司替戊醇有哪些不良反应？

On August 20, 2018, the new anticonvulsant drug (stiripentol) developed by Biocodex Sa for the treatment of epilepsy was approved by the US FDA for marketing. Its trade name is Diacomit. This medication is used to treat seizures with Dravet syndrome in people 2 years of age or older who are taking the antiepileptic drug clobazam. This drug is a capsule or oral suspension powder. The recommended daily dosage is 50 mg·kg-1, administered in two or three times. So, what are the adverse reactions of stiripentol?

What are the adverse reactions of stiripentol?

The main ones are somnolence, decreased appetite and weight, neutropenia and thrombocytopenia, withdrawal symptoms, risk for patients with phenylketonuria, suicidal behavior and ideation.

Adverse reactions in clinical trials of stiripentol

Because clinical trials are conducted under a variety of different conditions, the incidence of adverse reactions observed in the clinical trials of one drug cannot be directly compared with the incidence in the clinical trials of another drug, nor does it reflect the incidence observed in practice.

During the development of DIACOMIT for the treatment of seizures associated with Dravet syndrome, DIACOMIT was administered to 55 healthy male volunteers and 438 patients with Dravet syndrome, including 310 patients who received treatment for 12 months or more. Exposure conditions and duration varied widely and included single- and multiple-dose clinical pharmacology studies in healthy male volunteers, 2 randomized, double-blind, placebo-controlled, 12-week studies (Study 1 and Study 2) in patients with Dravet syndrome, and an open-label long-term study.

In Studies 1 and 2, 33 patients received DIACOMIT and 31 patients received placebo, and the treatment duration was 8 weeks. Adverse effects from these trials are listed below. About 53% of patients are female, and the average age is 9.2 years. Clobazam and valproate were used in all patients.

Adverse reactions led to discontinuation of DIACOMIT treatment in 2 patients: 1 patient experienced status epilepticus as an adverse reaction; and the 2nd patient experienced drowsiness, impaired balance, and salivation.

The most common adverse reactions (occurring in at least 10% of DIACOMIT-treated patients and more commonly than placebo) included somnolence (67%), decreased appetite (45%), agitation (27%), ataxia (27%), weight loss (27%), hypotonia (24%), nausea (15%), tremor (15%), dysarthria (12%), and insomnia (12%).

Recommended related articles: