Introduction: The RNA interference (RNAi) medication lumasiran reduces hepatic oxalate production in primary hyperoxaluria type 1 (PH1). Data outside clinical trials are scarce.
Methods: We report on retrospectively and observationally obtained data in 33 patients with PH1 (20 with preserved kidney function, 13 on dialysis) treated with lumasiran for a median of 18 months.
Front Surg
May 2022
Introduction: Frameshift in medical management as well as in surgical thinking is putting the patient as a whole is the focus, rather than just the disease. To optimize the treatment of our pediatric transplant patients in our institution, we changed in 2013 the transplant program setting, treating, and operating all patients with pediatric transplant exclusively in a pediatric environment. The aim of this study was to analyze whether or not this change had an impact on patients safety, patient population, and patients and transplant outcome.
View Article and Find Full Text PDFObjective: To introduce and evaluate a modified version of the "zipper method"-a treatment strategy alternating intravenous immunoglobulin (IVIG) and plasma exchange (PLEX) first reported for 9 pediatric cases of Guillain-Barré syndrome in 2018-for treatment of severe immune-mediated neurologic disorders in children.
Methods: The modified zipper method comprised longer intervals between PLEX-IVIG cycles (48hours instead of 24hours), more cycles (7-10 instead of 5), a consistent plasma volume exchange (instead of the original multistep approach), and variable infusion times for IVIGs (4-8hours). The modified zipper method was applied as an individual treatment approach once standard therapy failed.
Background: Peritoneal dialysis (PD) is the preferred dialysis modality for paediatric patients with end-stage kidney disease. Frequently, malnutrition is encountered. Percutaneous endoscopic gastrostomy (PEG) is the preferred mode of feeding because of its minimal invasive mode of placement and easy handling in daily life.
View Article and Find Full Text PDFBackground: Primary hyperoxaluria type 1 (PH1) is a multisystemic metabolic disorder caused by an excessive production of oxalate by the liver. The majority of patients presenting in early infancy have end-stage renal disease (ESRD). While awaiting the results of sRNAi trials, the current standard treatment is combined liver-kidney transplantation.
View Article and Find Full Text PDFBackground: Intravenous artesunate (ivA) is the standard treatment for severe malaria. Data systematically evaluating the use of ivA in pediatric patients outside malaria-endemic regions are limited. The aim of this case series was to summarize efficacy and safety of ivA for imported severe malaria in children in Germany.
View Article and Find Full Text PDFBackground: Thrombotic thrombocytopenic purpura (TTP) is an ultra-rare disorder in childhood and belongs to the microangiopathic hemolytic anemias (MAHA) and the thrombotic microangiopathies (TMA). In the acquired form, autoantibodies against ADAMTS13 inhibit cleaving of von Willebrand factor (vWF) multimers, thereby promoting their interaction with thrombocytes, causing TMA and MAHA. A recently introduced nanobody, caplacizumab, inhibits the binding of platelets to vWF.
View Article and Find Full Text PDFThe majority of multi-exon genes undergo alternative splicing to produce different mRNA transcripts and this may occur in a tissue-specific manner. Assessment of mRNA transcripts isolated from blood samples may sometimes be unhelpful in determining the affect on function of putative splice-site variants affecting kidney-specific mRNA transcripts. Here we present data demonstrating the power of using human urine-derived renal epithelial cells (hUREC) as a source of kidney RNA.
View Article and Find Full Text PDFBackground: Peritoneal dialysis (PD) catheter occlusion is a common complication with up to 36% of catheter obstructions described in the literature. We present a comparison of complications and outcome after implantation of PD catheters in a transplant surgical and a pediatric surgical department.
Methods: We retrospectively analyzed 154 PD catheters, which were implanted during 2009-2015 by transplant surgeons (TS, University Medical Center Hamburg-Eppendorf, Germany, n=85 catheters) and pediatric surgeons (PS, Charité University Medicine Berlin, Germany, n=69 catheters) in 122 children (median (range) age 3.
Background: The kidneys are essential for the metabolism of vitamin A (retinol) and its transport proteins retinol-binding protein 4 (RBP4) and transthyretin. Little is known about changes in serum concentration after living donor kidney transplantation (LDKT) as a consequence of unilateral nephrectomy; although an association of these parameters with the risk of cardiovascular diseases and insulin resistance has been suggested. Therefore we analyzed the concentration of retinol, RBP4, apoRBP4 and transthyretin in serum of 20 living-kidney donors and respective recipients at baseline as well as 6 weeks and 6 months after LDKT.
View Article and Find Full Text PDFEndoscopic subureteral injection of bulking agents has become a widespread treatment for vesicoureteral reflux (VUR) in children. Various biological and plastic materials have been introduced for this purpose with different success and complication rates. Evaluations of this method in previous studies have focused on the success rate of eliminating VUR, whereas complications have been less frequently reported in detail.
View Article and Find Full Text PDFGranulocyte/macrophage-colony stimulating factor (GM-CSF) and transforming growth factor (TGF)beta1 induce arteriogenesis in a nonischemic model of femoral artery ligation. Moreover, clinical trials demonstrated an improved collateralization after injection of bone marrow cells. In the present study, the expression of arteriogenic factors in bone marrow-derived macrophages (BMDM) was measured to verify the potential of these cells to influence collateral artery growth.
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