Cargando…

Safety and efficacy of long-term use of sodium oxybate for narcolepsy with cataplexy in routine clinical practice

BACKGROUND: Sodium oxybate is licensed in Europe for the treatment of narcolepsy with cataplexy in adults. The aim of this study was to assess the efficacy and safety of sodium oxybate in clinical practice in patients with narcolepsy and cataplexy refractory to other treatments. MATERIALS AND METHOD...

Descripción completa

Detalles Bibliográficos
Autores principales: Drakatos, Panagis, Lykouras, Dimosthenis, D'Ancona, Grainne, Higgins, Sean, Gildeh, Nadia, Macavei, Raluca, Rosenzweig, Ivana, Steier, Joerg, Williams, Adrian J., Muza, Rexford, Kent, Brian D., Leschziner, Guy
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier Science 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5727622/
https://www.ncbi.nlm.nih.gov/pubmed/28619187
http://dx.doi.org/10.1016/j.sleep.2017.03.028
_version_ 1783285919950831616
author Drakatos, Panagis
Lykouras, Dimosthenis
D'Ancona, Grainne
Higgins, Sean
Gildeh, Nadia
Macavei, Raluca
Rosenzweig, Ivana
Steier, Joerg
Williams, Adrian J.
Muza, Rexford
Kent, Brian D.
Leschziner, Guy
author_facet Drakatos, Panagis
Lykouras, Dimosthenis
D'Ancona, Grainne
Higgins, Sean
Gildeh, Nadia
Macavei, Raluca
Rosenzweig, Ivana
Steier, Joerg
Williams, Adrian J.
Muza, Rexford
Kent, Brian D.
Leschziner, Guy
author_sort Drakatos, Panagis
collection PubMed
description BACKGROUND: Sodium oxybate is licensed in Europe for the treatment of narcolepsy with cataplexy in adults. The aim of this study was to assess the efficacy and safety of sodium oxybate in clinical practice in patients with narcolepsy and cataplexy refractory to other treatments. MATERIALS AND METHODS: This was a retrospective single centre study including patients with severe narcolepsy with cataplexy refractory to other treatments, who were initiated on sodium oxybate between 2009 and 2015. Patients were allowed to be on other stimulants or/and anti-cataplectic agents. Epworth sleepiness scale (ESS) and weekly cataplexy events were recorded. Side effects (SEs) were recorded at every follow-up visit. RESULTS: 90 patients were prescribed sodium oxybate, with a total of 3116 patient-months of drug exposure. ESS and weekly cataplexy events were significantly reduced by sodium oxybate for all patients (ΔESS = 4.3 ± 4.4 and Δcataplexy = 21.8 ± 18.5 events/week; p < 0.0001, respectively). The required maintenance dose could not be predicted based upon gender, body mass index, or clinical factors. 60% of patients were able to reduce or come off other medications. Half of the patients experienced at least one SE, and 26.6% had to stop treatment due to limiting SEs. Nausea, mood swings and enuresis were the most commonly reported SEs. SEs that led to drug discontinuation, particularly psychosis, were associated with increasing age and were observed early after the initiation of the drug. CONCLUSIONS: Sodium oxybate provides a good clinical efficacy and acceptable safety profile in routine clinical practice for the treatment of patients suffering from narcolepsy with cataplexy. A quarter of patients experience SEs requiring withdrawal of the drug with older patients being more vulnerable to the more serious SEs.
format Online
Article
Text
id pubmed-5727622
institution National Center for Biotechnology Information
language English
publishDate 2017
publisher Elsevier Science
record_format MEDLINE/PubMed
spelling pubmed-57276222017-12-22 Safety and efficacy of long-term use of sodium oxybate for narcolepsy with cataplexy in routine clinical practice Drakatos, Panagis Lykouras, Dimosthenis D'Ancona, Grainne Higgins, Sean Gildeh, Nadia Macavei, Raluca Rosenzweig, Ivana Steier, Joerg Williams, Adrian J. Muza, Rexford Kent, Brian D. Leschziner, Guy Sleep Med Article BACKGROUND: Sodium oxybate is licensed in Europe for the treatment of narcolepsy with cataplexy in adults. The aim of this study was to assess the efficacy and safety of sodium oxybate in clinical practice in patients with narcolepsy and cataplexy refractory to other treatments. MATERIALS AND METHODS: This was a retrospective single centre study including patients with severe narcolepsy with cataplexy refractory to other treatments, who were initiated on sodium oxybate between 2009 and 2015. Patients were allowed to be on other stimulants or/and anti-cataplectic agents. Epworth sleepiness scale (ESS) and weekly cataplexy events were recorded. Side effects (SEs) were recorded at every follow-up visit. RESULTS: 90 patients were prescribed sodium oxybate, with a total of 3116 patient-months of drug exposure. ESS and weekly cataplexy events were significantly reduced by sodium oxybate for all patients (ΔESS = 4.3 ± 4.4 and Δcataplexy = 21.8 ± 18.5 events/week; p < 0.0001, respectively). The required maintenance dose could not be predicted based upon gender, body mass index, or clinical factors. 60% of patients were able to reduce or come off other medications. Half of the patients experienced at least one SE, and 26.6% had to stop treatment due to limiting SEs. Nausea, mood swings and enuresis were the most commonly reported SEs. SEs that led to drug discontinuation, particularly psychosis, were associated with increasing age and were observed early after the initiation of the drug. CONCLUSIONS: Sodium oxybate provides a good clinical efficacy and acceptable safety profile in routine clinical practice for the treatment of patients suffering from narcolepsy with cataplexy. A quarter of patients experience SEs requiring withdrawal of the drug with older patients being more vulnerable to the more serious SEs. Elsevier Science 2017-07 /pmc/articles/PMC5727622/ /pubmed/28619187 http://dx.doi.org/10.1016/j.sleep.2017.03.028 Text en © 2017 The Authors http://creativecommons.org/licenses/by/4.0/ This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Article
Drakatos, Panagis
Lykouras, Dimosthenis
D'Ancona, Grainne
Higgins, Sean
Gildeh, Nadia
Macavei, Raluca
Rosenzweig, Ivana
Steier, Joerg
Williams, Adrian J.
Muza, Rexford
Kent, Brian D.
Leschziner, Guy
Safety and efficacy of long-term use of sodium oxybate for narcolepsy with cataplexy in routine clinical practice
title Safety and efficacy of long-term use of sodium oxybate for narcolepsy with cataplexy in routine clinical practice
title_full Safety and efficacy of long-term use of sodium oxybate for narcolepsy with cataplexy in routine clinical practice
title_fullStr Safety and efficacy of long-term use of sodium oxybate for narcolepsy with cataplexy in routine clinical practice
title_full_unstemmed Safety and efficacy of long-term use of sodium oxybate for narcolepsy with cataplexy in routine clinical practice
title_short Safety and efficacy of long-term use of sodium oxybate for narcolepsy with cataplexy in routine clinical practice
title_sort safety and efficacy of long-term use of sodium oxybate for narcolepsy with cataplexy in routine clinical practice
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5727622/
https://www.ncbi.nlm.nih.gov/pubmed/28619187
http://dx.doi.org/10.1016/j.sleep.2017.03.028
work_keys_str_mv AT drakatospanagis safetyandefficacyoflongtermuseofsodiumoxybatefornarcolepsywithcataplexyinroutineclinicalpractice
AT lykourasdimosthenis safetyandefficacyoflongtermuseofsodiumoxybatefornarcolepsywithcataplexyinroutineclinicalpractice
AT danconagrainne safetyandefficacyoflongtermuseofsodiumoxybatefornarcolepsywithcataplexyinroutineclinicalpractice
AT higginssean safetyandefficacyoflongtermuseofsodiumoxybatefornarcolepsywithcataplexyinroutineclinicalpractice
AT gildehnadia safetyandefficacyoflongtermuseofsodiumoxybatefornarcolepsywithcataplexyinroutineclinicalpractice
AT macaveiraluca safetyandefficacyoflongtermuseofsodiumoxybatefornarcolepsywithcataplexyinroutineclinicalpractice
AT rosenzweigivana safetyandefficacyoflongtermuseofsodiumoxybatefornarcolepsywithcataplexyinroutineclinicalpractice
AT steierjoerg safetyandefficacyoflongtermuseofsodiumoxybatefornarcolepsywithcataplexyinroutineclinicalpractice
AT williamsadrianj safetyandefficacyoflongtermuseofsodiumoxybatefornarcolepsywithcataplexyinroutineclinicalpractice
AT muzarexford safetyandefficacyoflongtermuseofsodiumoxybatefornarcolepsywithcataplexyinroutineclinicalpractice
AT kentbriand safetyandefficacyoflongtermuseofsodiumoxybatefornarcolepsywithcataplexyinroutineclinicalpractice
AT leschzinerguy safetyandefficacyoflongtermuseofsodiumoxybatefornarcolepsywithcataplexyinroutineclinicalpractice