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Background Primary hyperoxaluria type 1 (PH1) is a rare genetic disease that causes progressive kidney damage and systemic oxalosis due to hepatic overproduction of oxalate. Lumasiran demonstrated efficacy and safety in the 6-month primary analysis period of the phase 3, multinational, open-label, single-arm ILLUMINATE-B study of infants and children Methods Of the 18 patients enrolled in the 6-month primary analysis period, all entered the EP and completed ≥ 6 additional months of lumasiran treatment (median (range) duration of total exposure, 17.8 (12.7–20.5) months). Results Lumasiran treatment was previously reported to reduce spot urinary oxalate:creatinine ratio by 72% at month 6, which was maintained at 72% at month 12; mean month 12 reductions in prespecified weight subgroups were 89%, 68%, and 71% for patients weighing Conclusions Lumasiran treatment provided sustained reductions in urinary and plasma oxalate through month 12 across all weight subgroups, with an acceptable safety profile, in infants and young children with PH1. Graphical abstract A higher resolution version of the Graphical abstract is available as Supplementary information

Background: Children with chronic kidney disease (CKD) generally have worse educational and psychosocial outcomes compared with their healthy peers. This can impair their ability to manage their treatment, which in turn can have long-term health consequences through to adulthood. We attempted to capture the experiences of children with CKD and to describe the perspectives of their parents and caregivers on access to educational and psychosocial support. Methods: Children with CKD (n = 34) and their caregivers (n = 62) were sampled via focus groups from pediatric hospitals in Australia, Canada, and the USA. Sixteen focus groups were convened and the transcripts were analyzed thematically. Results: We identified four themes: disruption to self-esteem and identity (emotional turmoil of adolescence, wrestling with the sick self, powerlessness to alleviate child’s suffering, balancing normality and protection); disadvantaged by lack of empathy and acceptance (alienated by ignorance, bearing the burden alone); a hidden and inaccessible support system (excluded from formal psychological support, falling behind due to being denied special considerations); and building resilience (finding partners in the journey, moving towards acceptance of the illness, re-establishing childhood). Conclusions: Children with CKD and their caregivers encountered many barriers in accessing psychosocial and educational support and felt extremely disempowered and isolated as a consequence. Improved availability and access to psychosocial and educational interventions are needed to improve the wellbeing and educational advancement of children with CKD. Graphical Abstract: A higher resolution version of the Graphical abstract is available as Supplementary information. [Figure not available: see fulltext.]

Idiopathic membranous nephropathy is an uncommon cause of nephrotic syndrome in children and can present treatment challenges. The current treatment options of steroids and cyclophosphamide, cyclosporine, or mycophenolate require prolonged treatment durations and the associated side effects may result in nonadherence in children, especially in adolescents. We report two adolescent patients with idiopathic membranous nephropathy with nephrotic range proteinuria and elevated anti-phospholipase A2 receptor levels who did not achieve remission with steroids and were later treated with rituximab. Both patients received two doses of rituximab and responded with remission. In addition, anti-PLA2R antibody levels normalized and/or significantly improved. Rituximab seems to be a safe and effective treatment option in children with idiopathic membranous nephropathy due to anti-PLA2R. Further studies are needed to evaluate this effectiveness.

Achieving dry weight during hemodialysis (HD) while minimizing symptoms is critical for optimizing patient outcome by preventing chronic fluid overload, hypertension, and cardiomyopathy. Dry weight changes frequently in children because of growth and development and waxing and waning of appetite. We have previously shown non-invasive hematocrit monitoring (NIVM) helps to decrease intradialytic symptoms, while still achieving target dry weights in children receiving chronic HD. In the current study, we prospectively evaluated an NIVM-guided ultrafiltration (UF) management algorithm to determine target dry weight in nine pediatric patients (mean age 16.6+/-2.8 years, mean weight 41.6+/-11.1 kg). Use of NIVM could potentially lead to overly aggressive UF with increased interdialytic symptoms, post treatment thirst, and interdialytic weight gain (IDWG). To evaluate the effectiveness of our NIVM UF algorithm, we studied the effect of three different NIVM-guided UF models (100%, 90%, and 80% UF models) on intradialytic and interdialytic symptoms, pre-/post-treatment blood pressure (BP), and percentage IDWG. To assess interdialytic symptoms, patients completed two questionnaires, one for each day between treatments. No statistically significant difference was seen between the three UF models with respect to intradialytic or interdialytic symptoms, pre-/post-HD BP, or percentage IDWG. Only one of nine patients received non-ACEI chronic antihypertensive medication, yet all patients had pre- and post-HD BP95th percentile for age and height. The current study suggests that routine determination of target dry weight using NIVM and aiming for 100% UF helps to achieve the target dry weight, reduces both the risk of chronic fluid overload and the need for antihypertensive medication, and does not lead to increased intra- or interdialytic symptomatology in pediatric patients treated with chronic HD.

Acute renal failure (ARF) with fluid overload (FO) occurs often in stem cell transplant (SCT) recipients. We have previously demonstrated that an increased percentage of FO prior to the initiation of continuous renal replacement therapy (CRRT) is associated with mortality in children with ARF. Based on these data, we devised a protocol for the prevention of FO in SCT patients with ARF. SCT patients with ARF and 5% FO were started on furosemide and low-dose dopamine. To allow for nutrition, medication, and blood product administration, RRT was initiated for patients withor =10% FO. There were 272 patients who received allogeneic SCT from 1999 to 2002. Of these, medical records of 26 SCT patients with a first episode of oliguric ARF were reviewed. The mean patient age was 13+/-5 years (range 2-23.5 years). Mean days to ARF after SCT were 28+/-29 days (range 2-90 days). Of the 26 patients, 11 (42%) survived an initial ARF episode. All 11 survivors either maintained10% FO during their course or re-attained10% FO with RRT treatment. Of the 15 non-survivors, 6 had10% FO at the time of death. Of 14 patients who received RRT, 4 (29%) survived. Mechanical ventilation and pediatric risk of mortality scoreor =10 at the time of admission to the intensive care unit were associated with lower survival ( P0.05). The use of one or more pressors, the presence of graft-versus-host disease, and septic shock were not correlated with survival. Our data demonstrate that maintenance of euvolemia (10% FO) is critical but not sufficient for survival in SCT patients with ARF, as all non-euvolemic patients died. We suggest that aggressive use of diuretics and early initiation of RRT to prevent worsening of FO may improve the survival of SCT patients.