Dextroamphetamine Treatment in Children With Hypothalamic Obesity

INTRODUCTION: Hypothalamic obesity (HO) in children has severe health consequences. Lifestyle interventions are mostly insufficient and currently no drug treatment is approved for children with HO. Amphetamines are known for their stimulant side-effect on resting energy expenditure (REE) and suppressing of appetite. Earlier case series have shown positive effects of amphetamines on weight in children with acquired HO. We present our experiences with dextroamphetamine treatment in the, up to now, largest cohort of children with HO. METHODS: A retrospective cohort evaluation was performed of children with HO treated with dextroamphetamine at two academic endocrine pediatric clinics. Off-label use of dextroamphetamine was initiated in patients with progressive, therapy-resistant acquired or congenital HO. Anthropometrics, REE, self-reported (hyperphagic) behavior and energy level, and side effects were assessed at start and during treatment. RESULTS: Nineteen patients with a mean age of 12.3 ± 4.0 years had been treated with dextroamphetamine. In two patients, ΔBMI SDS could not be evaluated due to short treatment duration or the simultaneous start of extensive lifestyle treatment. Mean treatment duration of the 17 evaluated patients was 23.7 ± 12.7 months. Fourteen patients (n = 10 with acquired HO, n = 4 with congenital HO) responded by BMI decline or BMI stabilization (mean ΔBMI SDS of -0.6 ± 0.8, after a mean period of 22.4 ± 10.5 months). In three patients, BMI SDS increased (mean ΔBMI SDS of +0.5 ± 0.1, after a mean period of 29.7 ± 22.6 months). In 11 responders, measured REE divided by predicted REE increased with +8.9%. Thirteen patients (68.4%) reported decreased hyperphagia, improvement of energy level and/or behavior during treatment. Two patients developed hypertension during treatment, which resulted in dosage adjustment or discontinuation of treatment. Twelve children continued treatment at last moment of follow-up. CONCLUSION: In addition to supportive lifestyle interventions, dextroamphetamine treatment may improve BMI in children with HO. Furthermore, dextroamphetamines have the potential to decrease hyperphagia and improve resting energy expenditure, behavior, and energy level. In patients with acquired HO, these effects seem to be more pronounced when compared to patients with congenital HO. Future studies are needed to support these results

The role of physical activity and fitness in peope with Cystic Fibrosis compared to healthy people

This thesis describes the physical activity behaviors and factors and settings which influence this behavior in Dutch youth with and without a chronic disease and disability. In addition, the (possible) changes in cardiorespiratory fitness as a result of improved Cystic Fibrosis (CF) care, possible limiting factors and new promising medication were described. The prevalence of severe fatigue and the relation between psychosocial and clinical measured factors were assessed. Higher levels of cardiorespiratory fitness are related to survival and higher quality of life scores in people with CF. Therefore, increasing our knowledge regarding cardiorespiratory fitness in this period subject to change, is clinically relevant. In 2010 up to 2014, only 29% of the Dutch youth met the physical activity guidelines (to be at least moderately active for at least 60 minutes per day) and 43% had at least two hours of screen time. A lot of daily physical activity comes from cycling to school. Unfortunately, physical activity of especially scholars from cluster schools is lower. The Dutch youth with a chronic disease is not able to participate unlimited in sports and exercise. More than half (55%) of our pediatric population (n=60) with CF, with no ventilatory limitation, has still low cardiorespiratory fitness levels, despite improved CF care. Children and adolescents performed a cardiopulmonary exercise test (CPET) to establish cardiorespiratory fitness levels (peak oxygen uptake- VO2peak). Patients were categorized as having low cardiorespiratory fitness when VO2peak relative to body weight < 82% of predicted. Logistic regression analysis showed that glucose intolerance and colonization with P. Aeruginosa were most strongly related to lower levels of fitness. The maximal cardiac output during maximal exercise, determined by stroke volume and heart rate, is lower in both children and adults with CF (n=19) compared to healthy people (n=22). A blunted heart rate response, also named chronotropic incompetence, might be a cause for this lower cardiac output. Training intensities based on predicted heart rates seem inadequate for people with CF. In our adult CF population (n=77), 26% reported to experience severe fatigue. Patients could be categorized as experiencing ‘severe fatigue’ when the score on the Checklist Individual Strength-20 questionnaire was at least 35. Linear regression analysis showed a relation between fatigue and pulmonary function and functional capacity, measured with the Modified Shuttle Test. Stronger relations were however found for fatigue and patient-reported outcome measures such as physical and emotional functioning. In a small group of people (n=7) with CF and the S1251N mutation, we found an increase in pulmonary function and body mass index after a median of 15 [IQR 13-16] months of treatment with ivacaftor, a CFTR modulator drug. Remarkable was a decrease in cardiorespiratory fitness, which might be explained by a lower work of breathing. Maintaining and improving cardiorespiratory fitness remains an important treatment aim for CF physiotherapy care

2017 Dutch Report Card+: Results From the First Physical Activity Report Card Plus for Dutch Youth With a Chronic Disease or Disability

Background: The Dutch Active Healthy Kids (AHK) Report Card+ (RC+) consolidates and translates research and assesses how the Netherlands is being responsible in providing physical activity (PA) opportunities for youth (&lt; 18 years) with a chronic disease or disability. The aim of this article is to summarize the results of the Dutch RC+.Methods: Nine indicators were graded using the AHK Global Alliance RC development process, which includes a synthesis of best available research, surveillance, policy and practice findings, and expert consensus. Two additional indicators were included: weight status and sleep.Results: Grades assigned were: Overall Physical Activity, D; Organized Sports Participation, B–; Active Play, C–; Active Transportation, A–; Sedentary Behavior, C; Sleep C; For Weight Status, Family and Peers, School, Community and Built Environment, Government Strategies, and Investments all INC.Conclusions: The youth with disabilities spend a large part of the day sedentary, since only 26% of them met the PA norm for healthy physical activity. Potential avenues to improve overall physical activity are changing behaviors regarding sitting, screen time, and active play. The Netherlands is on track regarding PA opportunities for youth with disabilities, however they are currently not able to participate unlimited in sports and exercise

2017 Dutch Report Card : Results From the First Physical Activity Report Card Plus for Dutch Youth With a Chronic Disease or Disability

Background: The Dutch Active Healthy Kids (AHK) Report Card+ (RC+) consolidates and translates research and assesses how the Netherlands is being responsible in providing physical activity (PA) opportunities for youth (< 18 years) with a chronic disease or disability. The aim of this article is to summarize the results of the Dutch RC+. Methods: Nine indicators were graded using the AHK Global Alliance RC development process, which includes a synthesis of best available research, surveillance, policy and practice findings, and expert consensus. Two additional indicators were included: weight status and sleep. Results: Grades assigned were: Overall Physical Activity, D; Organized Sports Participation, B-; Active Play, C-; Active Transportation, A-; Sedentary Behavior, C; Sleep C; For Weight Status, Family and Peers, School, Community and Built Environment, Government Strategies, and Investments all INC. Conclusions: The youth with disabilities spend a large part of the day sedentary, since only 26% of them met the PA norm for healthy physical activity. Potential avenues to improve overall physical activity are changing behaviors regarding sitting, screen time, and active play. The Netherlands is on track regarding PA opportunities for youth with disabilities, however they are currently not able to participate unlimited in sports and exercise

Is our Youth Cycling to Health? : Results From the Netherlands' 2016 Report Card on Physical Activity for Children and Youth

BACKGROUND: The Active Healthy Kids the Netherlands (AHKN) Report Card consolidates and translates research and assesses how the Netherlands is being responsible in providing physical activity (PA) opportunities for children and youth (<18 years). The primary aim of this article is to summarize the results of the 2016 AHKN Report Card. METHODS: Nine indicators were graded using the Active Healthy Kids Global Alliance report card development process, which includes a synthesis of the best available research, surveillance, policy and practice findings, and expert consensus. RESULTS: Grades assigned were: Overall Physical Activity Levels, D; Organized Sport Participation, B; Active Play, B; Active Transportation, A; Sedentary Behaviors, C; Family and Peers, B; School, C; Community and the Built Environment, A; Government Strategies and Investments, INC. CONCLUSIONS: Sedentary behavior and overall PA levels are not meeting current guidelines. However, the Dutch youth behaviors in sports, active transportation, and active play are satisfactory. Several modifiable factors of influence might be enhanced to improve these indicators or at least prevent regression. Although Dutch children accumulate a lot of daily PA through cycling, it is not enough to meet the current national PA guidelines of 60 minutes of moderate-to-vigorous PA per day

A systematic approach to interpreting the cardiopulmonary exercise test in pediatrics

The use of cardiopulmonary exercise testing 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. Clinical interpretation of the results of a cardiopulmonary exercise test in pediatrics requires specific knowledge with regard to pathophysiological responses and interpretative strategies that can be adapted to address concerns specific to the child’s medical condition or disability. In this review, the authors outline the 7-step interpretative approach that they apply in their outpatient clinic for diagnostic, prognostic, and evaluative purposes. This approach allows the pediatric clinician to interpret cardiopulmonary exercise testing results in a systematic order to support their physiological reasoning and clinical decision making

Is our Youth Cycling to Health? : Results From the Netherlands' 2016 Report Card on Physical Activity for Children and Youth

BACKGROUND: The Active Healthy Kids the Netherlands (AHKN) Report Card consolidates and translates research and assesses how the Netherlands is being responsible in providing physical activity (PA) opportunities for children and youth (<18 years). The primary aim of this article is to summarize the results of the 2016 AHKN Report Card. METHODS: Nine indicators were graded using the Active Healthy Kids Global Alliance report card development process, which includes a synthesis of the best available research, surveillance, policy and practice findings, and expert consensus. RESULTS: Grades assigned were: Overall Physical Activity Levels, D; Organized Sport Participation, B; Active Play, B; Active Transportation, A; Sedentary Behaviors, C; Family and Peers, B; School, C; Community and the Built Environment, A; Government Strategies and Investments, INC. CONCLUSIONS: Sedentary behavior and overall PA levels are not meeting current guidelines. However, the Dutch youth behaviors in sports, active transportation, and active play are satisfactory. Several modifiable factors of influence might be enhanced to improve these indicators or at least prevent regression. Although Dutch children accumulate a lot of daily PA through cycling, it is not enough to meet the current national PA guidelines of 60 minutes of moderate-to-vigorous PA per day

Prevalence of severe fatigue among adults with cystic fibrosis : A single center study

Background: With life expectancy increasing among patients with cystic fibrosis (CF), the prevalence of complications such as fatigue is also expected to increase. Our aim was to investigate the prevalence of severe fatigue among adults with CF and to identify factors associated with fatigue. Methods: Adult patients with CF receiving treatment at a single center were invited to complete three questionnaires. We then studied the associations between fatigue and clinically measured parameters and between fatigue and patient-reported outcomes. Results: A total of 77 patients (age 19–54 years; 56% males; mean FEV1: 63%) completed the questionnaires (43% response rate). The prevalence of severe fatigue among these patients was 26%. The variance in fatigue was explained partially by clinically measured parameters. However, patient-reported outcomes were stronger independently associated with fatigue and included the patients' reported respiratory symptoms, emotional functioning, and social functioning. Conclusions: Fatigue is a clinically important and highly prevalent issue among adults with CF and is associated with a significant reduction in health-related quality of life and participation in society. In addition, fatigue is associated more strongly with the patient's perception of symptoms and well-being than with clinically measured parameters

Physiological predictors of cardiorespiratory fitness in children and adolescents with cystic fibrosis without ventilatory limitation

Objectives: [1] To investigate the cardiorespiratory fitness (CRF) levels in children and adolescents with cystic fibrosis (CF) with no ventilatory limitation (ventilatory reserve ⩾ 15%) during exercise, and [2] to assess which physiological factors are related to CRF. Methods: A cross-sectional study design was used in 8- to 18-year-old children and adolescents with CF. Cardiopulmonary exercise testing was used to determine peak oxygen uptake normalized to body weight as a measure of CRF. Patients were defined as having ‘low CRF’ when CRF was less than 82%predicted. Physiological predictors used in this study were body mass index z-score, P. Aeruginosa lung infection, impaired glucose tolerance (IGT) including CF-related diabetes, CF-related liver disease, sweat chloride concentration, and self-reported physical activity. Backward likelihood ratio (LR) logistic regression analysis was used. Results: Sixty children and adolescents (51.7% boys) with a median age of 15.3 years (25th–75th percentile: 12.9–17.0 years) and a mean percentage predicted forced expiratory volume in 1 second of 88.5% (±16.9) participated. Mean percentage predicted CRF (ppVO 2peak/kg ) was 81.4% (±12.4, range: 51%–105%). Thirty-three patients (55.0%) were classified as having ‘low CRF’. The final model that best predicted low CRF included IGT ( p  = 0.085; Exp(B) = 6.770) and P. Aeruginosa lung infection (p = 0.095; Exp(B) = 3.945). This model was able to explain between 26.7% and 35.6% of variance. Conclusions: CRF is reduced in over half of children and adolescents with CF with normal ventilatory reserve. Glucose intolerance and P. Aeruginosa lung infection seem to be associated to low CRF in children and adolescents with CF