Profile of everolimus in the treatment of tuberous sclerosis complex: an evidence-based review of its place in therapy

Jamie K Capal, David Neal Franz Department of Neurology, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA Abstract: Tuberous sclerosis complex (TSC) is a relatively rare genetic disorder, affecting one in 6,000 births. Mammalian target of rapamycin (mTOR) inhibitors, su...

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Autores principales:	Capal JK, Franz DN
Formato:	article
Lenguaje:	EN
Publicado:	Dove Medical Press 2016
Materias:	Everolimus tuberous sclerosis complex subependymal giant cell astrocytomas seizures TAND Neurosciences. Biological psychiatry. Neuropsychiatry RC321-571 Neurology. Diseases of the nervous system RC346-429
Acceso en línea:	https://doaj.org/article/e977fd6235494b208646542590c76c31
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spelling	oai:doaj.org-article:e977fd6235494b208646542590c76c312021-12-02T03:38:33ZProfile of everolimus in the treatment of tuberous sclerosis complex: an evidence-based review of its place in therapy1178-2021https://doaj.org/article/e977fd6235494b208646542590c76c312016-08-01T00:00:00Zhttps://www.dovepress.com/profile-of-everolimus-in-the-treatment-of-tuberous-sclerosis-complex-a-peer-reviewed-article-NDThttps://doaj.org/toc/1178-2021Jamie K Capal, David Neal Franz Department of Neurology, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA Abstract: Tuberous sclerosis complex (TSC) is a relatively rare genetic disorder, affecting one in 6,000 births. Mammalian target of rapamycin (mTOR) inhibitors, such as everolimus, which have been previously used to prevent solid organ transplant rejection, augment anticancer treatment regimens, and prevent neovascularization of artificial cardiac stents, are now approved for treating TSC-related manifestations, such as subependymal giant cell astrocytomas and renal angiomyolipomas. The use of everolimus in treating subependymal giant cell astrocytomas is supported by long-term Phase II and III clinical trials. Seizures are a common feature in TSC, occurring in up to 96% of patients. While mTOR inhibitors currently do not have regulatory approval in treating this manifestation, small clinical studies have demonstrated beneficial outcomes with everolimus. Further evidence from a forthcoming Phase III clinical study may provide additional support for the use of everolimus for this indication. Also, there are no approved treatments for TSC-associated neuropsychiatric disorders, which include intellectual disability, behavioral difficulties, and autism spectrum disorder, but preclinical data and small studies have suggested that some neuropsychiatric symptoms may be improved through mTOR inhibition therapy. More evidence is needed, particularly regarding safety in young infants. This review focuses on the current evidence supporting the use of everolimus in neurologic and neuropsychiatric manifestations of TSC, and the place of everolimus in therapy. Keywords: everolimus, tuberous sclerosis complex, subependymal giant cell astrocytomas, seizures, TANDCapal JKFranz DNDove Medical PressarticleEverolimustuberous sclerosis complexsubependymal giant cell astrocytomasseizuresTANDNeurosciences. Biological psychiatry. NeuropsychiatryRC321-571Neurology. Diseases of the nervous systemRC346-429ENNeuropsychiatric Disease and Treatment, Vol Volume 12, Pp 2165-2172 (2016)
institution	DOAJ
collection	DOAJ
language	EN
topic	Everolimus tuberous sclerosis complex subependymal giant cell astrocytomas seizures TAND Neurosciences. Biological psychiatry. Neuropsychiatry RC321-571 Neurology. Diseases of the nervous system RC346-429
spellingShingle	Everolimus tuberous sclerosis complex subependymal giant cell astrocytomas seizures TAND Neurosciences. Biological psychiatry. Neuropsychiatry RC321-571 Neurology. Diseases of the nervous system RC346-429 Capal JK Franz DN Profile of everolimus in the treatment of tuberous sclerosis complex: an evidence-based review of its place in therapy
description	Jamie K Capal, David Neal Franz Department of Neurology, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA Abstract: Tuberous sclerosis complex (TSC) is a relatively rare genetic disorder, affecting one in 6,000 births. Mammalian target of rapamycin (mTOR) inhibitors, such as everolimus, which have been previously used to prevent solid organ transplant rejection, augment anticancer treatment regimens, and prevent neovascularization of artificial cardiac stents, are now approved for treating TSC-related manifestations, such as subependymal giant cell astrocytomas and renal angiomyolipomas. The use of everolimus in treating subependymal giant cell astrocytomas is supported by long-term Phase II and III clinical trials. Seizures are a common feature in TSC, occurring in up to 96% of patients. While mTOR inhibitors currently do not have regulatory approval in treating this manifestation, small clinical studies have demonstrated beneficial outcomes with everolimus. Further evidence from a forthcoming Phase III clinical study may provide additional support for the use of everolimus for this indication. Also, there are no approved treatments for TSC-associated neuropsychiatric disorders, which include intellectual disability, behavioral difficulties, and autism spectrum disorder, but preclinical data and small studies have suggested that some neuropsychiatric symptoms may be improved through mTOR inhibition therapy. More evidence is needed, particularly regarding safety in young infants. This review focuses on the current evidence supporting the use of everolimus in neurologic and neuropsychiatric manifestations of TSC, and the place of everolimus in therapy. Keywords: everolimus, tuberous sclerosis complex, subependymal giant cell astrocytomas, seizures, TAND
format	article
author	Capal JK Franz DN
author_facet	Capal JK Franz DN
author_sort	Capal JK
title	Profile of everolimus in the treatment of tuberous sclerosis complex: an evidence-based review of its place in therapy
title_short	Profile of everolimus in the treatment of tuberous sclerosis complex: an evidence-based review of its place in therapy
title_full	Profile of everolimus in the treatment of tuberous sclerosis complex: an evidence-based review of its place in therapy
title_fullStr	Profile of everolimus in the treatment of tuberous sclerosis complex: an evidence-based review of its place in therapy
title_full_unstemmed	Profile of everolimus in the treatment of tuberous sclerosis complex: an evidence-based review of its place in therapy
title_sort	profile of everolimus in the treatment of tuberous sclerosis complex: an evidence-based review of its place in therapy
publisher	Dove Medical Press
publishDate	2016
url	https://doaj.org/article/e977fd6235494b208646542590c76c31
work_keys_str_mv	AT capaljk profileofeverolimusinthetreatmentoftuberoussclerosiscomplexanevidencebasedreviewofitsplaceintherapy AT franzdn profileofeverolimusinthetreatmentoftuberoussclerosiscomplexanevidencebasedreviewofitsplaceintherapy
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Profile of everolimus in the treatment of tuberous sclerosis complex: an evidence-based review of its place in therapy

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