Treatment Patterns and Outcomes Among Patients with Lennox-Gastaut Syndrome from Real-World Clinical Practice Settings Across the USA, Europe, and Asia
Abstract number :
1.306
Submission category :
7. Anti-seizure Medications / 7E. Other
Year :
2023
Submission ID :
406
Source :
www.aesnet.org
Presentation date :
12/2/2023 12:00:00 AM
Published date :
Authors :
Presenting Author: Drishti Shah, PhD – Takeda Pharmaceutical Company, Ltd
J Scott Andrews, PharmD – Takeda Pharmaceutical Company, Ltd; Stevie Olsen, BSc – Adelphi Real World, Bollington, UK; Arturo Benitez, MD, MBA – Takeda Pharmaceutical Company, Ltd; Jeannine Roth, MSc Pharm – Takeda Pharmaceutical Company, Ltd; Hannah Connolly, MChem – Adelphi Real World, Bollington, UK; Vicente Villanueva, MD – Hospital Universitario y Politécnico La Fe, Valencia, Spain
Rationale:
Lennox-Gastaut syndrome (LGS) is highly treatment resistant with current options limited to generic antiseizure medications (ASMs) and a few specifically indicated for LGS. We assessed the real-world treatment patterns and burden, including reasons for treatment discontinuation, adverse events (AEs), and impact of AEs on quality of life (QoL) among patients with LGS.
Methods:
A retrospective analysis was conducted on data from the Adelphi LGS Disease Specific Programme (DSP TM) that uses real-world cross-sectional surveys. Neurologists/pediatric neurologists completed questionnaires for their next consecutive consulting patients with LGS. Information included patient demographics, clinical details, treatment history, AEs, and clinical and humanistic outcomes. Data were collected from July 2022 in France, Germany, Italy, Spain, UK, USA, China and Japan. Patients receiving orphan drugs specifically indicated (ODSI) for LGS were identified. Descriptive analyses were performed to describe treatment burden. Data from France and Japan were from an interim data cut (April 7, 2023); other data were final.
Results:
Physicians (n=224) provided data on 803 patients with LGS (Europe n=440; USA n=174; Asia n=189). Mean (SD) patient age was 14.7 (11.1) y and median age of onset was 3 years old. Patients were receiving a mean (SD; range) of 2.2 (1.3; 1–9) treatment regimens. The three most prescribed current treatments were valproate (55%), lamotrigine (27%) and clobazam (27%); 33% (n=269) were receiving LGS ODSI medications (15% cannabidiol; 19% rufinamide; 2% fenfluramine). For the most common seizure types, less than a third had well controlled tonic (27%), atypical absence (26%) and atonic (32%) seizures. Of patients who received > 1 treatment regimen (n=520), 76% had a new treatment added and 44% switched treatment. The most common reasons for discontinuation included not effective at reducing seizure frequency/severity (67%, 114/170), reduced efficacy with continued use (54%; 92), and AEs (eg, too many, too severe; 28%; 47). Similar discontinuation reasons were reported for LGS ODSIs. Rates of ≥ 1 AE on current regimen were similar overall (49%) and among those receiving LGS ODSI (45%; [mean, range: 2.6, 1–15 overall; 2.3, 1–15 LGS ODSI]), with the most reported being somnolence (24% overall; 23% LGS ODSI). AEs had at least some impact on QoL for most patients (overall: 84%; LGS ODSI: 80%) and a moderate to severe impact for over 1 in 5 (overall: 27%; LGS ODSI: 28%). Overall, 68% had been prescribed a rescue medication (74% ODSI subgroup) with diazepam the most common (28%). Additional data are shown in the tables.
Conclusions:
Patients with LGS experience high rates of treatment change due to lack of efficacy and AEs that impact QoL resulting in a substantial treatment burden. Currently available ASMs, including LGS ODSI medications, are limited by their AE profile and associated with inadequate seizure control, suggesting a need for new treatment options with a balanced risk benefit profile.
Funding:
Analysis funded by Takeda Pharmaceutical Company Ltd.
Anti-seizure Medications