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Ann Clin Transl Neurol. 2017 Apr 26;4(6):411-414. doi: 10.1002/acn3.408. eCollection 2017 Jun.

First-in-man allopregnanolone use in super-refractory status epilepticus.

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Department of Neurology Brigham and Women's Hospital Boston Massachusetts.
Harvard Medical School Boston Massachusetts.
Department of Neurology Duke University Medical Center and Veterans Affairs Medical Center Durham North Carolina.
Department of Neurology Massachusetts General Hospital Boston Massachusetts.
Clarus Ventures Cambridge Massachusetts.
Departments of Neurology and Pharmacology School of Medicine University of California Davis, Sacramento California.


Super-refractory status epilepticus (SRSE) is associated with high morbidity and mortality. Treatment of SRSE is complicated by progressive cortical hyperexcitability believed to result in part from synaptic GABA receptor internalization and desensitization. Allopregnanolone, a neurosteroid that positively modulates synaptic and extrasynaptic GABAA receptors, has been proposed as a novel treatment. We describe the first two patients with SRSE who were each successfully treated with a 120-h continuous infusion of allopregnanolone. Both patients recovered from prolonged SRSE with good cognitive outcomes.

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