Preparing for kidney replacement therapy in pediatric advanced CKD: a review of literature and defining a multi-disciplinary clinical approach to patient-caregiver education.

Pediatric patients with progressive chronic kidney disease (CKD) approaching kidney replacement therapy (KRT) make up a small population but carry significant morbidity and mortality. Patients and caregivers require comprehensive kidney failure education to ensure a smooth start to KRT. Choice of KRT modality can be influenced by medical comorbidities, patient/caregiver comprehension, and comfort with a particular modality, social and economic factors, and/or implicit bias of the health care team. As KRT modality can influence morbidity, mortality, and quality of life, we created a pediatric advanced CKD clinic to provide comprehensive KRT education and to promote informed decision-making for our advanced CKD patients and their caregivers.

Utilization Pattern for Eculizumab Among Children With Hemolytic Uremic Syndrome.

Background: Hemolytic uremic syndrome (HUS) is a complex disease with multi-organ involvement. Eculizumab therapy is recommended for treatment of complement mediated hemolytic uremic syndrome (cHUS). However, there are few studies evaluating eculizumab therapy among children with HUS. The primary objectives of the study were to describe and identify factors associated with eculizumab therapy in children with HUS. Design/Methods: This large, retrospective, multi-center, cohort study used the Pediatric Health Information System (PHIS) database to identify the index HUS-related hospitalization among patients ≤18 years of age from September 23, 2011 (Food and Drug Administration approval date of eculizumab) through December 31, 2018. Multivariate analysis was used to identify independent factors associated with eculizumab therapy during or after the index hospitalization. Results: Among 1,885 children included in the study, eculizumab therapy was noted in 167 children with a median age of 3.99 years (SD ± 4.7 years). Eculizumab therapy was administered early (within the first 7 days of hospitalization) among 65% of children who received the drug. Mortality during the index hospitalization among children with eculizumab therapy was 4.2 vs. 3.0% without eculizumab therapy (p = 0.309). Clinical factors independently associated with eculizumab therapy were encephalopathy [odds ratio (OR) = 3.09; p ≤ 0.001], seizure disorder (OR = 2.37; p = 0.006), and cardiac involvement (OR = 6.36, p < 0.001). Conclusion(s): Only 8.9% of children received eculizumab therapy. Children who presented with neurological and cardiac involvement with severe disease were more likely to receive eculizumab therapy, and children who received therapy received it early during their index hospitalization. Further prospective studies are suggested to confirm these findings.

Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series.

Spinal muscular atrophy is treated with onasemnogene abeparvovec, which replaces the missing survival motor neuron 1 gene via an adeno-associated virus vector. As of July 1, 2020, we had identified 3 infants who developed thrombotic microangiopathy following onasemnogene abeparvovec. Early recognition and treatment of drug-induced thrombotic microangiopathy may lessen mortality and morbidity.

Albuminuria in Pediatric Neurogenic Bladder: Identifying an Earlier Marker of Renal Disease.

OBJECTIVE: To investigate the role of albuminuria as a clinical marker of early renal disease in children with neurogenic bladder (NGB) in association with commonly used predictors of renal risk. METHODS: Catheterized urine was obtained from 40 patients with NGB at a tertiary pediatric hospital. Albumin-to-creatinine ratio (ACR) was analyzed for associations with estimated glomerular filtration rate, vesicoureteral reflux, hydronephrosis, bladder dynamics, and renal scarring. RESULTS: About 32% (13/40) of NGB patients had elevated ACR (≥30 mg/g. Elevated ACR was associated with Caucasian race, clean intermittent catheterization, hydronephrosis, and vesicoureteral reflux on univariate analysis. In multivariable analysis, presence of vesicoureteral reflux and use of anticholinergic medication were significant predictors of ACR elevation. CONCLUSION: Albuminuria is an established clinical predictor of renal disease and risk for progression to renal failure. The presence of albuminuria in NGB patients with urinary tract abnormalities suggests these patients may be at increased risk for progressive renal disease. This supports the clinical utility of adding ACR to the evaluation of renal risk in pediatric NGB.

Urinary Tract Infections.

Urinary tract infection (UTI) is the second most common bacterial infection in children and is considered a public health threat given the mounting rates of antibiotic-resistance among uropathogens. This article highlights recent encouraging developments in UTI research. Further work is necessary to translate the discoveries into accessible, cost-effective technologies that will aid clinicians in real-time decision-making.