journal article Open Access Jan 01, 2019

Everolimus for epilepsy and autism spectrum disorder in tuberous sclerosis complex: EXIST-3 substudy in Japan

Brain and Development Vol. 41 No. 1 pp. 1-10 · Elsevier BV
View at Publisher Save 10.1016/j.braindev.2018.07.003
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References
34
[1]
Neurological and neuropsychiatric aspects of tuberous sclerosis complex

Paolo Curatolo, Romina Moavero, Petrus J de Vries

The Lancet Neurology 2015 10.1016/s1474-4422(15)00069-1
[2]
Wang "Optimal management of seizures associated with tuberous sclerosis complex: current and emerging options" Neuropsychiatr Dis Treat (2014)
[3]
Curatolo "Autism spectrum disorders in tuberous sclerosis: pathogenetic pathways and implications for treatment" J Child Neurol (2010) 10.1177/0883073810361789
[4]
Vignoli "Autism spectrum disorder in tuberous sclerosis complex: searching for risk markers" Orphanet J Rare Dis (2015) 10.1186/s13023-015-0371-1
[5]
Stafstrom CE, Hagerman PJ, Pessah IN. Pathophysiology of epilepsy in autism spectrum disorders. In: Noebels JL, Avoli M, Rogawski MA, Olsen RW, Delgado-Escueta AV, editors. Jasper's Basic Mechanisms of the Epilepsies. Bethesda MD: Michael A Rogawski, Antonio V Delgado-Escueta, Jeffrey L Noebels, Massimo Avoli and Richard W Olsen; 2012. 10.1093/med/9780199746545.003.0071
[6]
Krueger "Everolimus treatment of refractory epilepsy in tuberous sclerosis complex" Ann Neurol (2013) 10.1002/ana.23960
[7]
Wong "Mammalian target of rapamycin (mTOR) inhibition as a potential antiepileptogenic therapy: From tuberous sclerosis to common acquired epilepsies" Epilepsia (2010) 10.1111/j.1528-1167.2009.02341.x
[8]
DeFilippis "Treatment of autism spectrum disorder in children and adolescents" Psychopharmacol Bull (2016)
[9]
mTOR, a Potential Target to Treat Autism Spectrum Disorder

Atsushi Sato

CNS & Neurological Disorders - Drug Targets 2016 10.2174/1871527315666160413120638
[10]
Meng "Role of the mTOR signaling pathway in epilepsy" J Neurol Sci (2013) 10.1016/j.jns.2013.05.029
[11]
Sato "Rapamycin reverses impaired social interaction in mouse models of tuberous sclerosis complex" Nat Commun (2012) 10.1038/ncomms2295
[12]
Crino "Biallelic TSC gene inactivation in tuberous sclerosis complex" Neurology (2010) 10.1212/wnl.0b013e3181e04325
[13]
Zeng "Rapamycin prevents epilepsy in a mouse model of tuberous sclerosis complex" Ann Neurol (2008) 10.1002/ana.21331
[14]
Meikle "Response of a neuronal model of tuberous sclerosis to mammalian target of rapamycin (mTOR) inhibitors: effects on mTORC1 and Akt signaling lead to improved survival and function" J Neurosci (2008) 10.1523/jneurosci.0955-08.2008
[15]
Cardamone "Mammalian target of rapamycin inhibitors for intractable epilepsy and subependymal giant cell astrocytomas in tuberous sclerosis complex" J Pediatr (2014) 10.1016/j.jpeds.2013.12.053
[16]
Muncy "Rapamycin reduces seizure frequency in tuberous sclerosis complex" J Child Neurol (2009) 10.1177/0883073808324535
[17]
Kilincaslan "Beneficial effects of everolimus on autism and attention-deficit/hyperactivity disorder symptoms in a group of patients with tuberous sclerosis complex" J Child Adolesc Psychopharmacol (2017) 10.1089/cap.2016.0100
[19]
Roach "Tuberous sclerosis complex consensus conference: revised clinical diagnostic criteria" J Child Neurol (1998) 10.1177/088307389801301206
[20]
National Cancer Institute. Common terminology criteria for adverse events, (CTCAE), version 4.03. June 14, 2010.
[21]
Ito "Validation of an interview-based rating scale developed in Japan for pervasive developmental disorders" Res Autism Spectr Disord (2012) 10.1016/j.rasd.2012.04.002
[22]
Curatolo "Early onset epileptic encephalopathy or genetically determined encephalopathy with early onset epilepsy? Lessons learned from TSC" Eur J Paediatr Neurol (2016) 10.1016/j.ejpn.2015.12.005
[23]
Franz "Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial" Lancet (2013) 10.1016/s0140-6736(12)61134-9
[25]
Ishii "Everolimus improves behavioral deficits in a patient with autism associated with tuberous sclerosis: a case report" Neuropsychiatr Electrophysiol (2015) 10.1186/s40810-015-0004-x
[26]
Kamio "Reliability and validity of the Pervasive Developmental Disorder (PDD)–Autism Society Japan Rating Scale (PARS): a behavior checklist for adolescent and adults with PDDs" Clin Psychiatry (Seishin-Igaku) (2006)
[27]
Song "Comparative analysis of autistic traits and behavioral disorders in Prader-Willi syndrome and Asperger disorder" Am J Med Genet A (2015) 10.1002/ajmg.a.36787
[28]
Sumi "Application of the final DSM-5 criteria for young children with autism spectrum disorder" Autism Open Access (2014)
[29]
de Vries PJ. The Neuroscience of TSC – are we at risk of getting lost between the bench and the bedside? International TSC Research Conference: From DNA to Human Therapies; September 23–26; Chicago 2009.
[30]
Davies "Sirolimus therapy for angiomyolipoma in tuberous sclerosis and sporadic lymphangioleiomyomatosis: a phase 2 trial" Clin Cancer Res (2011) 10.1158/1078-0432.ccr-11-0445
[31]
Krueger "Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis" N Engl J Med (2010) 10.1056/nejmoa1001671
[32]
Hatano "Outcomes of everolimus treatment for renal angiomyolipoma associated with tuberous sclerosis complex: a single institution experience in Japan" Int J Urol (2016) 10.1111/iju.13168
[33]
Davies "Management of everolimus-associated adverse events in patients with tuberous sclerosis complex: a practical guide" Orphanet J Rare Dis (2017) 10.1186/s13023-017-0581-9
[34]
Franz "Long-term use of everolimus in patients with tuberous sclerosis complex: Final results from the EXIST-1 study" PloS One (2016) 10.1371/journal.pone.0158476
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Published
Jan 01, 2019
Vol/Issue
41(1)
Pages
1-10
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Masashi Mizuguchi, Hiroko Ikeda, Kuriko Kagitani-Shimono, et al. (2019). Everolimus for epilepsy and autism spectrum disorder in tuberous sclerosis complex: EXIST-3 substudy in Japan. Brain and Development, 41(1), 1-10. https://doi.org/10.1016/j.braindev.2018.07.003