Use of carglumic acid in valproate-induced hyperammonemia: 25 pediatric cases.

Autor: Palomino Pérez LM; Section of Gastroenterology and Nutrition Hospital Infantil Universitario Niño Jesús Madrid Spain., Martín-Rivada Á; Section of Pediatrics Hospital Infantil Universitario Niño Jesús Madrid Spain., Cañedo Villaroya E; Section of Gastroenterology and Nutrition Hospital Infantil Universitario Niño Jesús Madrid Spain., García-Peñas JJ; Section of Neurology Hospital Infantil Universitario Niño Jesús Madrid Spain., Cuervas-Mons Vendrell M; Section of Pharmacy Hospital Infantil Universitario Niño Jesús Madrid Spain., Pedrón-Giner C; Section of Gastroenterology and Nutrition Hospital Infantil Universitario Niño Jesús Madrid Spain.
Jazyk: angličtina
Zdroj: JIMD reports [JIMD Rep] 2020 Jun 11; Vol. 55 (1), pp. 3-11. Date of Electronic Publication: 2020 Jun 11 (Print Publication: 2020).
DOI: 10.1002/jmd2.12131
Abstrakt: Hyperammonemic encephalopathy is a rare but potentially dangerous complication of the antiepileptic drug (AED) sodium valproate (VPA). We report a retrospective study of 25 pediatric patients, (15 females [60%]; age: 7.6 ± 4.9 years), with different underlying disorders, who suffered from hyperammonemia due to VPA and who were treated with carglumic acid (CA). The duration of treatment with VPA was 15 ± 1 month, with a dose of 40 ± 16.6 mg/kg/d. VPA blood levels were 75.5 ± 60 mg/L with seven patients being overdosed (>100 mg/L). Twenty-three patients received concomitant treatment with other AEDs. The initial dose of CA was 100 mg/kg. Subsequently, CA doses of 25 mg/kg were given to 22 patients every 6 hours (average treatment length 2.17 ± 1.1 days) until ammonemia was normalized. In nine patients, CA was used in combination with other drugs to treat hyperammonemia. In all cases, blood ammonia levels were brought under control and symptoms of hyperammonemia resolved. Two hours after CA administration, the average reduction in ammonium levels was 53 ± 29 and 88.6 ± 47.5 μmol/L at 24 hours, resulting in a statistically significant decrease when compared to pretreatment levels. There were no statistically significant differences between sexes, in the presence or not of cognitive impairment or previous carnitine treatment. There were no statistically significant differences when comparing treatment with CA plus ammonia scavengers vs CA alone. In 17 patients (68%) VPA was discontinued and 62% of the patients who maintained treatment had recurrent episodes of hyperammonemia.
Competing Interests: The authors declare no conflicts of interest.
(© 2020 The Authors. Journal of Inherited Metabolic Disease published by John Wiley & Sons Ltd on behalf of SSIEM.)
Databáze: MEDLINE
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