Atypical hemolytic uremic syndrome (aHUS) is a chronic life threatening condition that arises from genetic abnormalities resulting in uncontrolled complement amplifying activity. The introduction of eculizumab, the humanized monoclonal antibody, has brought about a paradigm shift in the management of aHUS. However, there are many knowledge gaps, diagnostic issues, access and cost issues, and patient or physician challenges associated with the use of this agent. Limited data on the natural history of aHUS along with the underlying genetic mutations make it difficult to predict the relapses and thereby raising concerns about the appropriate duration and monitoring of treatment. In this review, we discuss the safety and efficacy of eculizumab in patients with aHUS and its associated challenges.

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Renal cystic diseases are a clinically and genetically diverse group of renal diseases that can manifest in utero, infancy, or throughout childhood and adulthood. These diseases may be unilateral or bilateral with a single cyst or multiple cysts, or with increased echogenicity of the renal cortex without macroscopic cysts. Certain cystic renal diseases are life-threatening, with many developing chronic kidney and hepatic disease if not recognized early enough. Therefore, due to the prevalence and life-altering complications of this specific group of diseases in vulnerable populations, it is crucial for clinicians and healthcare providers to have an overall understanding of cystic diseases and how to pre-emptively detect and manage these conditions. In this review, we discuss in detail the epidemiology, genetics and pathophysiology, diagnosis, presentation, and management of numerous genetic and sporadic renal cystic diseases, such as polycystic kidney disease, multicystic dysplastic kidney, and calyceal diverticula, with an emphasis on prenatal care and pregnancy counseling.

Amiodarone may be considered for patients with junctional ectopic tachycardia refractory to treatment with sedation, analgesia, cooling, and electrolyte replacements. There are currently no published pediatric data regarding the hemodynamic effects of the newer amiodarone formulation, PM101, devoid of hypotensive agents used in the original amiodarone formulation. We performed a single-center, retrospective, descriptive study from January 2012 to December 2020 in a pediatric ICU. Thirty-three patients were included (22 male and 11 female) between the ages of 1.1 and 1,460 days who developed post-operative junctional ectopic tachycardia or other tachyarrhythmias requiring PM101. Data analysis was performed on hemodynamic parameters (mean arterial pressures and heart rate) and total PM101 (mg/kg) from hour 0 of amiodarone administration to hour 72. Adverse outcomes were defined as Vasoactive-Inotropic Score >20, patients requiring ECMO or CPR, or patient death. There was no statistically significant decrease in mean arterial pressures within the 6 hours of PM101 administration (p > 0.05), but there was a statistically significant therapeutic decrease in heart rate for resolution of tachyarrhythmia (p < 0.05). Patients received up to 25 mg/kg in an 8-hour time for rate control. Average rate control was achieved within 11.91 hours and average rhythm control within 62 hours. There were four adverse events around the time of PM101 administration, with three determined to not be associated with the medication. PM101 is safe and effective in the pediatric cardiac surgical population. Our study demonstrated that PM101 can be used in a more aggressive dosing regimen than previously reported in pediatric literature with the prior formulation.

nephrotic syndrome etiologies, such as membranous nephropathy (MN), focal segmental glomerulosclerosis (FSGS), minimal change disease (MCD), lupus nephritis (LN), IgA nephropathy (IgAN) and membranoproliferative glomerulonephritis (MPGN) has not been determined. This systematic review analyzed the published literature on ACTH therapy in various NS etiologies to determine its efficacy. Methods: A comprehensive search of MEDLINE, EMBASE, and Cochrane databases was conducted for articles through June 2019. Prospective and retrospective studies of randomized control trials, which studied synthetic or natural ACTH treatment in patients with known etiologies of NS, were included. Studies were excluded when they consisted of a single case report or didn't analyze the lone effect of ACTH in NS. Results: The initial search yielded a total of 348 papers and 21 papers were included. In 122 MN patients, there was an overall remission of 70% (85/122) and an overall remission of 43% (42/98) in FSGS patients. In other etiologies, there were overall remissions of 78% (11/14), 31% (5/16), 38% (8/21) and 62% (8/13) in MCD, LN, IgAN, and MPGN patients, respectively. Conclusions: ACTH showed benefits in proteinuria reduction across all etiologies of NS. However, more randomized controlled studies with larger population sets and longer follow-ups are imperative to establish causal benefits. New studies into its efficacy in children should be also be investigated.