Everolimus for treatment of tuberous sclerosis complex-associated neuropsychiatric disorders

dc.contributor.authorKrueger, Darcy A
dc.contributor.authorSahin, Mustafa
dc.date.accessioned2021-10-08T11:00:56Z
dc.date.available2021-10-08T11:00:56Z
dc.date.issued2017
dc.description.abstractAbstract Objective: To evaluate if short‐term treatment with everolimus was safe and could improve neurocognition and behavior in children with TSC. Methods: This was a prospective, double‐blind randomized, placebo‐controlled two‐center phase II study. Participants diagnosed with TSC and age 6–21 years were treated with 4.5 mg/m2 per day of oral everolimus (n = 32) or matching placebo (n = 15) taken once daily for 6 months. For efficacy, a comprehensive neurocognitive and behavioral evaluation battery was performed at baseline, 3 months, and 6 months. For safety, adverse events recorded continuously via patient diary were categorized and graded per NCI Common Toxicity Criteria for Adverse Events, version 3.0 (CTCAE 3.0). Analyses were performed on the intention‐to‐treat population (n = 47). Results: Nearly all assessment measures failed to demonstrate significant differences between the two groups at the end of 6 months. Only one measure each of executive function (Cambridge Neuropsychological Test Automated Battery Stockings of Cambridge) favoring placebo (P = 0.025) and social cognition (Social Responsiveness Scale Social Cognition Subscale) favoring everolimus (P = 0.011) was observed. A total of 473 adverse events (AE) were reported. The average number of total AE per subject was similar for both placebo and everolimus. Most were mild or moderate in severity and serious AE were rare. Interpretation While safe, oral everolimus administered once daily for 6 months did not significantly improve neurocognitive functioning or behavior in children with TSC.
dc.identifier.apacitationKrueger, D. A., & Sahin, M. (2017). Everolimus for treatment of tuberous sclerosis complex-associated neuropsychiatric disorders. <i>Annals of Clinical and Translational Neurology</i>, 4(12), 877 - 887. http://hdl.handle.net/11427/35048en_ZA
dc.identifier.chicagocitationKrueger, Darcy A, and Mustafa Sahin "Everolimus for treatment of tuberous sclerosis complex-associated neuropsychiatric disorders." <i>Annals of Clinical and Translational Neurology</i> 4, 12. (2017): 877 - 887. http://hdl.handle.net/11427/35048en_ZA
dc.identifier.citationKrueger, D.A. & Sahin, M. 2017. Everolimus for treatment of tuberous sclerosis complex-associated neuropsychiatric disorders. <i>Annals of Clinical and Translational Neurology.</i> 4(12):877 - 887. http://hdl.handle.net/11427/35048en_ZA
dc.identifier.issn2328-9503
dc.identifier.ris TY - Journal Article AU - Krueger, Darcy A AU - Sahin, Mustafa AB - Abstract Objective: To evaluate if short‐term treatment with everolimus was safe and could improve neurocognition and behavior in children with TSC. Methods: This was a prospective, double‐blind randomized, placebo‐controlled two‐center phase II study. Participants diagnosed with TSC and age 6–21 years were treated with 4.5 mg/m2 per day of oral everolimus (n = 32) or matching placebo (n = 15) taken once daily for 6 months. For efficacy, a comprehensive neurocognitive and behavioral evaluation battery was performed at baseline, 3 months, and 6 months. For safety, adverse events recorded continuously via patient diary were categorized and graded per NCI Common Toxicity Criteria for Adverse Events, version 3.0 (CTCAE 3.0). Analyses were performed on the intention‐to‐treat population (n = 47). Results: Nearly all assessment measures failed to demonstrate significant differences between the two groups at the end of 6 months. Only one measure each of executive function (Cambridge Neuropsychological Test Automated Battery Stockings of Cambridge) favoring placebo (P = 0.025) and social cognition (Social Responsiveness Scale Social Cognition Subscale) favoring everolimus (P = 0.011) was observed. A total of 473 adverse events (AE) were reported. The average number of total AE per subject was similar for both placebo and everolimus. Most were mild or moderate in severity and serious AE were rare. Interpretation While safe, oral everolimus administered once daily for 6 months did not significantly improve neurocognitive functioning or behavior in children with TSC. DA - 2017 DB - OpenUCT DP - University of Cape Town IS - 12 J1 - Annals of Clinical and Translational Neurology LK - https://open.uct.ac.za PY - 2017 SM - 2328-9503 T1 - Everolimus for treatment of tuberous sclerosis complex-associated neuropsychiatric disorders TI - Everolimus for treatment of tuberous sclerosis complex-associated neuropsychiatric disorders UR - http://hdl.handle.net/11427/35048 ER - en_ZA
dc.identifier.urihttp://hdl.handle.net/11427/35048
dc.identifier.vancouvercitationKrueger DA, Sahin M. Everolimus for treatment of tuberous sclerosis complex-associated neuropsychiatric disorders. Annals of Clinical and Translational Neurology. 2017;4(12):877 - 887. http://hdl.handle.net/11427/35048.en_ZA
dc.language.isoeng
dc.publisher.departmentDivision of Child and Adolescent Psychiatry
dc.publisher.facultyFaculty of Health Sciences
dc.sourceAnnals of Clinical and Translational Neurology
dc.source.journalissue12
dc.source.journalvolume4
dc.source.pagination877 - 887
dc.source.urihttps://dx.doi.org/10.1002/acn3.494
dc.subject.otherJournal Article
dc.titleEverolimus for treatment of tuberous sclerosis complex-associated neuropsychiatric disorders
dc.typeJournal Article
uct.type.publicationResearch
uct.type.resourceJournal Article
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