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Background Bioimpedance spectroscopy (BIS) with a whole-body model to distinguish excess fluid from major body tissue hydration can provide objective assessment of fluid status. BIS is integrated into the Body Composition Monitor (BCM) and is validated in adults, but not children. This study aimed to (1) assess agreement between BCM-measured total body water (TBW) and a gold standard technique in healthy children, (2) compare TBW_BCM with TBW from Urea Kinetic Modelling (UKM) in haemodialysis children and (3) investigate systematic deviation from zero in measured excess fluid in healthy children across paediatric age range. Methods TBW_BCM and excess fluid was determined from standard wrist-to-ankle BCM measurement. TBW_D2O was determined from deuterium concentration decline in serial urine samples over 5 days in healthy children. UKM was used to measure body water in children receiving haemodialysis. Agreement between methods was analysed using paired t test and Bland-Altman method comparison. Results In 61 healthy children (6-14 years, 32 male), mean TBW_BCM and TBW_D2O were 21.1 ± 5.6 and 20.5 ± 5.8 L respectively. There was good agreement between TBW_BCM and TBW_D2O (R.sup.2 = 0.97). In six haemodialysis children (4-13 years, 4 male), 45 concomitant measurements over 8 months showed good TBW_BCM and TBW_UKM agreement (mean difference - 0.4 L, 2SD = ± 3.0 L). In 634 healthy children (2-17 years, 300 male), BCM-measured overhydration was - 0.1 ± 0.7 L (10-90th percentile - 0.8 to + 0.6 L). There was no correlation between age and OH (p = 0.28). Conclusions These results suggest BCM can be used in children as young as 2 years to measure normally hydrated weight and assess fluid status., Author(s): Indranil Dasgupta [sup.1] , David Keane [sup.2] , Elizabeth Lindley [sup.2] , Ihab Shaheen [sup.3] , Kay Tyerman [sup.3] , Franz Schaefer [sup.4] , Elke Wühl [sup.4] , Manfred [...]

Background In patients on conventional hemodialysis (HD), morbidity is high and quality of life is poor. Intensified HD programs have been developed to help overcome these shortcomings, but very few pediatric dialysis centers have reported the implementation of such a HD program. Methods An online survey was sent to all 221 pediatric dialysis centers which participate in the International Pediatric Dialysis Network (IPDN). The aim of the survey was to assess the attitude of pediatric nephrologists towards intensified HD, the penetrance of intensified HD into their clinical practice and barriers to implementation. Results Of the 221 pediatric dialysis centers sent the survey respondents from 61% (134) replied. Among these respondents, 69% acknowledged being aware of the evidence in support of the use of intensified HD, independent of whether intensified HD was offered at their own center, and 50% associated the use of daily nocturnal HD with the best overall patient outcome. In contrast, only 2% of respondents were in favor of conventional HD. Overall, 38% of the respondents stated that at their center intensified HD is prescribed to a subgroup of patients, most commonly in the form of short daily HD sessions. The most important barriers to expansion of intensified HD programs were lack of adequate funding (66%) and shortage of staff (63%), whereas lack of expertise and of motivation were reported infrequently as obstacles (21 and 14%, respectively). Conclusion Intensified HD is considered by many pediatric nephrologists to be the dialysis modality most likely associated with the best patient outcome. The limited use of this treatment approach highlights the importance of defining and successfully addressing the barriers to implementation. Keywords Children. Home dialysis. Financial barriers. Intensified hemodialysis. Nocturnal hemodialysis, Introduction Juvenile onset of chronic kidney disease (CKD) is associated with excessive morbidity and mortality. The mortality from cardiovascular disease (CVD) is 1000-fold higher in children on hemodialysis (HD) than [...]

Context: Burosumab has been approved for the treatment of children and adults with X-linked hypophosphatemia (XLH). Real-world data and evidence for its efficacy in adolescents are lacking. Objective: To assess the effects of 12 months of burosumab treatment on mineral metabolism in children (aged <12 years) and adolescents (aged 12-18 years) with XLH. Design: Prospective national registry. Setting: Hospital clinics. Patients: A total of 93 patients with XLH (65 children, 28 adolescents). Main Outcome Measures: Z scores for serum phosphate, alkaline phosphatase (ALP), and renal tubular reabsorption of phosphate per glomerular filtration rate (TmP/GFR) at 12 months. Results: At baseline, patients showed hypophosphatemia (−4.4 SD), reduced TmP/GFR (−6.5 SD), and elevated ALP (2.7 SD, each P < .001 vs healthy children) irrespective of age, suggesting active rickets despite prior therapy with oral phosphate and active vitamin D in 88% of patients. Burosumab treatment resulted in comparable increases in serum phosphate and TmP/GFR in children and adolescents with XLH and a steady decline in serum ALP (each P < .001 vs baseline). At 12 months, serum phosphate, TmP/GFR, and ALP levels were within the age-related normal range in approximately 42%, 27%, and 80% of patients in both groups, respectively, with a lower, weight-based final burosumab dose in adolescents compared with children (0.72 vs 1.06 mg/kg, P < .01). Conclusions: In this real-world setting, 12 months of burosumab treatment was equally effective in normalizing serum ALP in adolescents and children, despite persistent mild hypophosphatemia in one-half of patients, suggesting that complete normalization of serum phosphate is not mandatory for substantial improvement of rickets in these patients. Adolescents appear to require lower weight-based burosumab dosage than children. [ABSTRACT FROM AUTHOR]

Notwithstanding two decades of policy and legislation in Europe, aimed to foster research and development in rare conditions, only 5–6% of rare diseases have dedicated treatments. Given with the huge number of conditions classed as rare (which is increasing all the time), this equates to major unmet need for patients (over 30 million in the EU alone). Worryingly, the pace of Research and Innovation in Europe is lagging behind other regions of the world, and a seismic shift in the way in which research is planned and delivered is required, in order to remain competitive and—most importantly—bring meaningful, disease-altering treatments to those who desperately need them. The European Reference Networks (ERNs), launched in 2017, hold major potential to alleviate many of these challenges, and more, but only if adequately supported (financially, technically, and via robust policies and infrastructure) to realise that potential: and even then, only if able to forge robust collaborations harnessing the expertise, resources, knowledge and data of all stakeholders involved in rare disease, including Industry. To-date, however, ERN-Industry interactions have been largely limited, for a range of reasons (concerning barriers both tangible and perceived). This Position Statement analyses these barriers, and explains how Together4RD is seeking to move the needle here, by learning from case studies, exploring frameworks for collaboration, and launching pilots to explore how best to plan and deliver multistakeholder interactions addressing real research needs. [ABSTRACT FROM AUTHOR]

Background We aimed to study the impact of obesity on urinary excretion of angiotensinogen (U-AGT) in prepubertal children, focusing on the duration of obesity and gender. Also, we aimed to evaluate whether plasma angiotensinogen (P-AGT) and hydrogen peroxide (H.sub.2O.sub.2) play a role in the putative association. Methods Cross-sectional evaluation of 305 children aged 8-9 years (160 normal weight, 86 overweight, and 59 obese). Anthropometric measurements and 24-h ambulatory blood pressure monitoring were performed. Angiotensinogen (AGT) was determined by a commercial enzyme-linked immunosorbent assay (ELISA) kit and H.sub.2O.sub.2 by a microplate fluorometric assay. Results U-AGT and P-AGT levels were similar across body mass index (BMI) groups and between sexes. However, boys who were overweight/obese since the age of 4 years presented lower levels of U-AGT compared with those of normal weight at the same age. In children who were overweight/obese since the age of 4, urinary H.sub.2O.sub.2 decreased with P-AGT. Conclusions A higher duration of obesity was associated with decreased U-AGT in boys, thus reflecting decreased intrarenal activity of the renin-angiotensin system. Also, children with a longer duration of obesity showed an inverse association between urinary H.sub.2O.sub.2 and P-AGT. Future studies should address whether these results reflect an early compensatory mechanism to limit obesity-triggered renal dysfunction., Author(s): Manuela Morato [sup.1] [sup.2] [sup.3] [sup.4] , Liane Correia-Costa [sup.5] [sup.6] [sup.7] , Teresa Sousa [sup.1] [sup.3] , Dina Cosme [sup.1] [sup.5] , Franz Schaefer [sup.8] , José Carlos [...]