Improvement of tuberous sclerosis complex (TSC) skin tumors during long-term treatment with oral sirolimus

Journal of the American Academy of Dermatology

Volumn 73, Issue 5, 2015, Pages 802-808

  Nathan, Neera ^a,b   Wang, Ji An ^a   Li, Shaowei ^a   Cowen, Edward W ^c   Haughey, Mary ^b   Moss, Joel ^b   Darling, Thomas N ^a  

^a UNIFORMED SERVICES UNIVERSITY OF THE HEALTH SCIENCES   (United States)

^b NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^c NATIONAL CANCER INSTITUTE   (United States)

Author keywords

angiofibromas; lymphangioleiomyomatosis; mechanistic target of rapamycin inhibitor; shagreen patch; sirolimus; tuberous sclerosis complex; ungual fibroma

Indexed keywords

RAPAMYCIN; ANTINEOPLASTIC ANTIBIOTIC; PROTEIN S6;

ACNE; ADULT; CLINICAL ARTICLE; CONFERENCE PAPER; DRUG INDUCED HEADACHE; DRUG RESPONSE; FEMALE; FIBROMA; FOOT EDEMA; HEMANGIOFIBROMA; HUMAN; HUMAN CELL; HUMAN TISSUE; HYPERTENSION; LONG TERM CARE; LYMPHANGIOLEIOMYOMATOSIS; MOUTH ULCER; NAIL DISEASE; PHOTOGRAPHY; PRIORITY JOURNAL; RETROSPECTIVE STUDY; SIDE EFFECT; SKIN TUMOR; SYSTEMIC THERAPY; TREATMENT DURATION; TUBEROUS SCLEROSIS COMPLEX SKIN TUMOR; ACNE VULGARIS; CHEMICALLY INDUCED; DRUG ADMINISTRATION; MIDDLE AGED; ORAL DRUG ADMINISTRATION; SKIN NEOPLASMS; TREATMENT OUTCOME; TUBEROUS SCLEROSIS;

ACNE VULGARIS; ADMINISTRATION, ORAL; ADULT; ANTIBIOTICS, ANTINEOPLASTIC; DRUG ADMINISTRATION SCHEDULE; FEMALE; HUMANS; MIDDLE AGED; ORAL ULCER; RETROSPECTIVE STUDIES; RIBOSOMAL PROTEIN S6; SIROLIMUS; SKIN NEOPLASMS; TREATMENT OUTCOME; TUBEROUS SCLEROSIS;

EID: 84944354899     PISSN: 01909622     EISSN: 10976787     Source Type: Journal    
DOI: 10.1016/j.jaad.2015.07.018     Document Type: Conference Paper

References (26)

1. J.J. Bissler, F.X. McCormack, L.R. Young, and et al. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis N Engl J Med 358 2008 140 151
2. D.A. Krueger, M.M. Care, K. Holland, and et al. Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis N Engl J Med 363 2010 1801 1811
3. F.X. McCormack, Y. Inoue, J. Moss, and et al. Efficacy and safety of sirolimus in lymphangioleiomyomatosis N Engl J Med 364 2011 1595 1606
4. D.M. Davies, P.J. de Vries, S.R. Johnson, and et al. Sirolimus therapy for angiomyolipoma in tuberous sclerosis and sporadic lymphangioleiomyomatosis: a phase 2 trial Clin Cancer Res 17 2011 4071 4081
5. A.M. Taveira-DaSilva, O. Hathaway, M. Stylianou, and J. Moss Changes in lung function and chylous effusions in patients with lymphangioleiomyomatosis treated with sirolimus Ann Intern Med 154 2011 797 805 W-292-3
6. S.L. Dabora, D.N. Franz, S. Ashwal, and et al. Multicenter phase 2 trial of sirolimus for tuberous sclerosis: kidney angiomyolipomas and other tumors regress and VEGF- D levels decrease PLoS One 6 2011 e23379
7. D.A. Krueger, M.M. Care, K. Agricola, C. Tudor, M. Mays, and D.N. Franz Everolimus long-term safety and efficacy in subependymal giant cell astrocytoma Neurology 80 2013 574 580
8. D.N. Franz, E. Belousova, S. Sparagana, and et al. Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicenter, randomized, placebo-controlled phase 3 trial Lancet 381 2013 125 132
9. J.J. Bissler, J.C. Kingswood, E. Radzikowska, and et al. Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicenter, randomized, double-blind, placebo-controlled trial Lancet 381 2013 817 824
10. D.A. Krueger, A.A. Wilfong, K. Holland-Bouley, and et al. Everolimus treatment of refractory epilepsy in tuberous sclerosis complex Ann Neurol 74 2013 679 687
11. J.C. Kingswood, S. Jozwiak, E.D. Belousova, and 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 29 2014 1203 1210
12. D.N. Franz, E. Belousova, S. Sparagana, and et al. Everolimus for subependymal giant cell astrocytoma in patients with tuberous sclerosis complex: 2-year open-label extension of the randomized EXIST-1 study Lancet Oncol 15 2014 1513 1520
13. T.N. Darling, J. Moss, and M. Mausner Dermatologic manifestations of tuberous sclerosis complex (TSC) D.J. Kwiatkowski, V.H. Whittemore, E.A. Thiele, Tuberous Sclerosis Complex 2010 Wiley-VCH Weinheim, Germany 285 310
14. H.M. Prince, C. McCormack, G. Ryan, and et al. Bexarotene capsules and gel for previously treated patients with cutaneous T-cell lymphoma: results of the Australian patients treated on phase II trials Australas J Dermatol 42 2001 91 97
15. M. Duvic, A.G. Martin, Y. Kim, and et al. Phase 2 and 3 clinical trial of oral bexarotene (Targretin capsules) for the treatment of refractory or persistent early-stage cutaneous T-cell lymphoma Arch Dermatol 137 2001 581 593
16. S. Li, F. Takeuchi, J.A. Wang, and et al. Mesenchymal-epithelial interactions involving epiregulin in tuberous sclerosis complex hamartomas Proc Natl Acad Sci U S A 105 2008 3539 3544
17. M.E. Tyburczy, J.A. Wang, S. Li, and et al. Sun exposure causes somatic second-hit mutations and angiofibroma development in tuberous sclerosis complex Hum Mol Genet 23 2014 2023 2029
18. H. Northrup, D.A. Krueger International Tuberous Sclerosis Complex Consensus Group Tuberous sclerosis complex diagnostic criteria update: recommendations of the 2012 International Tuberous Sclerosis Complex Consensus Conference Pediatr Neurol 49 2013 243 254
19. J.M. Teng, E.W. Cowen, M. Wataya-Kaneda, and et al. Dermatologic and dental aspects of the 2012 International Tuberous Sclerosis Complex Consensus Statements JAMA Dermatol 150 2014 1095 1101
20. S. Li, R.L. Thangapazham, J.A. Wang, and et al. Human TSC2-null fibroblast-like cells induce hair follicle neogenesis and hamartoma morphogenesis Nat Commun 2 2011 235
21. P. Frost, E. Berlanger, V. Mysore, and et al. Mammalian target of rapamycin inhibitors induce tumor cell apoptosis in vivo primarily by inhibiting VEGF expression and angiogenesis J Oncol 2013 2013 897025
22. S. Jozwiak, R.A. Schwartz, C.K. Janniger, R. Michalowicz, and J. Chmielik Skin lesions in children with tuberous sclerosis complex: their prevalence, natural course, and diagnostic significance Int J Dermatol 37 1998 911 917
23. J.S. Carew, K.R. Kelly, and S.T. Nawrocki Mechanisms of mTOR inhibitor resistance in cancer therapy Target Oncol 6 2011 17 27
24. N. Wagle, B.C. Grabiner, E.M. Van Allen, and et al. Response and acquired resistance to everolimus in anaplastic thyroid cancer N Engl J Med 371 2014 1426 1433
25. J. Tu, R.S. Foster, L.J. Bint, and A.R. Halbert Topical rapamycin for angiofibromas in pediatric patients with tuberous sclerosis: follow-up of a pilot study and promising future directions Australas J Dermatol 55 2014 63 69
26. P.E. Dill, G. De Bernardis, P. Weber, and U. Losch Topical everolimus for facial angiofibromas in the tuberous sclerosis complex. A first case report Pediatr Neurol 51 2014 109 113