A randomized controlled trial with everolimus for IQ and autism in tuberous sclerosis complex
| Autor: | Henriëtte A. Moll, Thijs van der Vaart, Marie-Claire Y. de Wit, Leontine W. ten Hoopen, Floor E. Jansen, André B. Rietman, Iris E. Overwater, Dimitris Rizopoulos, Sabine E. Mous, Karen Bindels-de Heus, Ype Elgersma |
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| Přispěvatelé: | Neurology, Child and Adolescent Psychiatry / Psychology, Pediatrics, Epidemiology, Neurosciences |
| Rok vydání: | 2019 |
| Předmět: |
Male
0301 basic medicine Pediatrics medicine.medical_specialty Adolescent law.invention Executive Function 03 medical and health sciences Tuberous sclerosis 0302 clinical medicine Double-Blind Method Randomized controlled trial Tuberous Sclerosis law Intellectual Disability Intellectual disability medicine Humans Everolimus Cognitive skill Autistic Disorder Child Social Behavior Intelligence Tests Problem Behavior business.industry Communication TOR Serine-Threonine Kinases Neuropsychology Cognition medicine.disease 030104 developmental biology Quality of Life Autism Female Neurology (clinical) Sleep business Immunosuppressive Agents 030217 neurology & neurosurgery medicine.drug |
| Zdroj: | Neurology, 93(2), E200-E209. Lippincott Williams & Wilkins |
| ISSN: | 1526-632X 0028-3878 |
| Popis: | ObjectiveTo investigate whether mammalian target of rapamycin inhibitor everolimus can improve intellectual disability, autism, and other neuropsychological deficits in children with tuberous sclerosis complex (TSC).MethodsIn this 12-month, randomized, double-blind, placebo-controlled trial, we attempted to enroll 60 children with TSC and IQ ResultsThirty-two children with TSC were randomized. Intention-to-treat analysis showed no benefit of everolimus on full-scale IQ (treatment effect −5.6 IQ points, 95% confidence interval −12.3 to 1.0). No effect was found on secondary outcomes, including autism and neuropsychological functioning, and questionnaires examining behavioral problems, social functioning, communication skills, executive functioning, sleep, quality of life, and sensory processing. All patients had adverse events. Two patients on everolimus and 2 patients on placebo discontinued treatment due to adverse events.ConclusionsEverolimus did not improve cognitive functioning, autism, or neuropsychological deficits in children with TSC. The use of everolimus in children with TSC with the aim of improving cognitive function and behavior should not be encouraged in this age group.Clinicaltrials.gov identifierNCT01730209.Classification of evidenceThis study provides Class I evidence that for children with TSC, everolimus does not improve intellectual disability, autism, behavioral problems, or other neuropsychological deficits. |
| Databáze: | OpenAIRE |
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