ABSTRACT
OBJECTIVE: To evaluate the effectiveness and tolerability of fenfluramine (FFA) in routine clinical practice treating real-world populations with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). METHODS: This was a retrospective analysis of patients with DS or LGS who initiated FFA treatment from 2018 to 2022 at a single center. Patient demographics, medical history, seizure characteristics, and treatment outcomes were collected from electronic medical records. Duration of FFA treatment, dosage regimens, seizure frequency, seizure severity, improvements in cognitive, social, and motor outcomes, and adverse events were extracted and analyzed. Effectiveness was assessed using ≥50 % sustained reduction in monthly seizure frequency vs baseline for ≥2 consecutive months at 12 months; seizure freedom was a secondary measure. RESULTS: Seizure frequency data was available for 56 of 68 patients included in the study. At 12 months, 50 patients (89.3 %) remained on FFA treatment; 58 % of these patients achieved a ≥50 % sustained response and 10 % experienced seizure freedom. Cognitive, motor, and social improvement were noted in 70.7 %, 36.2 %, and 27.6 % of patients, respectively. The total number of concomitant antiseizure medications was reduced by ≥1 in 29.4 % of patients. No differences were found between DS and LGS patients in these outcomes; age at start of FFA and age at the 12-month timepoint did not have an effect. At least one AE was experienced by 59.7% of patients; in 86.5% of the cases, AEs were plausibly related to treatment. While 70.3% of AEs were self-resolving and 81.8% of the remaining patients experienced mild AEs, 1 patient experienced a serious AE unrelated to FFA which resulted in the patient's death. There were no cases of pulmonary arterial hypertension or ventricular heart disease. SIGNIFICANCE: The effectiveness and tolerability of FFA treatment in patients with DS or LGS in this retrospective analysis of real-world data were consistent with those seen in randomized clinical trials.
Subject(s)
Epilepsies, Myoclonic , Lennox Gastaut Syndrome , Humans , Lennox Gastaut Syndrome/drug therapy , Anticonvulsants/adverse effects , Retrospective Studies , Fenfluramine/therapeutic use , Epilepsies, Myoclonic/drug therapy , SeizuresSubject(s)
Epilepsy, Frontal Lobe , Epilepsy , Humans , Seizures , Electroencephalography , Magnetic Resonance ImagingABSTRACT
PURPOSE: To determine the effectiveness and safety outcomes of cenobamate in a cohort of patients with highly refractory focal epilepsy in routine clinical practice. METHODS: Observational, retrospective, phase 4 study on subjects receiving cenobamate in three Spanish centers. The primary endpoint was the retention rate at the last follow-up. The main secondary endpoints were the 50%-responder and seizure-free rates at three months and the last follow-up. Other secondary endpoints were Global Clinical Impressions-Improvement (CGI-I) scores and treatment-emergent adverse events (TEAEs). RESULTS: Fifty-one patients with highly refractory focal epilepsy with 24.7 years of disease evolution, ten previously tried ASM, and a 23.5% of previous epilepsy surgery were included. The retention rate at the last follow-up was 80.4%. The 50% responder rate in focal seizures at three months was 56.5% (median reduction per month 51%, 0-74.6; p < 0.0001) and in focal to bilateral tonic-clonic seizures was 63.6% (median reduction per month 89%, 0-100; p = 0.022). A total of 54.3% of subjects reported a reduction in the intensity of focal seizures, and 66% manifested clinically significant satisfaction. Cenobamate allowed a significant decrease in concomitant ASM, especially sodium channel blockers. TEAEs were reported in 43.1% of individuals, 85% of whom resolved or improved, with no new safety findings. CONCLUSION: In this analysis of patients with highly refractory focal epilepsy treated with cenobamate according to standard clinical practice, there was evidence of a high reduction in both seizure frequency and intensity, with a manageable safety profile.
Subject(s)
Anticonvulsants , Drug Resistant Epilepsy , Epilepsies, Partial , Humans , Anticonvulsants/adverse effects , Drug Resistant Epilepsy/drug therapy , Drug Therapy, Combination , Epilepsies, Partial/drug therapy , Retrospective Studies , Seizures/drug therapy , Treatment OutcomeSubject(s)
Headache Disorders , Herpesvirus 7, Human/pathogenicity , Lymphocytosis , Roseolovirus Infections , Adult , Electroencephalography , Headache Disorders/cerebrospinal fluid , Headache Disorders/diagnosis , Headache Disorders/etiology , Headache Disorders/physiopathology , Herpesvirus 7, Human/isolation & purification , Humans , Lymphocytosis/cerebrospinal fluid , Lymphocytosis/diagnosis , Lymphocytosis/etiology , Magnetic Resonance Imaging , Male , Roseolovirus Infections/cerebrospinal fluid , Roseolovirus Infections/complications , Roseolovirus Infections/diagnosisABSTRACT
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