PMID- 37386842
OWN - NLM
STAT- MEDLINE
DCOM- 20240315
LR  - 20240315
IS  - 1542-6270 (Electronic)
IS  - 1060-0280 (Linking)
VI  - 58
IP  - 4
DP  - 2024 Apr
TI  - A Review of Topical Sirolimus for the Treatment of Facial Angiofibromas in 
      Tuberous Sclerosis Complex.
PG  - 428-433
LID - 10.1177/10600280231182421 [doi]
AB  - OBJECTIVE: This article assesses the efficacy, safety, pharmacology, and clinical 
      applications of topical sirolimus 0.2% gel for the treatment of tuberous 
      sclerosis complex (TSC)-associated facial angiofibromas. DATA SOURCES: A review 
      of the literature was conducted using the Medline (PubMed) and EMBASE databases 
      using the keywords topical sirolimus, rapamycin, Hyftor, and tuberous sclerosis. 
      STUDY SELECTION AND DATA EXTRACTION: Articles written in English and relevant to 
      the topic were included. DATA SYNTHESIS: In the phase 2 trial, the mean 
      improvement factor, a composite measure of improved tumor size and redness, was 
      achieved in all patient groups (P < 0.001) with significant responses among the 
      adult and pediatric subgroups at week 12. There were no serious adverse events 
      recorded. In the phase 3 trial, 60% of participants responded to treatment in the 
      sirolimus group compared with 0% in the placebo group with different response 
      rates between the adult and pediatric subgroups at week 12. Sirolimus gel had no 
      serious adverse events, and dry skin was the most common adverse reaction. 
      Patients who had completed the 12-week trials were then enrolled in a long-term 
      trial; angiofibromas had response rates of 78.2% to 0.2% sirolimus gel. RELEVANCE 
      TO PATIENT CARE AND CLINICAL PRACTICE IN COMPARISON TO EXISTING DRUGS: Topical 
      sirolimus 0.2% is a first-in-class, newly Food and Drug Administration 
      (FDA)-approved, mammalian target of rapamycin (mTOR) inhibitor that is a 
      promising and safe, noninvasive alternative to surgical procedures for 
      TSC-associated angiofibromas. CONCLUSIONS: Topical sirolimus 0.2% gel is a 
      moderately effective treatment for TSC-associated facial angiofibromas with an 
      adequate safety profile.
FAU - Dao, Diem-Phuong D
AU  - Dao DD
AD  - Center for Dermatology Research, Department of Dermatology, Wake Forest 
      University School of Medicine, Winston-Salem, NC, USA.
FAU - Pixley, Jessica N
AU  - Pixley JN
AUID- ORCID: 0000-0001-9049-1808
AD  - Center for Dermatology Research, Department of Dermatology, Wake Forest 
      University School of Medicine, Winston-Salem, NC, USA.
FAU - Akkurt, Zeynep M
AU  - Akkurt ZM
AD  - Center for Dermatology Research, Department of Dermatology, Wake Forest 
      University School of Medicine, Winston-Salem, NC, USA.
FAU - Feldman, Steven R
AU  - Feldman SR
AUID- ORCID: 0000-0002-0090-6289
AD  - Center for Dermatology Research, Department of Dermatology, Wake Forest 
      University School of Medicine, Winston-Salem, NC, USA.
AD  - Department of Pathology, Wake Forest University School of Medicine, 
      Winston-Salem, NC, USA.
AD  - Department of Social Sciences & Health Policy, Wake Forest University School of 
      Medicine, Winston-Salem, NC, USA.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20230629
PL  - United States
TA  - Ann Pharmacother
JT  - The Annals of pharmacotherapy
JID - 9203131
RN  - 0 (Immunosuppressive Agents)
RN  - W36ZG6FT64 (Sirolimus)
RN  - 0 (Gels)
SB  - IM
MH  - Adult
MH  - Humans
MH  - Child
MH  - *Tuberous Sclerosis/complications/drug therapy/pathology
MH  - *Angiofibroma/drug therapy/etiology
MH  - *Facial Neoplasms/etiology/chemically induced
MH  - Immunosuppressive Agents
MH  - Sirolimus/adverse effects
MH  - Gels/therapeutic use
OTO - NOTNLM
OT  - Hyftor
OT  - angiofibroma
OT  - rapamycin
OT  - sirolimus
OT  - tuberous sclerosis
COIS- Declaration of Conflicting InterestsThe authors declared the following potential 
      conflicts of interest with respect to the research, authorship, and/or 
      publication of this article: Dr SRF has received research, speaking and/or 
      consulting support from AbbVie, Accordant, Almirall, Alvotech, Amgen, Arcutis, 
      Arena, Argenx, Biocon, Boehringer Ingelheim, Bristol-Myers Squibb, Dermavant, Eli 
      Lilly and Company, Eurofins, Forte, Galderma, Helsinn, Janssen, Leo Pharma, 
      Micreos, Mylan, Novartis, Ono, Ortho Dermatology, Pfizer, Regeneron, Samsung, 
      Sanofi, Sun Pharma, UCB, Verrica, Voluntis, and vTv Therapeutics. He is founder 
      and part owner of Causa Research and holds stock in Sensal Health.
EDAT- 2023/06/30 06:43
MHDA- 2024/03/15 06:44
CRDT- 2023/06/30 02:36
PHST- 2024/03/15 06:44 [medline]
PHST- 2023/06/30 06:43 [pubmed]
PHST- 2023/06/30 02:36 [entrez]
AID - 10.1177/10600280231182421 [doi]
PST - ppublish
SO  - Ann Pharmacother. 2024 Apr;58(4):428-433. doi: 10.1177/10600280231182421. Epub 
      2023 Jun 29.