Long-Term Use of Stiripentol in Japanese Patients with Dravet Syndrome: Data Collected in 409 Patients in Real-World Setting
Abstract number :
2.225
Submission category :
7. Anti-seizure Medications / 7C. Cohort Studies
Year :
2021
Submission ID :
1826153
Source :
www.aesnet.org
Presentation date :
12/5/2021 12:00:00 PM
Published date :
Nov 22, 2021, 06:52 AM
Authors :
Miyuki Yamada, BS - Meiji Seika Pharma Co.,Ltd.; Yushi Inoue - Department of Clinical Research, National Epilepsy Center, NHO Shizuoka Institute of Epilepsy and Neurological Disorders, Shizuoka, Japan; Yoko Ohtsuka - Department of Child Neurology, Asahigawaso Rehabilitation and Medical Center, Okayama, Japan; Daisuke Matsui - MEIJI; Katsuyoshi Suzuki - MEIJI
Rationale: To evaluate the long-term efficacy and tolerability of add-on stiripentol (STP) treatment in Japanese patients with Dravet syndrome by a post-marketing surveillance study since its approval in 2012.
Methods: Updated interim analysis for up to 104 weeks of the prospective observational study in Japanese patients who received STP from November 2012 to July 2019. Patients administered STP for the first time after its approval were defined as “new patients,” and those who continued to take STP after domestic clinical studies were defined as “continuous-use patients.” Physicians recorded the safety, effectiveness and overall improvement of all patients taking STP. Effectiveness was evaluated by the responder rate which is defined as the proportion of patients who experienced a decrease of ≥50% (50% responder rate) and ≥75% (75% responder rate) in the number of seizures compared with the 4 weeks before starting STP. Overall improvement was rated by physicians on a 5-point scale based on the comprehensive assessment of the patient’s condition after STP treatment.
Results: Among the 594 patients registered in the central database, physicians recorded data from 411 patients including 376 “new patients” with a median age of 7 years [0.5–50] at the time of STP initiation. Among them, 95 patients (25.3%) started STP early in life, before the age of 2 years. STP was continuously administered for 104 weeks in 74.4 % (305/410 patients) and the 50% responder rate of convulsives seizures in “new patients” was 42.8% (110/257) and 75% responder rate 35.0% (90/257) after 101-104 weeks of STP administration. The overall Improvement with STP is marked or moderate in 45.3% (160/353) and notably in 54.4% (50/92) of patients ≤ 2 years old. The overall incidence of adverse drug reactions (ADRs) was 70.0% (263/376) for “new patients” and 44.1% (15/34) during the surveillance period for “continuous-use patients.” No marked difference regarding ADRs compared with previous reports as well as no new safety concerns related to STP have been noticed.
Conclusions: This study confirms the long-term efficacy and safety of STP as add-on therapy in patients with Dravet syndrome from 6 months to adulthood. Study in real world settings with high continuation rate suggests that STP is greatly accepted as add-on treatment in Japanese patients with Dravet syndrome.
Funding: Please list any funding that was received in support of this abstract.: MEIJI.
Anti-seizure Medications