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Verfasst von:Groothoff, Jaap [VerfasserIn]   i
 Sellier-Leclerc, Anne-Laure [VerfasserIn]   i
 Deesker, Lisa [VerfasserIn]   i
 Bacchetta, Justine [VerfasserIn]   i
 Schalk, Gesa [VerfasserIn]   i
 Tönshoff, Burkhard [VerfasserIn]   i
 Lipkin, Graham [VerfasserIn]   i
 Lemoine, Sandrine [VerfasserIn]   i
 Bowman, Thomas [VerfasserIn]   i
 Zhou, Jing [VerfasserIn]   i
 Hoppe, Bernd [VerfasserIn]   i
Titel:Nedosiran safety and efficacy in PH1
Titelzusatz:interim analysis of PHYOX3
Verf.angabe:Jaap Groothoff, Anne-Laure Sellier-Leclerc, Lisa Deesker, Justine Bacchetta, Gesa Schalk, Burkhard Tönshoff, Graham Lipkin, Sandrine Lemoine, Thomas Bowman, Jing Zhou and Bernd Hoppe
E-Jahr:2024
Jahr:May 2024
Umfang:10 S.
Illustrationen:Diagramme
Fussnoten:Gesehen am 22.01.2025
Titel Quelle:Enthalten in: Kidney international. Reports
Ort Quelle:Amsterdam : Elsevier, 2016
Jahr Quelle:2024
Band/Heft Quelle:9(2024), 5 vom: Mai, Seite 1387-1396
ISSN Quelle:2468-0249
Abstract:Introduction - Primary hyperoxaluria (PH) is a rare genetic disorder of hepatic glyoxylate metabolism. Nedosiran is an RNA interference (RNAi) therapeutic that the US Food and Drug Administration has approved for treatment of PH1. PHYOX3 is a trial evaluating monthly nedosiran in patients with PH. - Methods - In this PHYOX3 interim analysis, participants with PH1 who continued from a single-dose nedosiran trial (PHYOX1), with no previous kidney or liver transplantation, dialysis, or evidence of systemic oxalosis were eligible. The safety and efficacy of once-monthly nedosiran was assessed over 30 months. - Results - Thirteen participants completed PHYOX1 and continued into PHYOX3. At baseline, the mean (SD) and median (range) age was 24.2 (6.6) years and 23.0 (14-39) years, respectively; 53.8% were female and 61.5% were White. Mean estimated glomerular filtration rate (eGFR) remained stable (62-84.2 mL/min per 1.73 m2) to month 30. Mean 24-hour urinary oxalate (Uox) excretion showed a sustained reduction from baseline of ≥60% at every visit (months 2-30). From month 2, at least 10 of 13 (76.9%) participants achieved normal (<0.46 mmol/24h; upper limit of assay-normal [ULN]) or near-normal (≥0.46 to <0.60 mmol/24h; ≥ULN to <1.3 × ULN) 24-hour Uox excretion. All participants experienced ≥1 adverse event (AE), mostly mild or moderate in severity (primarily, injection site events). Three serious, not treatment-related AEs were reported; there were no deaths or study discontinuations due to AEs. - Conclusion - Nedosiran was well-tolerated in patients with PH1, and treatment resulted in a sustained, substantial reduction in Uox excretion for at least 30 months in this long-term study. No safety signals have been identified to date. The PHYOX3 study is ongoing.
DOI:doi:10.1016/j.ekir.2024.02.1439
URL:Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.

kostenfrei: Volltext: https://doi.org/10.1016/j.ekir.2024.02.1439
 kostenfrei: Volltext: https://www.sciencedirect.com/science/article/pii/S2468024924015298
 DOI: https://doi.org/10.1016/j.ekir.2024.02.1439
Datenträger:Online-Ressource
Sprache:eng
Sach-SW:glyoxylate metabolism
 nedosiran
 primary hyperoxaluria type 1
 RNAi therapy
 safety/efficacy
 urinary oxalate excretion
K10plus-PPN:1915385407
Verknüpfungen:→ Zeitschrift

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