Effect of desipramine on patients with ...
Type de document :
Article dans une revue scientifique: Article original
DOI :
PMID :
URL permanente :
Titre :
Effect of desipramine on patients with breathing disorders in rett syndrome
Auteur(s) :
Mancini, Josette [Auteur]
Dubus, Jean-Christophe [Auteur]
Jouve, Elisabeth [Auteur]
Roux, Jean-Christophe [Auteur]
Franco, Patricia [Auteur]
Lagrue, Emmanuelle [Auteur]
Castelnau, Pierre [Auteur]
Cances, Claude [Auteur]
Chaix, Yves [Auteur]
Rougeot-Jung, Christelle [Auteur]
Cornu, Catherine [Auteur]
Desportes, Vincent [Auteur]
Vallee, Louis [Auteur]
Bahi-Buisson, Nadia [Auteur]
Truillet, Romain [Auteur]
Attolini, Laurence [Auteur]
Villard, Laurent [Auteur]
Blin, Olivier [Auteur]
Micallef, Joelle [Auteur]
Dubus, Jean-Christophe [Auteur]
Jouve, Elisabeth [Auteur]
Roux, Jean-Christophe [Auteur]
Franco, Patricia [Auteur]
Lagrue, Emmanuelle [Auteur]
Castelnau, Pierre [Auteur]
Cances, Claude [Auteur]
Chaix, Yves [Auteur]
Rougeot-Jung, Christelle [Auteur]
Cornu, Catherine [Auteur]
Desportes, Vincent [Auteur]
Vallee, Louis [Auteur]
Bahi-Buisson, Nadia [Auteur]
Truillet, Romain [Auteur]
Attolini, Laurence [Auteur]
Villard, Laurent [Auteur]
Blin, Olivier [Auteur]
Micallef, Joelle [Auteur]
Titre de la revue :
Annals of clinical and translational neurology
Nom court de la revue :
Ann. Clin. Transl. Neurol.
Numéro :
5
Pagination :
118-127
Date de publication :
2018-02-01
ISSN :
2328-9503
Discipline(s) HAL :
Sciences du Vivant [q-bio]
Résumé en anglais : [en]
Rett Syndrome (RTT) is a severe neurodevelopmental condition with breathing disorders, affecting around one in 10,000 female births. Desipramine, a noradrenaline reuptake inhibitor, reduced the number of apneas in ...
Lire la suite >Rett Syndrome (RTT) is a severe neurodevelopmental condition with breathing disorders, affecting around one in 10,000 female births. Desipramine, a noradrenaline reuptake inhibitor, reduced the number of apneas in Mecp2-deficient mice, a model of RTT. We planned a phase 2 trial to test its efficacy and its safety on breathing patterns in 36 girls with RTT. The trial was a 6-month, multicenter, randomized, double-blind, placebo-controlled study registered with ClinicalTrials.gov, number NCT00990691. Girls diagnosed according to clinical examination and confirmed by genotyping were randomly assigned in a 1:1:1 ratio to receive 2-3 mg/kg Desipramine per day (high Desipramine), 1-2 mg/kg Desipramine per day (low Desipramine), or a placebo. The primary outcome was the change of apnea hypopnea index (AHI), defined by the number of apnea and hypopnea events per hour, assessed at 6 months from baseline. Intention-to-treat analysis was applied. The median change in AHI from baseline to 6 months was -31 (IQR: -37 to -11) for the high Desipramine, -17.5 (IQR: -31 to 13) for the low Desipramine, and -13 (IQR:-31 to 0) for the placebo group. We did not find any significant difference in these changes between the groups (Pr P This first clinical trial of desipramine did not show clinical efficacy. Although required further studies, the significant correlation between Desipramine concentrations and improvement of AHI provided additional and relevant reasons to test the noradrenergic pathway in RTT.Lire moins >
Lire la suite >Rett Syndrome (RTT) is a severe neurodevelopmental condition with breathing disorders, affecting around one in 10,000 female births. Desipramine, a noradrenaline reuptake inhibitor, reduced the number of apneas in Mecp2-deficient mice, a model of RTT. We planned a phase 2 trial to test its efficacy and its safety on breathing patterns in 36 girls with RTT. The trial was a 6-month, multicenter, randomized, double-blind, placebo-controlled study registered with ClinicalTrials.gov, number NCT00990691. Girls diagnosed according to clinical examination and confirmed by genotyping were randomly assigned in a 1:1:1 ratio to receive 2-3 mg/kg Desipramine per day (high Desipramine), 1-2 mg/kg Desipramine per day (low Desipramine), or a placebo. The primary outcome was the change of apnea hypopnea index (AHI), defined by the number of apnea and hypopnea events per hour, assessed at 6 months from baseline. Intention-to-treat analysis was applied. The median change in AHI from baseline to 6 months was -31 (IQR: -37 to -11) for the high Desipramine, -17.5 (IQR: -31 to 13) for the low Desipramine, and -13 (IQR:-31 to 0) for the placebo group. We did not find any significant difference in these changes between the groups (Pr P This first clinical trial of desipramine did not show clinical efficacy. Although required further studies, the significant correlation between Desipramine concentrations and improvement of AHI provided additional and relevant reasons to test the noradrenergic pathway in RTT.Lire moins >
Langue :
Anglais
Audience :
Internationale
Vulgarisation :
Non
Établissement(s) :
CHU Lille
CNRS
Inserm
Université de Lille
CNRS
Inserm
Université de Lille
Collections :
Équipe(s) de recherche :
Troubles cognitifs dégénératifs et vasculaires
Date de dépôt :
2019-11-27T14:29:43Z