[article]
| Titre : |
A pilot dose finding study of pioglitazone in autistic children |
| Type de document : |
texte imprimé |
| Auteurs : |
Lucia CAPANO, Auteur ; Annie DUPUIS, Auteur ; Jessica BRIAN, Auteur ; Deepali MANKAD, Auteur ; Lisa GENORE, Auteur ; Rianne HASTIE ADAMS, Auteur ; Sharon SMILE, Auteur ; Toni LUI, Auteur ; Dina ODROBINA, Auteur ; Jane A. FOSTER, Auteur ; Evdokia ANAGNOSTOU, Auteur |
| Article en page(s) : |
59p. |
| Langues : |
Anglais (eng) |
| Mots-clés : |
Autism spectrum disorder Clinical trial Cytokines Drug therapy Efficacy Inflammation Maximum tolerated dose (MTD) Physiological effects of drugs Pioglitazone Safety profile Treatment |
| Index. décimale : |
PER Périodiques |
| Résumé : |
Background: Pioglitazone is a promising compound for treatment of core autism spectrum disorder (ASD) symptoms as it targets multiple relevant pathways, including immune system alterations. Objective: This pilot study aimed to elucidate the maximum tolerated dose, safety, preliminary evidence of efficacy, and appropriate outcome measures in autistic children ages 5-12 years old. Methods: We conducted a 16-week prospective cohort, single blind, single arm, 2-week placebo run-in, dose-finding study of pioglitazone. Twenty-five participants completed treatment. A modified dose finding method was used to determine safety and dose response among three dose levels: 0.25 mg/kg, 0.5 mg/kg, and 0.75 mg/kg once daily. Results: Maximum tolerated dose: there were no serious adverse events (SAEs) and as such the maximum tolerated dose within the range tested was 0.75 mg/Kg once daily.Safety: overall, pioglitazone was well tolerated. Two participants discontinued intervention due to perceived non-efficacy and one due to the inability to tolerate interim blood work. Three participants experienced mild neutropenia.Early evidence of efficacy: statistically significant improvement was observed in social withdrawal, repetitive behaviors, and externalizing behaviors as measured by the Aberrant Behavior Checklist (ABC), Child Yale-Brown Obsessive Compulsive Scale (CY-BOCS), and Repetitive Behavior Scale-Revised (RBS-R). Forty-six percent of those enrolled were deemed to be global responders. Conclusions and relevance: Pioglitazone is well-tolerated and shows a potential signal in measures of social withdrawal, repetitive, and externalizing behaviors. Randomized controlled trials using the confirmed dose are warranted. Trial registration: ClinicalTrials.gov, NCT01205282. Registration date: September 20, 2010. |
| En ligne : |
https://dx.doi.org/10.1186/s13229-018-0241-5 |
| Permalink : |
https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=371 |
in Molecular Autism > 9 (2018) . - 59p.
[article] A pilot dose finding study of pioglitazone in autistic children [texte imprimé] / Lucia CAPANO, Auteur ; Annie DUPUIS, Auteur ; Jessica BRIAN, Auteur ; Deepali MANKAD, Auteur ; Lisa GENORE, Auteur ; Rianne HASTIE ADAMS, Auteur ; Sharon SMILE, Auteur ; Toni LUI, Auteur ; Dina ODROBINA, Auteur ; Jane A. FOSTER, Auteur ; Evdokia ANAGNOSTOU, Auteur . - 59p. Langues : Anglais ( eng) in Molecular Autism > 9 (2018) . - 59p.
| Mots-clés : |
Autism spectrum disorder Clinical trial Cytokines Drug therapy Efficacy Inflammation Maximum tolerated dose (MTD) Physiological effects of drugs Pioglitazone Safety profile Treatment |
| Index. décimale : |
PER Périodiques |
| Résumé : |
Background: Pioglitazone is a promising compound for treatment of core autism spectrum disorder (ASD) symptoms as it targets multiple relevant pathways, including immune system alterations. Objective: This pilot study aimed to elucidate the maximum tolerated dose, safety, preliminary evidence of efficacy, and appropriate outcome measures in autistic children ages 5-12 years old. Methods: We conducted a 16-week prospective cohort, single blind, single arm, 2-week placebo run-in, dose-finding study of pioglitazone. Twenty-five participants completed treatment. A modified dose finding method was used to determine safety and dose response among three dose levels: 0.25 mg/kg, 0.5 mg/kg, and 0.75 mg/kg once daily. Results: Maximum tolerated dose: there were no serious adverse events (SAEs) and as such the maximum tolerated dose within the range tested was 0.75 mg/Kg once daily.Safety: overall, pioglitazone was well tolerated. Two participants discontinued intervention due to perceived non-efficacy and one due to the inability to tolerate interim blood work. Three participants experienced mild neutropenia.Early evidence of efficacy: statistically significant improvement was observed in social withdrawal, repetitive behaviors, and externalizing behaviors as measured by the Aberrant Behavior Checklist (ABC), Child Yale-Brown Obsessive Compulsive Scale (CY-BOCS), and Repetitive Behavior Scale-Revised (RBS-R). Forty-six percent of those enrolled were deemed to be global responders. Conclusions and relevance: Pioglitazone is well-tolerated and shows a potential signal in measures of social withdrawal, repetitive, and externalizing behaviors. Randomized controlled trials using the confirmed dose are warranted. Trial registration: ClinicalTrials.gov, NCT01205282. Registration date: September 20, 2010. |
| En ligne : |
https://dx.doi.org/10.1186/s13229-018-0241-5 |
| Permalink : |
https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=371 |
|  |
Faire une suggestion Affiner la rechercheA pilot dose finding study of pioglitazone in autistic children / Lucia CAPANO in Molecular Autism, 9 (2018)
