Rationale: Stiripentol (STP) was approved in August 2018 by the US FDA and is currently indicated “
for the treatment of seizures associated with Dravet syndrome in patients taking clobazam who are 6 months of age and older and weighing 7 kg or more”. The STIRUS study was designed to collect data in patients that have been prescribed STP since the
US marketing authorization.
Methods: STIRUS is a US retrospective, non-interventional, observational, multicentre chart review study conducted in Dravet Syndrome patients who initiated STP after FDA approval (08/21/2018) and were maintained on STP for a minimum of 3 months. Data regarding seizure types and frequency, status epilepticus, previous and current treatments, and the clinical status of patients during the 3 months prior to STP initiation, the first 3 months on STP and the last 3 months on STP (irrespective of STP discontinuation) were collected. The data collected was analysed to determine treatment patterns of STP use in the US following marketing authorization, the efficacy of STP treatment on seizure frequency, and the impact of STP on Dravet Syndrome-associated comorbidities and patient and caregiver quality of life after initiation of STP.
Results: 10 US sites were recruited to participate in the STIRUS study, and 100 STP patients were included in the retrospective review. For each patient, relevant data were extracted from existing medical records by the study physician or a delegate. Interim analysis indicates that, in accordance with the European recommendation of gradual dosage escalation, STP is initiated at around 10 mg/kg/day and then increased to the maximum dosage of 35 mg/kg/day, which is nevertheless lower than the 50 mg/kg/day dosage indicated in the US labelling. Once the study is completely recruited, the prespecified efficacy and safety criteria will be calculated. Analysis of STP utilization with other antiseizures medications, including fenfluramine and cannabidiol, will also be conducted.
Conclusions: The STIRUS study will provide real world evidence of post-marketing utilization of STP utilization in the US. In addition, this study will provide continued evidence of the management of Dravet Syndrome patients for intervals of greater than 10 years; including those US patients treated with STP before and after US marketing authorization.
Funding: This study was sponsored by Biocodex