{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,28]],"date-time":"2026-01-28T08:41:27Z","timestamp":1769589687107,"version":"3.49.0"},"reference-count":12,"publisher":"Springer Science and Business Media LLC","issue":"9","license":[{"start":{"date-parts":[[2020,5,16]],"date-time":"2020-05-16T00:00:00Z","timestamp":1589587200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2020,5,16]],"date-time":"2020-05-16T00:00:00Z","timestamp":1589587200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"name":"Charit\u00e9"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Pediatr Nephrol"],"published-print":{"date-parts":[[2020,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec>\n<jats:title>Background<\/jats:title>\n<jats:p>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. Recently, Stiripentol has been reported as a promising drug in the treatment of primary hyperoxaluria by reducing urinary oxalate (U<jats:sub>Ox<\/jats:sub>). Stiripentol is an anti-convulsive drug used in the treatment of children suffering from Dravet syndrome. It causes blockage of the last step in oxalate production by inhibition of hepatic lactate dehydrogenase 5 (LDH5).<\/jats:p>\n<\/jats:sec><jats:sec>\n<jats:title>Case<\/jats:title>\n<jats:p>We administered Stiripentol as compassionate use in an anuric infant with dialysis-dependent PH1 over a period of 4\u00a0months. Although achieving plasma concentrations of Stiripentol that were recently reported to lower U<jats:sub>Ox<\/jats:sub> excretion, we did not observe significant reduction to plasma oxalate concentrations (P<jats:sub>Ox<\/jats:sub>).<\/jats:p>\n<\/jats:sec><jats:sec>\n<jats:title>Conclusion<\/jats:title>\n<jats:p>We conclude that Stiripentol may not be useful to reduce P<jats:sub>Ox<\/jats:sub> in PH patients with advanced chronic kidney disease (CKD), but larger studies are needed to confirm this finding.<\/jats:p>\n<\/jats:sec>","DOI":"10.1007\/s00467-020-04585-5","type":"journal-article","created":{"date-parts":[[2020,5,16]],"date-time":"2020-05-16T09:03:17Z","timestamp":1589619797000},"page":"1787-1789","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":22,"title":["Stiripentol fails to lower plasma oxalate in a dialysis-dependent PH1 patient"],"prefix":"10.1007","volume":"35","author":[{"given":"Caroline","family":"Kempf","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anja","family":"Pfau","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Johannes","family":"Holle","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Karen","family":"M\u00fcller-Schl\u00fcter","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Philip","family":"Bufler","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Felix","family":"Knauf","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3478-1576","authenticated-orcid":false,"given":"Dominik","family":"M\u00fcller","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2020,5,16]]},"reference":[{"issue":"7","key":"4585_CR1","doi-asserted-by":"publisher","first-page":"649","DOI":"10.1056\/NEJMra1301564","volume":"369","author":"P Cochat","year":"2013","unstructured":"Cochat P, Rumsby G (2013) Primary hyperoxaluria. 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Kidney Int Rep 2(6):1050\u20131058","journal-title":"Kidney Int Rep"},{"key":"4585_CR11","doi-asserted-by":"publisher","first-page":"271","DOI":"10.2147\/DDDT.S154388","volume":"12","author":"A Reimers","year":"2018","unstructured":"Reimers A et al (2018) Reference ranges for antiepileptic drugs revisited: a practical approach to establish national guidelines. Drug Des Devel Ther 12:271\u2013280","journal-title":"Drug Des Devel Ther"},{"key":"4585_CR12","unstructured":"(2019) Biocodex, evaluation of the efficacy of stiripentol (Diacomit) as monotherapy for the treatment of primary hyperoxaluria. 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FK reports personal fees from Allena, Oxthera, Fresenius, Sanofi and grant support from Dicerna pharmaceuticals, outside the submitted work.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}}]}}