Intravenous ganaxolone in pediatric super-refractory status epilepticus: A single hospital experience.

Autor: Singh RK; Department of Pediatrics, Atrium Health/Levine Children's Pediatric Neurology Care, 100 Medical Park Drive Suite 310 E, Concord, NC 28025, USA., Singh R; Department of Pediatrics, Atrium Health/Levine Children's Pediatric Neurology Care, 100 Medical Park Drive Suite 310 E, Concord, NC 28025, USA., Stewart A; Department of Pediatrics, Atrium Health/Levine Children's Pediatric Neurology Care, 100 Medical Park Drive Suite 310 E, Concord, NC 28025, USA., Van Poppel K; Department of Pediatrics, Atrium Health/Levine Children's Pediatric Neurology Care, 100 Medical Park Drive Suite 310 E, Concord, NC 28025, USA., Klinger S; Department of Pediatrics, Atrium Health/Levine Children's Pediatric Neurology Care, 100 Medical Park Drive Suite 310 E, Concord, NC 28025, USA., Hulihan J; Department of Pediatrics, Atrium Health/Levine Children's Pediatric Neurology Care, 100 Medical Park Drive Suite 310 E, Concord, NC 28025, USA., Van Heusen H; Department of Pediatrics, Atrium Health/Levine Children's Pediatric Neurology Care, 100 Medical Park Drive Suite 310 E, Concord, NC 28025, USA., Vaitkevicius H; Department of Pediatrics, Atrium Health/Levine Children's Pediatric Neurology Care, 100 Medical Park Drive Suite 310 E, Concord, NC 28025, USA., Gasior M; Department of Pediatrics, Atrium Health/Levine Children's Pediatric Neurology Care, 100 Medical Park Drive Suite 310 E, Concord, NC 28025, USA.
Jazyk: angličtina
Zdroj: Epilepsy & behavior reports [Epilepsy Behav Rep] 2022 Oct 06; Vol. 20, pp. 100567. Date of Electronic Publication: 2022 Oct 06 (Print Publication: 2022).
DOI: 10.1016/j.ebr.2022.100567
Abstrakt: Synaptic GABA A receptor (GABA A R) internalization contributes to the drug resistant nature of super-refractory status epilepticus (SRSE). Ganaxolone is a 3β-methylated synthetic analog of the endogenous neuroactive steroid, allopregnanolone, that has positive allosteric modulatory activity on synaptic and extrasynaptic GABA A receptors. Ganaxolone is currently in clinical trials to treat rare pediatric seizure disorders and established and refractory SE. Two pediatric patients with SRSE (age 17 and age 7) were treated under emergency investigational new drug (E-IND) applications with intravenous (IV) ganaxolone administered as an initial bolus and a maintenance infusion for up to 4.5 days with intermittent IV boluses as-needed followed by taper on day 5 and transitioned to chronic treatment using ganaxolone suspension. Adjunctive ganaxolone was effective in terminating SRSE in both patients, safely permitting IV anesthetics to be weaned. Seizure control has been maintained after transitioning to enteric ganaxolone. Further investigation of ganaxolone as a safe and effective treatment for SRSE is warranted.
Competing Interests: The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Rani K. Singh – Reports research funding from the Pediatric Epilepsy Research Foundation, has served on the advisory board for Zogenix, Prasco, and AKPharma, and is a member of the executive board of the Western Region of the Epilepsy foundation of North Carolina. Joseph Hulihan, Heather van Heusen, Henrikas Vaitkevicius, Maciej Gasior are all employees of Marinus Pharmaceuticals, Inc.
(© 2022 The Authors.)
Databáze: MEDLINE
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