Erratum: 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 (The Lancet (2013) 381(9861) (125–132) (S0140673612611349) (10.1016/S0140-6736(12)61134-9));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

The Lancet

Volumn 381, Issue 9861, 2013, Pages 125-132

  Franz, David Neal ^a   Belousova, Elena ^b   Sparagana, Steven ^c   Bebin, E Martina ^d   Frost, Michael ^e   Kuperman, Rachel ^f   Witt, Olaf ^g   Kohrman, Michael H ^h   Flamini, J Robert ⁱ   Wu, Joyce Y ^j   Curatolo, Paolo ^k   De Vries, Petrus J ^l   Whittemore, Vicky H ^m   Thiele, Elizabeth A ⁿ   Ford, James P ^o   Shah, Gaurav ^o   Cauwel, Helene ^o   Lebwohl, David ^o   Sahmoud, Tarek ^o   Jozwiak, Sergiusz ^p  

^a CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^b Moscow Research Institute of Paediatry and Paediatric Surgery   (Russian Federation)

^c TEXAS SCOTTISH RITE HOSPITAL FOR CHILDREN   (United States)

^d UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^e MINNESOTA EPILEPSY GROUP   (United States)

^f CHILDREN S HOSPITAL OAKLAND   (United States)

^g UNIVERSITY OF HEIDELBERG   (Germany)

^h UNIVERSITY OF CHICAGO   (United States)

ⁱ CHILDREN'S HEALTHCARE OF ATLANTA   (United States)

^j MATTEL CHILDREN S HOSPITAL   (United States)

^k UNIVERSITY OF ROME TOR VERGATA   (Italy)

^l UNIVERSITY OF CAPE TOWN   (South Africa)

^m NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE   (United States)

ⁿ Pediatric Epilepsy Program   (United States)

^o NOVARTIS PHARMA AG   (Switzerland)

^p CHILDREN'S MEMORIAL HEALTH INSTITUTE   (Poland)

more..

Author keywords

[No Author keywords available]

Indexed keywords

EVEROLIMUS; PLACEBO;

ADOLESCENT; ADULT; AMENORRHEA; ARTICLE; AUSTRALIA; BELGIUM; CANADA; CHILD; CONTROLLED STUDY; CONVULSION; DEATH; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; DRUG WITHDRAWAL; FEMALE; FEVER; GERMANY; HERPES ZOSTER; HUMAN; HYDROCEPHALUS; INFANT; INTERSTITIAL PNEUMONIA; ITALY; MAJOR CLINICAL STUDY; MALE; MOUTH ULCER; MULTICENTER STUDY; NETHERLANDS; NEWBORN; PHASE 3 CLINICAL TRIAL; PNEUMONIA; POLAND; PRESCHOOL CHILD; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RUSSIAN FEDERATION; SCHOOL CHILD; SIDE EFFECT; STOMATITIS; SUBEPENDYMAL GIANT CELL ASTROCYTOMA; TREATMENT RESPONSE; TUBEROUS SCLEROSIS; UNITED KINGDOM; UNITED STATES; UPPER RESPIRATORY TRACT INFECTION;

EID: 84872088946     PISSN: 01406736     EISSN: 1474547X     Source Type: Journal    
DOI: 10.1016/S0140-6736(13)60050-1     Document Type: Erratum

References (32)

1. Franz DN, Bissler JJ, McCormack FX. Tuberous sclerosis complex: neurological, renal and pulmonary manifestations. Neuropediatrics 2010; 41: 199-208.
2. Borkowska J, Schwartz RA, Kotulska K, Jozwiak S. Tuberous sclerosis complex: tumors and tumorigenesis. Int J Dermatol 2011; 50: 13-20.
3. Curatolo P, Bombardieri R, Jozwiak S. Tuberous sclerosis. Lancet 2008; 372: 657-68.
4. Adriaensen ME, Schaefer-Prokop CM, Stijnen T, Duyndam DA, Zonnenberg BA, Prokop M. Prevalence of subependymal giant cell tumors in patients with tuberous sclerosis and a review of the literature. Eur J Neurol 2009; 16: 691-96.
5. Torres OA, Roach ES, Delgado MR, et al. Early diagnosis of subependymal giant cell astrocytoma in patients with tuberous sclerosis. J Child Neurol 1998; 13: 173-77.
6. Goh S, Butler W, Thiele EA. Subependymal giant cell tumors in tuberous sclerosis complex. Neurology 2004; 63: 1457-61.
7. Moavero R, Pinci M, Bombardieri R, Curatolo P. The management of subependymal giant cell tumors in tuberous sclerosis: a clinician's perspective. Childs Nerv Syst 2011; 27: 1203-10.
8. Sun P, Krueger D, Liu J, Guo A, Rogerio J, Kohrman M. Outcomes of resecting subependymal giant cell astrocytoma (SEGA) among patients with SEGA-related tuberous sclerosis complex: a national claims database analysis. Curr Med Res Opin 2012; 28: 1-7.
9. Roszkowski M, Drabik K, Barszcz S, Jozwiak S. Surgical treatment of intraventricular tumors associated with tuberous sclerosis. Childs Nerv Syst 1995; 11: 335-39.
10. Huang J, Manning BD. The TSC1-TSC2 complex: a molecular switchboard controlling cell growth. Biochem J 2008; 412: 179-90.
11. Dabora SL, Jozwiak S, Franz DN, et al. Mutational analysis in a cohort of 224 tuberous sclerosis patients indicates increased severity of TSC2, compared with TSC1, disease in multiple organs. Am J Hum Genet 2001; 68: 64-80.
12. Sancak O, Nellist M, Goedbloed M, et al. Mutational analysis of the TSC1 and TSC2 genes in a diagnostic setting: genotype: phenotype correlations and comparison of diagnostic DNA techniques in tuberous sclerosis complex. Eur J Hum Genet 2005; 13: 731-41.
13. Au KS, Williams AT, Roach ES, et al. Genotype/phenotype correlation in 325 individuals referred for a diagnosis of tuberous sclerosis complex in the United States. Genet Med 2007; 9: 88-100.
14. Wienecke R, Fackler I, Linsenmaier U, Mayer K, Licht T, Kretzler M. Antitumoral activity of rapamycin in renal angiomyolipoma associated with tuberous sclerosis complex. Am J Kidney Dis 2006; 48: e27-29.
15. Franz DN, Leonard J, Tudor C, et al. Rapamycin causes regression of astrocytomas in tuberous sclerosis complex. Ann Neurol 2006; 59: 490-98.
16. Bissler JJ, McCormack FX, Young LR, et al. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med 2008; 358: 140-51.
17. Hofbauer GF, Marcollo-Pini A, Corsenca A, et al. The mTOR inhibitor rapamycin significantly improves facial angiofibroma lesions in a patient with tuberous sclerosis. Br J Dermatol 2008; 159: 473-75.
18. Koenig MK, Butler IJ, Northrup H. Regression of subependymal giant cell astrocytoma with rapamycin in tuberous sclerosis complex. J Child Neurol 2008; 23: 1238-39.
19. Morton JM, McLean C, Booth SS, Snell GI, Whitford HM. Regression of pulmonary lymphangioleiomyomatosis (PLAM)-associated retroperitoneal angiomyolipoma post-lung transplantation with rapamycin treatment. J Heart Lung Transplant 2008; 27: 462-65.
20. Taille C, Debray MP, Crestani B. Sirolimus treatment for pulmonary lymphangioleiomyomatosis. Ann Intern Med 2007; 146: 687-88.
21. Krueger DA, Care MM, Holland K, et al. Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis. N Engl J Med 2010; 363: 1801-11.
22. Roach ES, Gomez MR, Northrup H. Tuberous sclerosis complex consensus conference: revised clinical diagnostic criteria. J Child Neurol 1998; 13: 624-28.
23. Hyman MH, Whittemore VH. National Institutes of Health consensus conference: tuberous sclerosis complex. Arch Neurol 2000; 57: 662-65.
24. Fouladi M, Laningham F, Wu J, et al. Phase I study of everolimus in pediatric patients with refractory solid tumors. J Clin Oncol 2007; 25: 4806-12.
25. Duvic M, Martin AG, Kim Y, et al. Phase 2 and 3 clinical trial or oral bexarotene (Targretin capsules) for the treatment of refractory or persistent early-stage cutaneous T-cell lymphoma. Arch Dermatol 2001; 137: 581-93.
26. Heald P, Mehlmauer M, Martin AG, Crowley CA, Yocum RC, Reich SD. Topical bexarotene therapy for patients with refractory or persistent early-stage cutaneous T-cell lymphoma: results of the phase III clinical trial. J Am Acad Dermatol 2003; 49: 801-15.
27. NCI. Cancer Therapy Evaluation Program. Common terminology criteria for adverse events, version 3.0. Bethesda, MD: National Cancer Institute, 2006.
28. Stokes ME, Davis CS, Koch GG. Categorical data analysis using the SAS system. 2nd edn. Cary, NC: John Wiley and Sons, 2000.
29. de Ribaupierre S, Dorfmuller G, Bulteau C, et al. Subependymal giant-cell astrocytomas in pediatric tuberous sclerosis disease: when should we operate? Neurosurgery 2007; 60: 83-89.
30. Yalon M, Ben-Sira L, Constantini S, Toren A. Regression of subependymal giant cell astrocytomas with RAD001 (Everolimus) in tuberous sclerosis complex. Childs Nerv Syst 2011; 27: 179-81.
31. Perek-Polnik M, Jozwiak S, Jurkiewicz E, Perek D, Kotulska K. Eff ective 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: 83-85.
32. Roa J, Tena-Sempere M. Energy balance and puberty onset: emerging role of central mTOR signaling. Trends Endocrinol Metab 2010; 21: 519-28.