{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,9]],"date-time":"2026-01-09T23:04:44Z","timestamp":1767999884627,"version":"3.49.0"},"reference-count":61,"publisher":"Springer Science and Business Media LLC","issue":"5","license":[{"start":{"date-parts":[[2016,2,29]],"date-time":"2016-02-29T00:00:00Z","timestamp":1456704000000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Drugs"],"published-print":{"date-parts":[[2016,4]]},"DOI":"10.1007\/s40265-016-0552-9","type":"journal-article","created":{"date-parts":[[2016,2,29]],"date-time":"2016-02-29T13:34:19Z","timestamp":1456752859000},"page":"551-565","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":68,"title":["The Role of mTOR Inhibitors in the Treatment of Patients with Tuberous Sclerosis Complex: Evidence-based and Expert Opinions"],"prefix":"10.1007","volume":"76","author":[{"given":"Paolo","family":"Curatolo","sequence":"first","affiliation":[]},{"given":"Marit","family":"Bj\u00f8rnvold","sequence":"additional","affiliation":[]},{"given":"Patricia E.","family":"Dill","sequence":"additional","affiliation":[]},{"given":"Jos\u00e9 Carlos","family":"Ferreira","sequence":"additional","affiliation":[]},{"given":"Martha","family":"Feucht","sequence":"additional","affiliation":[]},{"given":"Christoph","family":"Hertzberg","sequence":"additional","affiliation":[]},{"given":"Anna","family":"Jansen","sequence":"additional","affiliation":[]},{"given":"Sergiusz","family":"J\u00f3\u017awiak","sequence":"additional","affiliation":[]},{"given":"J. Christopher","family":"Kingswood","sequence":"additional","affiliation":[]},{"given":"Katarzyna","family":"Kotulska","sequence":"additional","affiliation":[]},{"given":"Alfons","family":"Macaya","sequence":"additional","affiliation":[]},{"given":"Romina","family":"Moavero","sequence":"additional","affiliation":[]},{"given":"Rima","family":"Nabbout","sequence":"additional","affiliation":[]},{"given":"Bernard A.","family":"Zonnenberg","sequence":"additional","affiliation":[]}],"member":"297","published-online":{"date-parts":[[2016,2,29]]},"reference":[{"issue":"3","key":"552_CR1","doi-asserted-by":"crossref","first-page":"319","DOI":"10.1038\/ejhg.2008.170","volume":"17","author":"M Nellist","year":"2009","unstructured":"Nellist M, van den Heuvel D, Schluep D, Exalto C, Goedbloed M, Maat-Kievit A, et al. Missense mutations to the TSC1 gene cause tuberous sclerosis complex. Eur J Hum Genet. 2009;17(3):319\u201328.","journal-title":"Eur J Hum Genet."},{"issue":"4","key":"552_CR2","doi-asserted-by":"crossref","first-page":"404","DOI":"10.2174\/1570159X11209040404","volume":"10","author":"P Curatolo","year":"2012","unstructured":"Curatolo P, Moavero R. mTOR inhibitors in tuberous sclerosis complex. Curr Neuropharmacol. 2012;10(4):404\u201315.","journal-title":"Curr Neuropharmacol."},{"issue":"9639","key":"552_CR3","doi-asserted-by":"crossref","first-page":"657","DOI":"10.1016\/S0140-6736(08)61279-9","volume":"372","author":"P Curatolo","year":"2008","unstructured":"Curatolo P, Bombardieri R, Jozwiak S. Tuberous sclerosis. Lancet. 2008;372(9639):657\u201368.","journal-title":"Lancet."},{"key":"552_CR4","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1186\/1824-7288-39-57","volume":"39","author":"R Moavero","year":"2013","unstructured":"Moavero R, Coniglio A, Garaci F, Curatolo P. Is mTOR inhibition a systemic treatment for tuberous sclerosis? Ital J Pediatr. 2013;39:57.","journal-title":"Ital J Pediatr."},{"key":"552_CR5","doi-asserted-by":"crossref","first-page":"243","DOI":"10.1016\/j.pediatrneurol.2013.08.001","volume":"49","author":"H Northrup","year":"2013","unstructured":"Northrup H, Krueger DA, International Tuberous Sclerosis Complex Consensus Group. Tuberous sclerosis complex diagnostic criteria update: recommendations of the 2012 international tuberous sclerosis complex consensus conference. Pediatr Neurol. 2013;49:243\u201354.","journal-title":"Pediatr Neurol."},{"issue":"4","key":"552_CR6","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1016\/j.pediatrneurol.2013.08.002","volume":"49","author":"DA Krueger","year":"2013","unstructured":"Krueger DA, Northrup H, International Tuberous Sclerosis Complex Consensus Group. Tuberous sclerosis complex surveillance and management: recommendations of the 2012 international tuberous sclerosis complex consensus conference. Pediatr Neurol. 2013;49(4):255\u201365.","journal-title":"Pediatr Neurol."},{"issue":"7","key":"552_CR7","doi-asserted-by":"crossref","first-page":"733","DOI":"10.1016\/S1474-4422(15)00069-1","volume":"14","author":"P Curatolo","year":"2015","unstructured":"Curatolo P, Moavero R, de Vries PJ. Neurological and neuropsychiatric aspects of tuberous sclerosis complex. Lancet Neurol. 2015;14(7):733\u201345.","journal-title":"Lancet Neurol."},{"key":"552_CR8","doi-asserted-by":"crossref","first-page":"51","DOI":"10.2165\/11207730-000000000-00000","volume":"14","author":"MP Curran","year":"2012","unstructured":"Curran MP. Everolimus: in patients with subependymal giant cell astrocytoma associated with tuberous sclerosis complex. Paediatric Drugs. 2012;14:51\u201360.","journal-title":"Paediatric Drugs."},{"key":"552_CR9","doi-asserted-by":"crossref","first-page":"1181","DOI":"10.1586\/era.11.93","volume":"11","author":"DN Franz","year":"2011","unstructured":"Franz DN. Everolimus: an mTOR inhibitor for the treatment of tuberous sclerosis. Expert Rev Anticancer Ther. 2011;11:1181\u201392.","journal-title":"Expert Rev Anticancer Ther."},{"issue":"19","key":"552_CR10","doi-asserted-by":"crossref","first-page":"1801","DOI":"10.1056\/NEJMoa1001671","volume":"363","author":"DA Krueger","year":"2010","unstructured":"Krueger DA, Care MM, Holland K, Agricola K, Tudor C, Mangeshkar P, et al. Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis. N Engl J Med. 2010;363(19):1801\u201311.","journal-title":"N Engl J Med."},{"issue":"9869","key":"552_CR11","doi-asserted-by":"crossref","first-page":"817","DOI":"10.1016\/S0140-6736(12)61767-X","volume":"381","author":"JJ Bissler","year":"2013","unstructured":"Bissler JJ, Kingswood JC, Radzikowska E, Zonnenberg BA, Frost M, Belousova E, et al. Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet. 2013;381(9869):817\u201324.","journal-title":"Lancet."},{"issue":"9861","key":"552_CR12","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1016\/S0140-6736(12)61134-9","volume":"381","author":"DN Franz","year":"2013","unstructured":"Franz DN, Belousova E, Sparagana S, Bebin EM, Frost M, Kuperman R, et al. 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;381(9861):125\u201332.","journal-title":"Lancet."},{"issue":"5","key":"552_CR13","doi-asserted-by":"crossref","first-page":"679","DOI":"10.1002\/ana.23960","volume":"74","author":"DA Krueger","year":"2013","unstructured":"Krueger DA, Wilfong AA, Holland-Bouley K, Anderson AE, Agricola K, Tudor C, et al. Everolimus treatment of refractory epilepsy in tuberous sclerosis complex. Ann Neurol. 2013;74(5):679\u201387.","journal-title":"Ann Neurol."},{"key":"552_CR14","doi-asserted-by":"crossref","first-page":"929","DOI":"10.1002\/ana.24523","volume":"78","author":"DN Franz","year":"2015","unstructured":"Franz DN, Agricola K, Mays M, Tudor C, Care MM, Holland-Bouley K, et al. Everolimus for subependymal giant cell astrocytoma: 5-year final analysis. Ann Neurol. 2015;78:929\u201338. doi: 10.1002\/ana.24523 .","journal-title":"Ann Neurol."},{"issue":"4","key":"552_CR15","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1016\/j.ejpn.2012.12.008","volume":"17","author":"S J\u00f3\u017awiak","year":"2013","unstructured":"J\u00f3\u017awiak S, Nabbout R, Curatolo P, participants of the TSC Consensus Meeting for SEGA and Epilepsy Management. Management of subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex (TSC): clinical recommendations. Eur J Paediatr Neurol. 2013;17(4):348\u201352.","journal-title":"Eur J Paediatr Neurol."},{"issue":"1","key":"552_CR16","doi-asserted-by":"crossref","first-page":"36","DOI":"10.1016\/j.ejpn.2012.10.005","volume":"17","author":"S Amin","year":"2013","unstructured":"Amin S, Carter M, Edwards RJ, Pople I, Aquilina K, Merrifield J, et al. The outcome of surgical management of subependymal giant cell astrocytoma in tuberous sclerosis complex. Eur J Paediatr Neurol. 2013;17(1):36\u201344.","journal-title":"Eur J Paediatr Neurol."},{"issue":"6","key":"552_CR17","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1016\/j.pediatrneurol.2013.08.017","volume":"49","author":"J Roth","year":"2013","unstructured":"Roth J, Roach ES, Bartels U, J\u00f3\u017awiak S, Koenig MK, Weiner HL, et al. Subependymal giant cell astrocytoma: diagnosis, screening, and treatment. Recommendations from the international tuberous sclerosis complex consensus conference 2012. Pediatr Neurol. 2013;49(6):439\u201344.","journal-title":"Pediatr Neurol."},{"issue":"1","key":"552_CR18","doi-asserted-by":"crossref","first-page":"83","DOI":"10.1016\/j.ejpn.2011.09.006","volume":"16","author":"M Perek-Polnik","year":"2012","unstructured":"Perek-Polnik M, J\u00f3\u017awiak S, Jurkiewicz E, Perek D, Kotulska K. Effective everolimus treatment of inoperable, life-threatening subependymal giant cell astrocytoma and intractable epilepsy in a patient with tuberous sclerosis complex. Eur J Paediatr Neurol. 2012;16(1):83\u20135.","journal-title":"Eur J Paediatr Neurol."},{"key":"552_CR19","unstructured":"Afinitor\u00ae (everolimus). Prescribing information. Novartis, revised July 2014. http:\/\/www.pharma.us.novartis.com\/product\/pi\/pdf\/afinitor.pdf . Accessed 1 Sept 2014."},{"issue":"4","key":"552_CR20","doi-asserted-by":"crossref","first-page":"307","DOI":"10.1016\/j.pediatrneurol.2013.12.004","volume":"50","author":"K Kotulska","year":"2014","unstructured":"Kotulska K, Borkowska J, Roszkowski M, Mandera M, Daszkiewicz P, Drabik K, et al. Surgical treatment of subependymal giant cell astrocytoma in tuberous sclerosis complex patients. Pediatr Neurol. 2014;50(4):307\u201312.","journal-title":"Pediatr Neurol."},{"issue":"6","key":"552_CR21","doi-asserted-by":"crossref","first-page":"574","DOI":"10.1212\/WNL.0b013e3182815428","volume":"80","author":"DA Krueger","year":"2013","unstructured":"Krueger DA, Care MM, Agricola K, Tudor C, Mays M, Franz DN. Everolimus long-term safety and efficacy in subependymal giant cell astrocytoma. Neurology. 2013;80(6):574\u201380.","journal-title":"Neurology."},{"key":"552_CR22","unstructured":"ClinicalTrials.gov identifier: NCT01713946. A placebo-controlled study of efficacy and safety of 2 trough-ranges of everolimus as adjunctive therapy in patients with tuberous sclerosis complex (TSC) and refractory partial-onset seizures (EXIST-3). Accessed 1 Sept 2014."},{"issue":"12","key":"552_CR23","doi-asserted-by":"crossref","first-page":"4071","DOI":"10.1158\/1078-0432.CCR-11-0445","volume":"17","author":"DM Davies","year":"2011","unstructured":"Davies DM, de Vries PJ, Johnson SR, McCartney DL, Cox JA, Serra AL, et al. Sirolimus therapy for angiomyolipoma in tuberous sclerosis and sporadic lymphangioleiomyomatosis: a phase 2 trial. Clin Cancer Res. 2011;17(12):4071\u201381.","journal-title":"Clin Cancer Res."},{"issue":"2","key":"552_CR24","doi-asserted-by":"crossref","first-page":"140","DOI":"10.1056\/NEJMoa063564","volume":"358","author":"JJ Bissler","year":"2008","unstructured":"Bissler JJ, McCormack FX, Young LR, Elwing JM, Chuck G, Leonard JM, et al. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med. 2008;358(2):140\u201351.","journal-title":"N Engl J Med."},{"issue":"9","key":"552_CR25","doi-asserted-by":"crossref","first-page":"e23379","DOI":"10.1371\/journal.pone.0023379","volume":"6","author":"SL Dabora","year":"2011","unstructured":"Dabora SL, Franz DN, Ashwal S, Sagalowsky A, DiMario FJ Jr, Miles D, et al. Multicenter phase 2 trial of sirolimus for tuberous sclerosis: kidney angiomyolipomas and other tumors regress and VEGF-D levels decrease. PLoS One. 2011;6(9):e23379.","journal-title":"PLoS One."},{"issue":"13","key":"552_CR26","doi-asserted-by":"crossref","first-page":"1513","DOI":"10.1016\/S1470-2045(14)70489-9","volume":"15","author":"DN Franz","year":"2014","unstructured":"Franz DN, Belousova E, Sparagana S, Bebin EM, Frost M, Kuperman R, et al. Everolimus for supependymal giant cell astrocytoma in patients with tuberous sclerosis complex: 2-year open-label extension of the randomised EXIST-1 study. Lancet Oncol. 2014;15(13):1513\u201320.","journal-title":"Lancet Oncol."},{"issue":"8","key":"552_CR27","doi-asserted-by":"crossref","first-page":"1203","DOI":"10.1007\/s00381-011-1406-0","volume":"27","author":"R Moavero","year":"2011","unstructured":"Moavero R, Pinci M, Bombardieri R, Curatolo P. The management of subependymal giant cell tumors in tuberous sclerosis: a clinician\u2019s perspective. Childs Nerv Syst. 2011;27(8):1203\u201310.","journal-title":"Childs Nerv Syst."},{"key":"552_CR28","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1159\/000060178","volume":"136","author":"VE Torres","year":"2001","unstructured":"Torres VE, King BF, McKusick MA, Bjornsson J, Zincke H. Update on tuberous sclerosis complex. Contrib Nephrol. 2001;136:33\u201349.","journal-title":"Contrib Nephrol."},{"issue":"4","key":"552_CR29","doi-asserted-by":"crossref","first-page":"735","DOI":"10.1016\/j.yebeh.2011.08.037","volume":"22","author":"R Cusmai","year":"2011","unstructured":"Cusmai R, Moavero R, Bombardieri R, Vigevano F, Curatolo P. Long-term neurological outcome in children with early-onset epilepsy associated with tuberous sclerosis. Epilepsy Behav. 2011;22(4):735\u20139.","journal-title":"Epilepsy Behav."},{"issue":"5","key":"552_CR30","doi-asserted-by":"crossref","first-page":"424","DOI":"10.1016\/j.ejpn.2011.03.010","volume":"15","author":"S J\u00f3\u017awiak","year":"2011","unstructured":"J\u00f3\u017awiak S, Kotulska K, Doma\u0144ska-Pakie\u0142a D, Lojszczyk B, Syczewska M, Chmielewski D, et al. Antiepileptic treatment before the onset of seizures reduces epilepsy severity and risk of mental retardation in infants with tuberous sclerosis complex. Eur J Paediatr Neurol. 2011;15(5):424\u201331.","journal-title":"Eur J Paediatr Neurol."},{"issue":"4","key":"552_CR31","doi-asserted-by":"crossref","first-page":"458","DOI":"10.1016\/j.ejpn.2013.12.006","volume":"18","author":"D Doma\u0144ska-Pakie\u0142a","year":"2014","unstructured":"Doma\u0144ska-Pakie\u0142a D, Kaczorowska M, Jurkiewicz E, Kotulska K, Dunin-W\u0105sowicz D, J\u00f3\u017awiak S. EEG abnormalities preceding the epilepsy onset in tuberous sclerosis complex patients\u2014a prospective study of 5 patients. Eur J Paediatr Neurol. 2014;18(4):458\u201368.","journal-title":"Eur J Paediatr Neurol."},{"issue":"6","key":"552_CR32","doi-asserted-by":"crossref","first-page":"582","DOI":"10.1016\/j.ejpn.2012.05.004","volume":"16","author":"P Curatolo","year":"2012","unstructured":"Curatolo P, J\u00f3\u017awiak S, Nabbout R, TSC Consensus Meeting for SEGA and Epilepsy Management. Management of epilepsy associated with tuberous sclerosis complex (TSC): clinical recommendations. Eur J Paediatr Neurol. 2012;16(6):582\u20136.","journal-title":"Eur J Paediatr Neurol."},{"issue":"2","key":"552_CR33","doi-asserted-by":"crossref","first-page":"146","DOI":"10.1016\/j.ejpn.2009.03.003","volume":"14","author":"R Bombardieri","year":"2010","unstructured":"Bombardieri R, Pinci M, Moavero R, Cerminara C, Curatolo P. Early control of seizures improves long-term outcome in children with tuberous sclerosis complex. Eur J Paediatr Neurol. 2010;14(2):146\u20139.","journal-title":"Eur J Paediatr Neurol."},{"key":"552_CR34","doi-asserted-by":"crossref","first-page":"e57445","DOI":"10.1371\/journal.pone.0057445","volume":"8","author":"B Zhang","year":"2013","unstructured":"Zhang B, McDaniel SS, Rensing NR, Wong M. Vigabatrin inhibits seizures and mTOR pathway activation in a mouse model of tuberous sclerosis complex. PLoS One. 2013;8:e57445.","journal-title":"PLoS One."},{"issue":"2","key":"552_CR35","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1111\/j.1528-1167.2008.01988.x","volume":"50","author":"LJ Willmore","year":"2009","unstructured":"Willmore LJ, Abelson MB, Ben-Menachem E, Pellock JM, Shields WD. Vigabatrin: 2008 update. Epilepsia. 2009;50(2):163\u201373.","journal-title":"Epilepsia."},{"key":"552_CR36","first-page":"CD001770","volume":"6","author":"EC Hancock","year":"2013","unstructured":"Hancock EC, Osborne JP, Edwards SW. Treatment of infantile spasms. Cochrane Database Syst Rev. 2013;6:CD001770.","journal-title":"Cochrane Database Syst Rev."},{"issue":"3","key":"552_CR37","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1016\/j.eplepsyres.2013.02.016","volume":"105","author":"K Zhang","year":"2013","unstructured":"Zhang K, Hu WH, Zhang C, Meng FG, Chen N, Zhang JG. Predictors of seizure freedom after surgical management of tuberous sclerosis complex: a systematic review and meta-analysis. Epilepsy Res. 2013;105(3):377\u201383.","journal-title":"Epilepsy Res."},{"key":"552_CR38","first-page":"31","volume":"5","author":"F Pittau","year":"2014","unstructured":"Pittau F, Grouiller F, Spinelli L, Seeck M, Michel CM, Vulliemoz S. The role of functional neuroimaging in pre-surgical epilepsy evaluation. Front Neurol. 2014;5:31.","journal-title":"Front Neurol."},{"issue":"5","key":"552_CR39","doi-asserted-by":"crossref","first-page":"479","DOI":"10.1016\/j.ejpn.2013.03.002","volume":"17","author":"K Kotulska","year":"2013","unstructured":"Kotulska K, Chmielewski D, Borkowska J, Jurkiewicz E, Kuczy\u0144ski D, Kmie\u0107 T, et al. Long-term effect of everolimus on epilepsy and growth in children under 3\u00a0years of age treated for subependymal giant cell astrocytoma associated with tuberous sclerosis complex. Eur J Paediatr Neurol. 2013;17(5):479\u201385.","journal-title":"Eur J Paediatr Neurol."},{"issue":"1","key":"552_CR40","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1016\/j.pediatrneurol.2010.03.003","volume":"43","author":"N Zamponi","year":"2010","unstructured":"Zamponi N, Petrelli C, Passamonti C, Moavero R, Curatolo P. Vagus nerve stimulation for refractory epilepsy in tuberous sclerosis. Pediatr Neurol. 2010;43(1):29\u201334.","journal-title":"Pediatr Neurol."},{"issue":"10","key":"552_CR41","doi-asserted-by":"crossref","first-page":"1684","DOI":"10.1111\/j.1528-1167.2005.00266.x","volume":"46","author":"EH Kossoff","year":"2005","unstructured":"Kossoff EH, Thiele EA, Pfeifer HH, McGrogan JR, Freeman JM. Tuberous sclerosis complex and the ketogenic diet. Epilepsia. 2005;46(10):1684\u20136.","journal-title":"Epilepsia."},{"issue":"1\u20132","key":"552_CR42","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1016\/j.eplepsyres.2011.10.021","volume":"99","author":"AM Larson","year":"2012","unstructured":"Larson AM, Pfeifer HH, Thiele EA. Low glycemic index treatment for epilepsy in tuberous sclerosis complex. Epilepsy Res. 2012;99(1\u20132):180\u20132.","journal-title":"Epilepsy Res."},{"issue":"3","key":"552_CR43","doi-asserted-by":"crossref","first-page":"454","DOI":"10.1016\/j.yebeh.2009.08.018","volume":"16","author":"RE Elliott","year":"2009","unstructured":"Elliott RE, Carlson C, Kalhorn SP, Moshel YA, Weiner HL, Devinsky O, et al. Refractory epilepsy in tuberous sclerosis: vagus nerve stimulation with or without subsequent resective surgery. Epilepsy Behav. 2009;16(3):454\u201360.","journal-title":"Epilepsy Behav."},{"key":"552_CR44","doi-asserted-by":"crossref","first-page":"e7","DOI":"10.1111\/j.1528-1167.2011.02981.x","volume":"52","author":"SS McDaniel","year":"2011","unstructured":"McDaniel SS, Rensing NR, Thio LL, Yamada KA, Wong M. The ketogenic diet inhibits the mammalian target of rapamycin (mTOR) pathway. Epilepsia. 2011;52:e7\u201311.","journal-title":"Epilepsia."},{"issue":"4","key":"552_CR45","doi-asserted-by":"crossref","first-page":"243","DOI":"10.1016\/j.pediatrneurol.2013.08.001","volume":"49","author":"H Northrup","year":"2013","unstructured":"Northrup H, Krueger DA, International Tuberous Sclerosis Complex Consensus Group. Tuberous sclerosis complex diagnostic criteria update: recommendations of the 2012 international tuberous sclerosis complex consensus conference. Pediatr Neurol. 2013;49(4):243\u201354.","journal-title":"Pediatr Neurol."},{"issue":"1","key":"552_CR46","doi-asserted-by":"crossref","first-page":"e15","DOI":"10.1159\/000320891","volume":"118","author":"BP Dixon","year":"2011","unstructured":"Dixon BP, Hulbert JC, Bissler JJ. Tuberous sclerosis complex renal disease. Nephron Exp Nephrol. 2011;118(1):e15\u201320.","journal-title":"Nephron Exp Nephrol."},{"key":"552_CR47","doi-asserted-by":"crossref","unstructured":"Kingswood JC, Demuth D, Nasuti P, Lucchese L, Gray E, Magestro M. Real-world assessment of renal involvement in tuberous sclerosis complex (TSC) patients in the United Kingdom (UK) [abstract 318]. Eur Urol Suppl. 2014;13(1):e318\u2013a.","DOI":"10.1016\/S1569-9056(14)60313-4"},{"issue":"6","key":"552_CR48","doi-asserted-by":"crossref","first-page":"1203","DOI":"10.1093\/ndt\/gfu013","volume":"29","author":"JC Kingswood","year":"2014","unstructured":"Kingswood JC, Jozwiak S, Belousova ED, Frost MD, Kuperman RA, Bebin EM, et al. The effect of everolimus on renal angiomyolipoma in patients with tuberous sclerosis complex being treated for subependymal giant cell astrocytoma: subgroup results from the randomized, placebo-controlled, Phase 3 trial EXIST-1. Nephrol Dial Transplant. 2014;29(6):1203\u201310.","journal-title":"Nephrol Dial Transplant."},{"issue":"1","key":"552_CR49","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1093\/ndt\/gfv249","volume":"31","author":"JJ Bissler","year":"2016","unstructured":"Bissler JJ, Kingswood JC, Radzikowska E, Zonnenberg BA, Frost M, Belousova E, et al. Everoliums for renal angiomyolipoma in patients with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis: extension of a randomized controlled trial. Nephrol Dial Transplant. 2016;31(1):111\u20139.","journal-title":"Nephrol Dial Transplant."},{"issue":"3","key":"552_CR50","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1016\/j.trre.2014.03.002","volume":"28","author":"B Kaplan","year":"2014","unstructured":"Kaplan B, Qazi Y, Wellen JR. Strategies for the management of adverse events associated with mTOR inhibitors. Transplant Rev (Orlando). 2014;28(3):126\u201333.","journal-title":"Transplant Rev (Orlando)."},{"issue":"4","key":"552_CR51","doi-asserted-by":"crossref","first-page":"808","DOI":"10.1093\/annonc\/mdu009","volume":"25","author":"HS Rugo","year":"2014","unstructured":"Rugo HS, Pritchard KI, Gnant M, Noguchi S, Piccart M, Hortobagyi G, et al. Incidence and time course of everolimus-related adverse events in postmenopausal women with hormone receptor-positive advanced breast cancer: insights from BOLERO-2. Ann Oncol. 2014;25(4):808\u201315.","journal-title":"Ann Oncol."},{"key":"552_CR52","unstructured":"Bissler JJ, Kingswood JC, Zonnenberg BA, Frost M, Belousova E, Sauter M, et al. Effect of everolimus on renal function in patients with tuberous sclerosis complex (TSC): results from EXIST-1 and EXIST-2 [abstract TO006]. Presented at the 51st ERA-EDTA Congress, 31 May to 3 June 2014, Amsterdam, The Netherlands."},{"issue":"3","key":"552_CR53","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1093\/hmg\/ddp483","volume":"19","author":"D Adhikari","year":"2010","unstructured":"Adhikari D, Zheng W, Shen Y, Gorre N, H\u00e4m\u00e4l\u00e4inen T, Cooney AJ, et al. Tsc\/mTORC1 signaling in oocytes governs the quiescence and activation of primordial follicles. Hum Mol Genet. 2010;19(3):397\u2013410.","journal-title":"Hum Mol Genet."},{"issue":"10","key":"552_CR54","doi-asserted-by":"crossref","first-page":"e45868","DOI":"10.1371\/journal.pone.0045868","volume":"7","author":"M Braun","year":"2012","unstructured":"Braun M, Young J, Reiner CS, Poster D, Krauer F, Kistler AD, et al. Low-dose oral sirolimus and the risk of menstrual-cycle disturbances and ovarian cysts: analysis of the randomized controlled SUISSE ADPKD trial. PLoS One. 2012;7(10):e45868.","journal-title":"PLoS One."},{"issue":"9","key":"552_CR55","doi-asserted-by":"crossref","first-page":"934","DOI":"10.1038\/ki.2010.269","volume":"78","author":"GH Struijk","year":"2010","unstructured":"Struijk GH, Minnee RC, Koch SD, Zwinderman AH, van Donselaar-van der Pant KA, Idu MM, et al. Maintenance immunosuppressive therapy with everolimus preserves humoral immune responses. Kidney Int. 2010;78(9):934\u201340.","journal-title":"Kidney Int."},{"key":"552_CR56","unstructured":"ClinicalTrials.gov identifier: NCT01954693. A study of everolimus in the treatment of neurocognitive problems in tuberous sclerosis (TRON). Accessed 1 Sept 2014."},{"key":"552_CR57","unstructured":"ClinicalTrials.gov identifier: NCT01730209. Efficacy of RAD001\/everolimus in autism and neuropsychological deficits in children with tuberous sclerosis complex (RAPIT). Accessed 1 Sept 2014."},{"key":"552_CR58","unstructured":"ClinicalTrials.gov identifier: NCT01929642. Rapalogues for autism phenotype in TSC: a feasibility study (RAPT). Accessed 26 Jan 2016."},{"key":"552_CR59","unstructured":"ClinicalTrials.gov identifier: NCT02634931. Long-term trial of topical sirolimus to angiofibroma in patient with tuberous sclerosis complex. Accessed 26 Jan 2016."},{"key":"552_CR60","unstructured":"ClinicalTrials.gov identifier: NCT02635789. Phase III trial of topical formulation of sirolimus to skin lesions in patients with tuberous sclerosis complex (TSC). Accessed 26 Jan 2016."},{"issue":"9","key":"552_CR61","doi-asserted-by":"crossref","first-page":"182","DOI":"10.1186\/s13023-014-0182-9","volume":"26","author":"JC Kingswood","year":"2014","unstructured":"Kingswood JC, Bruzzi P, Curatolo P, de Vries PJ, Fladrowski C, Hertzberg C, et al. TOSCA\u2014first international registry to address knowledge gaps in the natural history and management of tuberous sclerosis complex. Orphanet J Rare Dis. 2014;26(9):182. doi: 10.1186\/s13023-014-0182-9 .","journal-title":"Orphanet J Rare Dis."}],"container-title":["Drugs"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-016-0552-9.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/article\/10.1007\/s40265-016-0552-9\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-016-0552-9","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,9,5]],"date-time":"2019-09-05T03:22:30Z","timestamp":1567653750000},"score":1,"resource":{"primary":{"URL":"http:\/\/link.springer.com\/10.1007\/s40265-016-0552-9"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,2,29]]},"references-count":61,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2016,4]]}},"alternative-id":["552"],"URL":"https:\/\/doi.org\/10.1007\/s40265-016-0552-9","relation":{},"ISSN":["0012-6667","1179-1950"],"issn-type":[{"value":"0012-6667","type":"print"},{"value":"1179-1950","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,2,29]]}}}