Authors :
Presenting Author: Simona Lattanzi, MD, PhD – Neurology Dept, Università Politecnica delle Marche, Ancona, Italy
Barbara Belmessieri, MD – Paediatric Neurology and Psychiatry Unit, Spedali Civili Children's Hospital, University of Brescia, Brescia, Italy
Francesca Bisulli, MD – Department of Biomedical and NeuroMotor Sciences, Alma Mater Studiorum, University of Bologna, Bologna, Italy
Paolo Bonanni, MD – Epilepsy and Neurophysiology Unit, IRCCS Eugenio Medea, Conegliano, Treviso, Italy
Antonietta Coppola, MD – Department of Neuroscience, Reproductive and Odontostomatological Sciences, Federico II University, Naples, Italy
Francesca Darra, MD – Unit of Child Neuropsychiatry, Department of Engineering for Innovation Medicine, University of Verona, Full Member of the ERN EpiCARE Verona, Verona, Italy
Giuseppe d'Orsi, MD – Neurology Unit, Epilepsy Centre, IRCCS Casa Sollievo della Sofferenza, San Giovanni Rotondo (FG), Italy
Fedele Dono, MD – Department of Neuroscience, Imaging and Clinical Science, "G. d'Annunzio" University of Chieti-Pescara, Chieti, Italy
Antonio Gambardella, MD – Department of Medical and Surgical Sciences, Institute of Neurology, Magna Græcia University, Catanzaro, Italy
Alfonso Giordano, MD – Neurology Unit, A.O.U “L. Vanvitelli”, Naples, Italy
Carlo Fusco, MD – Child Neurology and Psychiatry Unit, IRCCS “Arcispedale Santa Maria Nuova”, Reggio Emilia, Italy
Stefano Meletti, PhD, MD – UNIMORE
Rita Monni, MD – Child Neuropsychiatry Unit, ASST Mantova, Mantova, Italy
Amanda Papa, MD – Department of Child Neurology and Psychiatry, AOU Maggiore della Carità Novara, Novara, Italy
Marta Piccioli, MD – Neurology Unit, PO San Filippo Neri, ASL Roma 1, Rome, Italy
Eliana Parente, MD – Pediatric Neurology Unit and Epilepsy Center, Fatebenefratelli Hospital, ASST Fatebenefratelli Sacco, Milan, Italy
Eleonora Rosati, MD – Emergency Neurology, Careggi University Hospital, Florence, Italy
Cindy Tiseo, MD – Epilepsy center; Ospedale S.S. Filippo e Nicola, Avezzano (AQ), Italy
Giancarlo Di Gennaro, MD – Epilepsy Center, IRCCS Neuromed, Pozzilli, Isernia, Italy
Alfredo D'Aniello, MD – Epilepsy Center, IRCCS Neuromed, Pozzilli, Isernia, Italy
Rationale:
The aim of this study was to assess the real-world effectiveness and tolerability of fenfluramine (FFA) in adults with Lennox-Gastaut syndrome (LGS).
Methods:
We performed a retrospective chart review of adults (≥16 years) with LGS receiving FFA at 18 centres in Italy. Data were extracted from clinical charts up to 6 months after treatment.
Results:
Thirty-seven adults with LGS were identified. The median age was 29 (interquartile range, 19-41) years and 24 (64.9%) were males. Baseline characteristics of participants are summarized in Table 1.
The mean percentage reduction in frequency of drop-seizures was 48.1% at 3 months and 53.0% at 6 months after starting FFA treatment; a ≥50% reduction in frequency of drop seizures was observed in 51.9% of the participants at 3 and 6 months. The mean percentage reduction in frequency of non-drop seizures was 39.0% at 3 months and 37.7% at 6 months; a ≥50% reduction in frequency of non-drop seizures was reported by 39.4% and 36.4% of participants at 3 and 6 months.
Among subjects continuing treatment, the median FFA dose at 6 months was 0.35 mg/kg/die. The mean number of 3-month days free of drop seizures increased from 51.4 at baseline to 67.5 at 6-month follow-up. The mean longest interval between drop seizures increased from 28.0 at baseline to 43.7 days at 6 months.
There was a reduction in the mean number of prolonged seizures that required the use of rescue medications in the last 3 months from 1.2 before starting FFA to 0.6 at 6-month follow-up. Discontinuation of one concomitant antiseizure medication (ASM) occurred in 7/37 (18.9%) and discontinuation of two concomitant ASMs in 1/37 (2.7%) participants.
The 6-month retention rate was 86.5%; treatment discontinuation occurred in 5 subjects due to adverse events. Adverse events occurred in 32.4% of participants and the most common included somnolence and agitation/behavioural disorders.
Subjects rated as slightly improved, much improved, or very much improved for overall condition from baseline to 6 months as measured by the Physician Global Impression of Change scale and Caregiver Global Impression of Change were 73.0% and 78.4%, respectively; 45.9% of the participants were rated as “much improved” or “very much improved” (i.e., a clinically meaningful improvement) by both their physician and caregiver. Behavior was reported as improved in 6 (16.2%) adults.
Conclusions:
In adults with LGS treated according to everyday clinical practice, adjunctive FFA demonstrated effectiveness and favourable tolerability profile. A decrease in concomitant drug load and improvements in global functioning and behavior were reported when treatment with FFA was initiated in adulthood.
Funding: None