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

被引：7

作者：

Singh, Rani K. ^{[1
]}

Singh, Rajdeep ^{[1
]}

Stewart, Alejandra ^{[1
]}

Van Poppel, Katherine ^{[1
]}

Klinger, Sarah ^{[1
]}

Hulihan, Joseph ^{[1
]}

Van Heusen, Heather ^{[1
]}

Vaitkevicius, Henrikas ^{[1
]}

Gasior, Maciej ^{[1
]}

机构：

[1] Dept Pediat, Atrium Hlth Levine Childrens Pediat Neurol Care, 100 Med Pk Dr Suite 310 E, Concord, NC 28025 USA

来源：

EPILEPSY & BEHAVIOR REPORTS | 2022年 / 20卷

关键词：

Status epilepticus; Super-refractory status epilepticus; Pediatric; Neurosteroids; CONVULSIVE STATUS EPILEPTICUS; ADJUNCTIVE THERAPY; GABA(A) RECEPTORS; CHILDREN; TRAFFICKING; FREQUENCY; EFFICACY;

D O I：

10.1016/j.ebr.2022.100567

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Synaptic GAB(A)A receptor (GABAAR) internalization contributes to the drug resistant nature of super-refractory status epilepticus (SRSE). Ganaxolone is a 3 beta-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. (c) 2022 The Authors. Published by Elsevier Inc.

引用

页数：7

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