Lumasiran shows continued promise in infants and young children with primary hyperoxaluria type 1
Lumasiran, a liver-directed RNA interference therapeutic, showed reductions in urinary and plasma oxalates in infants and young children with primary hyperoxaluria type 1 and was well-tolerated over 60 months of therapy, in the ILLUMINATE-B trial.Dr Wesley Hayes (Great Ormond Street Hospital for Children NHS, UK) reported long-term results from ILLUMINATE-B, a phase 3, open-label, single-arm study assessing lumasiran in children…
Ravulizumab improves long-term quality-of-life in atypical haemolytic uraemic syndrome
Continued long-term therapy with ravulizumab showed sustained quality-of-life improvements in both adults and paediatric patients with atypical haemolytic uraemic syndrome enrolled in two phase 3 trials.The analysis was based on data from two completed phase 3 trials in adult patients and paediatric patients with atypical haemolytic uraemic syndrome who were naïve to complement C5 inhibitors (NCT02949128, NCT03131219) [1]. In these…
Anti-PAPP-A antibody shows preclinical promise for autosomal dominant polycystic kidney disease
Pregnancy-associated plasma protein-A (PAPP-A) is involved in the development of autosomal dominant polycystic kidney disease. In preclinical mouse models, the anti-PAPP-A monoclonal antibody ABBV-CLS-628 was shown to reduce kidney volume and improve eGFR.Dr Georgios Koukos (Calico Life Sciences, CA, USA) presented data on ABBV-CLS-628 in 3 different mouse models, including a fast-progressing non-orthologous disease model, an inducible and fast-progressing disease…
Nefecon maintains kidney function regardless of baseline status over 24 months in IgA nephropathy
In the phase 3 NefIgArd trial, nefecon versus placebo showed improvement in eGFR and proteinuria in patients with IgA nephropathy, over 9 months of therapy and the subsequent 15 months of follow-up, regardless of baseline eGFR status. Prof. Jonathan Barratt (University of Leicester, UK) described a subanalysis of NefIgArd, a phase 3 trial assessing nefecon 16 mg/day versus placebo in…
Iptacopan improves mesenchymal and capillary glomerular C3 deposition in patients with C3 glomerulopathy
Following 6 months of treatment in the APPEAR-C3G trial, iptacopan versus placebo decreased complement C3 deposition in the glomeruli, a known factor associated with prognosis in patients with C3 glomerulopathy.APPEAR-C3G was a phase 3, randomised, double-blind trial in patients with C3 glomerulopathy with C3 levels <77 mg/dL, proteinuria ≥1.0 g/g, and eGFR ≥30 ml/min/1.73 m2 [1]. Treatment consisted of 6…
Sibeprenlimab is an emerging treatment option for IgA nephropathy
In patients with IgA nephropathy at risk of progression, sibeprenlimab versus placebo has shown a 50% reduction in urinary protein-creatinine ratio (uPCR) after 9 months of treatment, with similar safety, according to the VISIONARY trial.Prof. Vlado Perkovic (University of New South Wales, Australia) reported interim results from VISIONARY (NCT05248646), an ongoing, phase 3, multicentre, randomised, double-blind trial of sibeprenlimab, an…
Pegcetacoplan is treatment option for adolescents with C3G or IC-MPGN
The C3/C3b inhibitor pegcetacoplan has shown efficacy in reducing proteinuria and stabilising eGFR compared with placebo in the subgroup of adolescents with C3 glomerulopathy or primary (idiopathic) immune complex membranoproliferative glomerulonephritis (IC-MPGN) in the VALIANT trial, consistent with the overall results.Dr Antonio Mastrangelo (Policlinico of Milan, Italy) presented a post-hoc analysis of the double-blind, randomised, phase 3 VALIANT (NCT05067127) trial,…
Mineral receptor antagonists not beneficial for patients undergoing dialysis
According to a meta-analysis, mineral receptor antagonists (MRAs), specifically spironolactone and eplerenone, do not lead to cardiac death or reduce all-cause mortality but increase rates of hyperkalaemia and gynecomastia/breast pain in patients undergoing dialysis. The meta-analysis presented by Dr Lonnie Pyne (McMaster University, Canada) included a total of 19 randomised-controlled trials (9 not part of previous meta-analyses), including the large…
Spironolactone not useful in dialysis with cardiovascular risk
Spironolactone versus placebo did not provide benefit in terms of cardiovascular mortality or hospitalisation from heart failure, but increased severe hyperkalaemia in patients with cardiovascular risk undergoing dialysis, according to results of the ACHIEVE trial.Dr Michael Walsh (McMaster University, Canada) presented the phase 3 ACHIEVE trial (NCT03020303), which included eligible patients in an initial run-in period lasting ≥7 weeks, receiving…
Acute eGFR dip following empagliflozin initiation not associated with acute kidney injury
According to a large meta-analysis, empagliflozin versus placebo improves measures of acute kidney injury, which in turn are not associated with acute eGFR dip following treatment initiation in patients with type 2 diabetes, heart failure, and chronic kidney disease.The analysis presented by Dr Natalie Staplin (University of Oxford, UK) used participant-level data from 4 large trials of empagliflozin (across patients…
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