Please use this identifier to cite or link to this item:
https://ahro.austin.org.au/austinjspui/handle/1/31005
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DC Field | Value | Language |
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dc.contributor.author | Knupp, Kelly G | - |
dc.contributor.author | Scheffer, Ingrid E | - |
dc.contributor.author | Ceulemans, Berten | - |
dc.contributor.author | Sullivan, Joseph | - |
dc.contributor.author | Nickels, Katherine C | - |
dc.contributor.author | Lagae, Lieven | - |
dc.contributor.author | Guerrini, Renzo | - |
dc.contributor.author | Zuberi, Sameer M | - |
dc.contributor.author | Nabbout, Rima | - |
dc.contributor.author | Riney, Kate | - |
dc.contributor.author | Agarwal, Anupam | - |
dc.contributor.author | Lock, Michael | - |
dc.contributor.author | Dai, David | - |
dc.contributor.author | Farfel, Gail M | - |
dc.contributor.author | Galer, Bradley S | - |
dc.contributor.author | Gammaitoni, Arnold R | - |
dc.contributor.author | Polega, Shikha | - |
dc.contributor.author | Davis, Ronald | - |
dc.contributor.author | Gil-Nagel, Antonio | - |
dc.date | 2022 | - |
dc.date.accessioned | 2022-10-21T04:39:32Z | - |
dc.date.available | 2022-10-21T04:39:32Z | - |
dc.date.issued | 2023 | - |
dc.identifier.citation | Epilepsia 2023; 64(1) | en |
dc.identifier.uri | https://ahro.austin.org.au/austinjspui/handle/1/31005 | - |
dc.description.abstract | To evaluate the long-term safety and effectiveness of fenfluramine in patients with Lennox-Gastaut syndrome (LGS). Eligible patients with LGS who completed a 14-week phase 3 randomized clinical trial enrolled in an open-label extension (OLE; NCT03355209). All patients were initially started on 0.2 mg/kg/day fenfluramine and, after 1 month, were titrated to effectiveness and tolerability, which were assessed at 3-month intervals. The protocol-specified treatment duration was 12 months, but COVID-19-related delays resulted in 142 patients completing their final visit after 12 months. As of 10/19/2020, 247 patients were enrolled in the OLE. Mean age was 14.3±7.6 years (79 [32%] adults) and median fenfluramine treatment duration was 364 days; 88.3% of patients received 2-4 concomitant antiseizure medications. Median percentage change in monthly drop seizure frequency was -28.6% over the entire OLE (n=241) and -50.5% at Month 15 (n=142) (P<0.0001); 75/241 patients (31.1%) experienced ≥50% reduction in drop seizure frequency. Median percentage change in non-drop seizure frequency was -45.9% (n=192; P=0.0038). Generalized tonic-clonic seizures (GTCS) and tonic seizures were most responsive to treatment, with median reductions over the entire OLE of 48.8% (P<0.0001; n=106) and 35.8% (P<0.0001; n=186), respectively. A total of 37.6% (95% CI: 31.4%, 44.1%; n=237) of investigators and 35.2% of caregivers (95% CI: 29.1%, 41.8%; n=230) rated patients as "Much Improved"/"Very Much Improved" on the Clinical Global Impression of Improvement (CGI-I) scale. The most frequent treatment-emergent adverse events were decreased appetite (16.2%) and fatigue (13.4%). No cases of valvular heart disease (VHD) or pulmonary arterial hypertension (PAH) were observed. Patients with LGS experienced sustained reductions in drop seizure frequency on fenfluramine treatment, with a particularly robust reduction in frequency of GTCS, the key risk factor for SUDEP. Fenfluramine was generally well tolerated; VHD or PAH was not observed long-term. Fenfluramine may provide an important long-term treatment option for LGS. | en |
dc.language.iso | eng | - |
dc.subject | Lennox-Gastaut syndrome | en |
dc.subject | developmental and epileptic encephalopathies | en |
dc.subject | fenfluramine | en |
dc.subject | long-term open-label extension | en |
dc.title | Fenfluramine provides clinically meaningful reduction in frequency of drop seizures in patients with Lennox-Gastaut syndrome: interim analysis of an open-label extension study. | en |
dc.type | Journal Article | en |
dc.identifier.journaltitle | Epilepsia | en |
dc.identifier.affiliation | University of Colorado, Children's Hospital Colorado, Aurora, CO, USA. | en |
dc.identifier.affiliation | School of Clinical Medicine, University of Queensland, St Lucia, QLD, Australia | en |
dc.identifier.affiliation | Neuroscience Unit, Queensland Children's Hospital, South Brisbane, QLD, Australia | en |
dc.identifier.affiliation | Austin Health | en |
dc.identifier.affiliation | Department of Paediatric Neurology, Antwerp University Hospital, Antwerp, Belgium. | en |
dc.identifier.affiliation | University of California San Francisco Weill Institute for Neurosciences, Benioff Children's Hospital, San Francisco, CA, USA.. | en |
dc.identifier.affiliation | Mayo Clinic, Department of Neurology, Rochester, MN, USA. | en |
dc.identifier.affiliation | Member of the European Reference Network EpiCARE; Department of Paediatric Neurology, University of Leuven, Leuven, Belgium.. | en |
dc.identifier.affiliation | Pediatric Neurology and Neurogenetics Unit, Anna Meyer Children's Hospital, University of Florence, Florence, Italy; IRCCS Fondazione Stella Maris, Pisa, Italy.. | en |
dc.identifier.affiliation | Paediatric Neurosciences Research Group, Royal Hospital for Children, Glasgow, United Kingdom.. | en |
dc.identifier.affiliation | Reference Centre for Rare Epilepsies, Hôpital Universitaire Necker-Enfants Malades, APHP, Member of EPICARE, Institut Imagine, Université Paris Cité, Paris, France.. | en |
dc.identifier.affiliation | Formerly Zogenix, Inc. (now a part of UCB), Emeryville, CA, USA.. | en |
dc.identifier.affiliation | Independent consultant, Zogenix, Inc. (now a part of UCB); Haiku, HI, USA.. | en |
dc.identifier.affiliation | Syneos Health, Morrisville, NC, USA.. | en |
dc.identifier.affiliation | Formerly Zogenix, Inc. (now a part of UCB), Emeryville, CA, USA.. | en |
dc.identifier.affiliation | Zogenix, Inc. (now a part of UCB), Emeryville, CA, USA.. | en |
dc.identifier.affiliation | Neurology and Epilepsy Research Center, Orlando, FL, USA.. | en |
dc.identifier.affiliation | Hospital Ruber Internacional, Madrid, Spain.. | en |
dc.identifier.doi | 10.1111/epi.17431 | en |
dc.type.content | Text | en |
dc.identifier.orcid | https://orcid.org/0000-0002-1967-0827 | en |
dc.identifier.orcid | https://orcid.org/0000-0002-2311-2174 | en |
dc.identifier.orcid | https://orcid.org/0000-0002-6579-3035 | en |
dc.identifier.orcid | https://orcid.org/0000-0002-7118-0139 | en |
dc.identifier.orcid | https://orcid.org/0000-0002-7272-7079 | en |
dc.identifier.orcid | https://orcid.org/0000-0001-5877-4074 | en |
dc.identifier.orcid | https://orcid.org/0000-0002-1122-3555 | en |
dc.identifier.orcid | https://orcid.org/0000-0002-4735-3327 | en |
dc.identifier.orcid | https://orcid.org/0000-0002-4775-2862 | en |
dc.identifier.pubmedid | 36196777 | - |
local.name.researcher | Scheffer, Ingrid E | |
item.languageiso639-1 | en | - |
item.cerifentitytype | Publications | - |
item.openairecristype | http://purl.org/coar/resource_type/c_18cf | - |
item.grantfulltext | none | - |
item.openairetype | Journal Article | - |
item.fulltext | No Fulltext | - |
crisitem.author.dept | Epilepsy Research Centre | - |
Appears in Collections: | Journal articles |
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