Topical rapamycin therapy to alleviate the cutaneous manifestations of tuberous sclerosis complex: A double-blind, randomized, controlled trial to evaluate the safety and efficacy of topically applied rapamycin

Drugs in R and D

Volumn 12, Issue 3, 2012, Pages 121-126

  Koenig, Mary Kay ^a   Hebert, Adelaide A ^a   Roberson, Joan ^a   Samuels, Joshua ^a   Slopis, John ^b   Woerner, Audrey ^c   Northrup, Hope ^a  

^a UNIVERSITY OF TEXAS MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

^c HARVARD MEDICAL SCHOOL   (United States)

Author keywords

Fibroma; Sebaceous adenoma; Sirolimus; Topical; Tuberous sclerosis

Indexed keywords

RAPAMYCIN;

ADOLESCENT; ADULT; ARTICLE; CLINICAL ARTICLE; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; DRUG ABSORPTION; DRUG BLOOD LEVEL; DRUG EFFICACY; DRUG SAFETY; ERYTHROCYTE COUNT; FACE ANGIOFIBROMA; FACE TUMOR; FEMALE; HEMANGIOFIBROMA; HUMAN; LEUKOCYTE COUNT; MALE; PRIORITY JOURNAL; PROSPECTIVE STUDY; RANDOMIZED CONTROLLED TRIAL; THROMBOCYTE COUNT; TREATMENT OUTCOME; TUBEROUS SCLEROSIS;

ADMINISTRATION, TOPICAL; ADOLESCENT; ADULT; ANGIOFIBROMA; DOUBLE-BLIND METHOD; FACIAL NEOPLASMS; FEMALE; HUMANS; MALE; PROSPECTIVE STUDIES; SIROLIMUS; TUBEROUS SCLEROSIS; YOUNG ADULT;

EID: 84865839005     PISSN: 11745886     EISSN: 11796901     Source Type: Journal    
DOI: 10.2165/11634580-000000000-00000     Document Type: Article

References (27)

1. Schwartz RA, Fernandez G, Kotulska K, et al. Tuberous sclerosis complex: advances in diagnosis, genetics, and management. J Am Acad Dermatol 2007; 57: 189-202
2. Kane Y. The ''bumps'' on my face. J Am Acad Dermatol 2004; 51: S11-2
3. El-MusaKA, Shehadi RS, Shehadi S. Extensive facial adenoma sebaceum: successful treatment with mechanical dermabrasion: case report. Brit J Plast Surg 2005; 58: 1143-7
4. Finch TM, Hindson C, Cotterill JA. Successful treatment of adenoma sebaceum with the potassium titanyl phosphate laser. Clin Exp Dermatol 1998; 23: 201-3
5. Hori K, Soejima K, Nozaki M, et al. Treatment of facial angiofibromas of tuberous sclerosis using cultured epithelial autografts. Ann Plast Surg 2006; 57: 415-7
6. Kaufman AJ, Grekin RC, Geisse JK, et al. Treatment of adenoma sebaceum with the copper vapor laser. J Am Acad Dermatol 1995; 33: 770-4
7. Papadavid E, Markey A, Bellaney G, et al. Carbon dioxide and pulsed dye laser treatment of angiofibromas in 29 patients with tuberous sclerosis. Brit J Plast Surg 2002; 147: 337-42
8. Verheyden CN. Treatment of the facial angiofibromas of tuberous sclerosis. Plast Reconstr Surg 1996; 98: 777-83
9. Bittencourt RC, Huilgol SC, Seed PT, et al. Treatment of angiofibromas with scanning carbon dioxide laser: a clinicopathologic study with long-term follow-up. J Am Acad Dermatol 2001; 45: 731-5
10. Li S, Takeuchi F, Wang J, et al. Mesenchymal-epithelial interactions involving epiregulin in tuberous sclerosis complex hamaratomas. PNAS 2008; 105: 3539-44
11. Darling TN. Hamartomas and tubers from defects in harmartin- tuberin. J Am Acad Dermatol 2004; 51: S9-11
12. Bissler JJ, McCormack FX, Young LR, et al. Rapamycin for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. New Engl J Med 2008; 358: 140-51
13. Franz DN, Leonard J, Tudor C, et al. Rapamycin causes regression of astrocytomas in tuberous sclerosis complex. Ann Neurol 2006; 59: 490-8
14. Koenig MK, Butler IJ, Northrup H. Regression of subependymal giant cells astrocytoma with rapamycin in tuberous sclerosis complex. J Child Neurol 2008; 23: 1238-9
15. Glasgow CG, Steagall WK, Taveira-DaSilva A, et al. Lymphangioleiomyomatosis (LAM): molecular insights lead to targeted therapies. Resp Med 2010; 104: S45-58
16. Paghdal KV, Schwartz RA. Sirolimus (rapamycin): from the soil of Easter Island to a bright future. J Am Acad Dermatol 2007; 57: 1046-50
17. Roach ES, DiMario FJ, Kandt RS, et al. Tuberous sclerosis consensus conference: recommendations for diagnostic evaluation. National Tuberous Sclerosis Association. J Child Neurol 1999; 14: 401-7
18. Foster RS, Bint LJ, Halbert AR. Topical 0.1% rapamycin for angiofibromas in paediatric patients with tuberous sclerosis: a pilot study of four patients. Australas J Dermatol 2012; 53: 52-6
19. Truchuelo T, Diaz-Ley B, Rios L, et al. Facial angiofibromas treated with topical rapamycin: an excellent choice with fast response. Dermatol Online J 2012; 18: 15
20. Wataya-Kaneda M, Tanaka M, Nakamura A, et al. A novel application of topical rapamycin formulation, an inhibitor of mTOR, for patients with hypomelanotic macules in tuberous sclerosis complex. Arch Dermatol 2012; 148: 138-9
21. DeKlotz CM, Ogram AE, Singh S, et al. Dramatic improvement of facial angiofibromas in tuberous sclerosis with topical rapamycin: optimizing a treatment protocol. Arch Dermatol 2011; 147: 1116-7
22. Salido R, Garnacho-Saucedo G, Cuevas-Asencio I, et al. Sustained clinical effectiveness and favorable safety profile of topical sirolimus for tuberous sclerosis-associated facial angiofibroma. J Eur Acad Dermatol Venereol. Epub 2011 Aug 11
23. Valeron-Almazan P, Vitiello M, Abuchar A, et al. Topical rapamycin solution to treat multiple facial angiofibromas in a patient with tuberous sclerosis. Actas Dermosifiliogr 2012; 103: 165-6
24. Mutizwa MM, Berk DR, Anadkat MJ. Treatment of facial angiofibromas with topical application of oral rapamycin solution (1mg mL-1) in two patients with tuberous sclerosis. Br J Dermatol 2011; 165: 922-3
25. Wataya-Kaneda M, Tanaka M, Nakamura A, et al. A topical combination of rapamycin and tacrolimus for the treatment of angiofibroma due to tuberous sclerosis complex (TSC): a pilot study of nine Japanese patients with TSC of different disease severity. Br J Dermatol 2011; 165: 912-6
26. Kaufman McNamara E, Curtis AR, Fleischer Jr AB. Successful treatment of angiofibromata of tuberous sclerosis complex with rapamycin. J Dermatolog Treat 2012; 23: 46-8
27. Haemel AK, O'Brian AL, Teng JM. Topical rapamycin: a novel approach to facial angiofibromas in tuberous sclerosis. Arch Dermatol 2010; 146: 715-8