantity and quality, and physical activity and sedentary behaviour in children and adolescents with cerebral palsy (CP). METHODS: An observational cross-sectional study was conducted. In total, 36 children with spastic CP (mean age 15y 4mo, SD 2y 6mo; classified as Gross Motor Function Classification System levels I (25), II (9), III (1) and IV (1)) were included. Active time, sedentary time and sleep quantity were measured using an activity monitor for 7 consecutive days. RESULTS: Total sleep duration of children with CP ranged between 7.2 and 11.2 h. No significant correlations were found between active time and sleep quantity for total week, weekdays, and weekend days. Moderate negative correlations were found between sedentary time and sleep quantity during total week (r = -0.456, P = 0.005), weekdays (r = -0.453, P = 0.006) and weekend days (r = -0.48, P = 0.003). CONCLUSIONS: Our findings suggest that children with CP are getting the recommended sleep duration, and that sedentary behaviour is correlated with sleep quantity in children with CP and may be more applicable to children with better motor functions. Future studies using more elaborate, objective sleep quantity and quality measures are recommended.

Longitudinal development of cancer-related fatigue and physical activity in childhood cancer patients.

PURPOSE: Cancer-related fatigue is one of the most distressing side effects of childhood cancer treatment. Physical activity can decrease fatigue and has positive effects on other health outcomes. Most research on physical activity pertains to adults, and the few studies that focus on children have limited follow-up time. This study evaluates cancer-related fatigue in children and its association with physical activity over a one-year time period. METHODS: Sixty-eight children with cancer (7-18 years) were recruited during or within the first year after treatment. Physical activity (Actical activity monitor) and cancer-related fatigue (Pediatric Quality-of-Life Questionnaire Multidimensional Fatigue Scale (PedsQL-MFS), self- and parent- reports) were assessed at baseline, 4 months, and 12 months. PedsQL-MFS scores were compared with Dutch norms. Longitudinal association of cancer-related fatigue with physical activity was evaluated (No. NTR 1531). RESULTS: Generally, PedsQL-MFS scores were worse than norms at baseline and 4 months, and recovered by 12 months except for the parent-proxy scores in adolescents. Younger children (≤12 years) self-reported comparable or better scores than norms. Physical activity generally improved over time, but patients mostly remained sedentary. During follow-up, increased physical activity was associated with less cancer-related fatigue. CONCLUSION: Cancer-related fatigue in children improves over time, and increased physical activity is associated with less cancer-related fatigue. Given the sedentary lifestyle of this population, the positive effect of physical activity on cancer-related fatigue, and the many other health benefits of an active lifestyle, it is important to stimulate physical activity in childhood cancer patients and survivors.

Assessment of fatigability in patients with spinal muscular atrophy: development and content validity of a set of endurance tests.

BACKGROUND: Fatigability has emerged as an important dimension of physical impairment in patients with Spinal Muscular Atrophy (SMA). At present reliable and valid outcome measures for both mildly and severely affected patients are lacking. Therefore the primary aim of this study is the development of clinical outcome measures for fatigability in patients with SMA across the range of severity. METHODS: We developed a set of endurance tests using five methodological steps as recommended by the 'COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN). In this iterative process, data from multiple sources were triangulated including a scoping review of scientific literature, input from a scientific and clinical multidisciplinary expert panel and three pilot studies including healthy persons (N = 9), paediatric patients with chronic disorders (N = 10) and patients with SMA (N = 15). RESULTS: Fatigability in SMA was operationalised as the decline in physical performance. The following test criteria were established; one method of testing for patients with SMA type 2-4, a set of outcome measures that mimic daily life activities, a submaximal test protocol of repetitive activities over a longer period; external regulation of pace. The scoping review did not generate suitable outcome measures. We therefore adapted the Endurance Shuttle Walk Test for ambulatory patients and developed the Endurance Shuttle Box and Block Test and the - Nine Hole Peg Test for fatigability testing of proximal and distal arm function. Content validity was established through input from experts and patients. Pilot testing showed that the set of endurance tests are comprehensible, feasible and meet all predefined test criteria. CONCLUSIONS: The development of this comprehensive set of endurance tests is a pivotal step to address fatigability in patients with SMA.

Physical activity and sedentary behaviour in children with spina bifida.

AIM: To describe active and sedentary time in children with spina bifida and to compare their physical activity on weekdays versus weekends. METHOD: In this exploratory cross-sectional study, data from 13 Canadian and 22 Dutch children with spina bifida (14 females, 21 males; mean age 10y 11mo, standard deviation [SD] 3y 6mo, range 5y 6mo-18y; Hoffer classification distribution: community [n=28], household [n=3], non-functional [n=3], and non-ambulator [n=1]) were analysed. Objective measures of physical activity and sedentary behaviour were obtained by using ActiGraph or Actiheart activity monitors. Data for the participants wearing the ActiGraph were compared with age- and sex-matched controls that were developing typically using independent-samples t-tests. Activity data collected on weekdays was compared to those on weekends. RESULTS: ActiGraph data demonstrated children with spina bifida spent more time sedentary (mean [SD] 49.5min/h [5.78]) and less time in moderate to vigorous physical activity (mean [SD] 2.33min/h [1.61]) compared with the typically developing group (mean [SD] 41.0min/h [5.76] and 5.46min/h [2.13], p=0.001 and p<0.001 respectively). For both ActiGraph- and Actiheart-derived data, physical activity and sedentary time were not significantly different between weekdays and weekends. INTERPRETATION: Children with spina bifida have reduced levels of physical activity and increased sedentary behaviour, with no statistical differences seen between weekdays and weekends. Several methodological issues related to activity monitoring warrant consideration when choosing the appropriate method to quantify physical activity and sedentary behaviour. WHAT THIS PAPER ADDS: Reduced levels of physical activity and sedentary time were quantified in children with spina bifida. Objective quantification of physical behaviour in ambulatory and non-ambulatory school-aged children with spina bifida is possible.

ACTIVIDAD FÍSICA Y COMPORTAMIENTO SEDENTARIO EN NIÑOS CON ESPINA BÍFIDA: OBJETIVO: Describir el tiempo activo y sedentario en niños con espina bífida y comparar su actividad física entre semana y fines de semana. MÉTODO: En este estudio exploratorio de corte transversal, datos de 13 niños canadienses y 22 holandeses con espina bífida (14 mujeres, 21 varones; edad media 10 años 11 meses, desviación estándar [DE] 3 años 6 meses, rango 5 años 6 meses - 18 años; se analizaron en base a la distribución basada en la clasificación de Hoffer: comunidad [n = 28], hogar [n = 3], no funcional [n = 3] y no ambulatorios [n = 1]). Se obtuvieron medidas objetivas de actividad física y comportamiento sedentario mediante el uso de monitores de actividad ActiGraph o Actiheart. Los datos de los participantes que usaban el ActiGraph se compararon con los niños con desarrollo típicos como controles, apareados por edad y sexo usando pruebas t de muestras independientes. Los datos de actividad recopilados durante la semana se compararon con los de los fines de semana. RESULTADOS: Los datos de ActiGraph demostraron que los niños con espina bífida pasaron más tiempo sedentarios (media [DE] 49,5min / h [5,78]) y menos tiempo en actividades físicas moderadas a intensas (media [DE] 2,33min / h [1,61]) en comparación con el grupo control (media [DE] 41,0min / h [5,76] y 5,46min / h [2,13], p = 0,001 y p <0,001 respectivamente). Tanto para los datos derivados de ActiGraph como de Actiheart, la actividad física y el tiempo sedentario no fueron significativamente diferentes entre semana y fines de semana. INTERPRETACIÓN: Los niños con espina bífida tienen niveles reducidos de actividad física y un mayor comportamiento sedentario, sin diferencias estadísticas observadas entre semana y fines de semana. Al elegir un método apropiado para cuantificar la actividad física y el comportamiento sedentario se deben considerar varios problemas metodológicos relacionados con el monitoreo de la actividad en esta población.

ATIVIDADE FÍSICA E COMPORTAMENTO SEDENTÁRIO EM CRIANÇAS COM ESPINHA BÍFIDA: OBJETIVO: Descrever o tempo ativo e sedentário de crianças com espinha bífida e comparar sua atividade física durante a semana versus fins de semana. MÉTODO: Neste estudo exploratório transversal, dados de 13 crianças canadenses e 22 crianças holandesas com espinha bífida (14 do sexo feminino, 21 do sexo masculino; médida de idade 10a 11m, desvio padrão [DP] 3a 6m, variação 5a 6m-18a; Distribuição da classificação de Hoffer: comunidade [n=28], domiciliar [n=3], não funcional [n=3], e não ambulatório [n=1]) foram analisados. Medidas objetivas de atividade física e comportamento sedentário foram obtidas por meio de monitores de atividade ActiGraph ou Actiheart. Dados para os participantes que usaram ActiGraph foram comparados com controles pareados por idade e sexo que tinha desenvolvimento típico usando testes t para amostras independentes. Dados de atividade coletados durante a semana foram comparados com os dados de finais de semana. RESULTADOS: Os dados do ActiGraph demonstraram que crianças com espinha bífida passaram mais tempo sedentário (média [DP] 49,5min/h [5,78]) e menos tempo em atividade física vigorosa (média [DP] 2,33min/h [1,61]) comparados com o grupo com desenvolvimento típico (média [DP] 41,0min/h [5,76] e 5,46min/h [2,13], p=0,001 and p<0,001 respectivamente). Para os dados derivados do ActiGraph- e Actiheart, a atividade física e o tempo sedentário não foram significativamente diferentes comparando os dias da semana com os fins de semana. INTERPRETAÇÃO: Crianças com espinha bífida têm níveis reduzidos de atividade física e comportamento sedentário aumentado, sem diferenças estatísticas quando comparados os dias da semana. Várias questões metodológicas relacionadas ao monitoramento de atividades indicam necessidade de refletir quanto à escolha do método apropriado para quantificar a ativiade física e comportamento sedentário.

Peak oxygen uptake cut-points to identify children at increased cardiometabolic risk - The PANIC Study.

We aimed to develop cut-points for directly measured peak oxygen uptake ( V ˙ O 2 peak ) to identify boys and girls at increased cardiometabolic risk using different scaling methods to control for body size and composition. Altogether 352 children (186 boys, 166 girls) aged 9-11 years were included in the analyses. We measured V̇O2peak directly during a maximal cycle ergometer exercise test and lean body mass (LM) by bioelectrical impedance. We computed a sex- and age-specific cardiometabolic risk score (CRS) by summing important cardiometabolic risk factors and defined increased cardiometabolic risk as >1 standard deviation above the mean of CRS. Receiver operating characteristics curves were used to detect V̇O2peak cut-points for increased cardiometabolic risk. Boys with V̇O2peak <45.8 mL kg body mass (BM)-1  min-1 (95% confidence interval [CI] = 45.1 to 54.6, area under the curve [AUC] = 0.86, P < 0.001) and <63.2 mL kg LM-1  min-1 (95% CI =52.4 to 67.5, AUC = 0.65, P = 0.006) had an increased CRS. Girls with V̇O2peak <44.1 mL kg BM-1  min-1 (95% CI = 44.0 to 58.6, AUC = 0.67, P = 0.013) had an increased CRS. V̇O2peak scaled by BM-0.49 and LM-0.77 derived from log-linear allometric modeling poorly predicted increased cardiometabolic risk in boys and girls. In conclusion, directly measured V ˙ O 2 peak <45.8 mL kg BM-1  min-1 among boys and <44.1 mL kg BM-1  min-1 among girls were cut-points to identify those at increased cardiometabolic risk. Appropriately controlling for body size and composition reduced the ability of cardiorespiratory fitness to identify children at increased cardiometabolic risk.