Successful everolimus therapy for SEGA in pediatric patients with tuberous sclerosis complex

Child's Nervous System

Volumn 29, Issue 12, 2013, Pages 2301-2305

  Cappellano, A M ^a   Senerchia, A A ^a   Adolfo, F ^a   Paiva, P M ^a   Pinho, R ^a   Covic, A ^a   Cavalheiro, S ^a   Saba, N ^a  

^a FEDERAL UNIVERSITY OF SÃO PAULO   (Brazil)

Author keywords

Everolimus; mTOR; Subependymal giant cell astrocytoma; Tuberous sclerosis complex

Indexed keywords

CARBAMAZEPINE; EVEROLIMUS; OXCARBAZEPINE; PHENOBARBITAL; PHENYTOIN; RISPERIDONE; VALPROIC ACID;

ANTICONVULSANT THERAPY; ARTICLE; CANCER CHEMOTHERAPY; CASE REPORT; CHILD; CHILD BEHAVIOR; COGNITION; COMPUTER ASSISTED TOMOGRAPHY; DIARRHEA; DISEASE CONTROL; DISEASE COURSE; DRUG BLOOD LEVEL; DRUG DOSE INCREASE; DRUG DOSE TITRATION; DRUG RESPONSE; DRUG SUBSTITUTION; DRUG TOLERABILITY; DRUG WITHDRAWAL; ELECTROENCEPHALOGRAPHY; EPILEPTIC DISCHARGE; FEMALE; HUMAN; HYPERTRIGLYCERIDEMIA; INTRACTABLE EPILEPSY; NUCLEAR MAGNETIC RESONANCE IMAGING; PRESCHOOL CHILD; PRIORITY JOURNAL; SEIZURE; STOMATITIS; SUBEPENDYMAL GIANT CELL ASTROCYTOMA; TUBEROUS SCLEROSIS;

ADOLESCENT; ANTINEOPLASTIC AGENTS; ASTROCYTOMA; BRAIN NEOPLASMS; CHILD, PRESCHOOL; FEMALE; HUMANS; MALE; SIROLIMUS; TUBEROUS SCLEROSIS;

EID: 84890560599     PISSN: 02567040     EISSN: 14330350     Source Type: Journal    
DOI: 10.1007/s00381-013-2170-0     Document Type: Article

References (21)

1. Orlova KA, Crino PB (2010) The tuberous sclerosis complex. Ann N Y Acad Sci 1184:87-105
2. Osborne JP, Fryer A, Webb D (1991) Epidemiology of tuberous sclerosis. Ann N Y Acad Sci 615:125-127
3. Crino PB, Nathanson KL, Henske EP (2006) The tuberous sclerosis complex. N Engl J Med 355:1345-1356
4. Curatolo P, Bombardieri R (2008) Tuberous sclerosis. Handb Clin Neurol 87:129-151
5. Curatolo P, Porfirio MC, Manzi B, Seri S (2004) Autism in tuberous sclerosis. Eur J Paediatr Neurol 8:327-332
6. Curatolo P, D'Argenzio L, Cerminara C, Bombardieri R (2008) Management of epilepsy in tuberous sclerosis complex. Expert Rev Neurother 8:457-467
7. Adriaensen ME, Schaefer-Prokop CM, Stijnen T, Duyndam DA, Zonnenberg BA, Prokop M (2009) Prevalence of subependymal giant cell tumors in patients with tuberous sclerosis and a review of the literature. Eur J Neurol 16:691-696
8. Berhouma M (2010) Management of subependymal giant cell tumors in tuberous sclerosis complex: the neurosurgeon's perspective. World J Pediatr 6:103-110
9. Moavero R, Pinci M, Bombardieri R, Curatolo P (2011) The management of subependymal giant cell tumors in tuberous sclerosis: a clinician's perspective. Childs Nerv Syst 27:1203-1210
10. Yalon M, Ben-Sira L, Constantini S, Toren A (2011) Regression of subependymal giant cell astrocytomas with RAD001 (Everolimus) in tuberous sclerosis complex. Childs Nerv Syst 27:179-181
11. Franz DN, Leonard J, Tudor C, Chuck G, Care M, Sethuraman G, Dinopoulos A, Thomas G, Crone KR (2006) Rapamycin causes regression of astrocytomas in tuberous sclerosis complex. Ann Neurol 59:490-498
12. Krueger DA, Care MM, Holland K, Agricola K, Tudor C, Mangeshkar P, Wilson KA, Byars A, Sahmoud T, Franz DN (2010) Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis. N Engl J Med 363:1801-1811
13. Franz DN, Belousova E, Sparagana S, Bebin EM, Frost M, Kuperman R, Witt O, Kohrman MH, Flamini JR, Wu JY, Curatolo P, de Vries PJ, Whittemore VH, Thiele EA, Ford JP, Shah G, Cauwel H, Lebwohl D, Sahmoud T, Jozwiak S (2013) 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 381(9861):125-132
14. Krueger DA, Care MM, Agricola K, Tudor C, Mays M, Franz DN (2013) Everolimus long-term safety and efficacy in subependymal giant cell astrocytoma. Neurology 80:574-580
15. Goh S, Butler W, Thiele EA (2004) Subependymal giant cell tumors in tuberous sclerosis complex. Neurology 63:1457-1461
16. Bollo RJ, Kalhorn SP, Carlson C, Haegeli V, Devinsky O, Weiner HL (2008) Epilepsy surgery and tuberous sclerosis complex: special considerations. Neurosurg Focus 25:E13
17. Franz DN (2011) Everolimus: an mTOR inhibitor for the treatment of tuberous sclerosis. Expert Rev Anticancer Ther 11:1181-1192
18. Jiang T, Jia G, Ma Z, Luo S, Zhang Y (2011) The diagnosis and treatment of subependymal giant cell astrocytoma combined with tuberous sclerosis. Childs Nerv Syst 27:55-62
19. Huang S, Houghton PJ (2001) Mechanisms of resistance to rapamycins. Drug Resist Updat 4:378-391
20. Yu J, Parkhitko A, Henske EP (2011) Autophagy: an "Achilles" heel of tumorigenesis in TSC and LAM. Autophagy 7:1400-1401
21. Lam C, Bouffet E, Tabori U, Mabbott D, Taylor M, Bartels U (2010) Rapamycin (sirolimus) in tuberous sclerosis associated pediatric central nervous system tumors. Pediatr Blood Cancer 54:476-479