{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,17]],"date-time":"2026-08-17T23:15:14Z","timestamp":1787008514777,"version":"build-2736575974"},"reference-count":49,"publisher":"Oxford University Press (OUP)","issue":"5","license":[{"start":{"date-parts":[[2023,7,18]],"date-time":"2023-07-18T00:00:00Z","timestamp":1689638400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Dermatology Specialties Limited Partnership"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2023,10,25]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Facial angiofibromas (FAs) are a major feature of tuberous sclerosis complex (TSC). Topical rapamycin can successfully treat FAs. A new stabilized cream formulation that protects rapamycin from oxidation has been developed in 0.5% and 1% concentrations.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objectives<\/jats:title>\n                    <jats:p>To assess the efficacy and safety of a novel, stabilized topical rapamycin cream formulation.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This multicentre double-blind randomized placebo-controlled dose\u2013response phase II\/III study with a parallel design included participants aged 6\u201365\u2005years with FAs of mild or moderate severity according to the Investigator\u2019s Global Assessment (IGA) scale. Participants were randomized to one of three treatment arms: topical rapamycin 0.5%, topical rapamycin 1% or placebo. Treatment was applied once daily for 26 weeks. Safety and efficacy measures were assessed at days 14, 56, 98, 140 and 182. The primary endpoint was the percentage of participants achieving IGA scores of \u2018clear\u2019 or \u2018almost clear\u2019 after 26 weeks of treatment. Secondary measures included Facial Angiofibroma Severity Index (FASI) and participant- and clinician-reported percentage-based improvement. Safety measures included the incidence of treatment-emergent adverse events and blood rapamycin concentration changes over time.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Participants (n = 107) were randomized to receive either rapamycin 1% (n = 33), rapamycin 0.5% (n = 36) or placebo (n = 38). All treated participants were included in the final analysis. The percentage of participants with a two-grade IGA improvement was greater in the rapamycin 0.5% treatment group (11%) and rapamycin 1% group (9%) than in the placebo group (5%). However, this was not statistically significant [rapamycin 0.5%: odds ratio (OR) 1.71, 95% confidence interval (CI) 0.36\u20138.18 (P = 0.50); rapamycin 1%: OR 1.68, 95% CI 0.33\u20138.40 (P = 0.53)]. There was a statistically significant difference in the proportion of participants treated with rapamycin cream that achieved at least a one-grade improvement in IGA [rapamycin 0.5%: 56% (OR 4.73, 95% CI 1.59\u201314.10; P = 0.005); rapamycin 1%: 61% (OR 5.14, 95% CI 1.70\u201315.57; P = 0.004); placebo: 24%]. Skin adverse reactions were more common in patients following rapamycin application (64%) vs. placebo (29%).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Both rapamycin cream formulations (0.5% and 1%) were well tolerated, and either strength could lead to clinical benefit in the treatment of FA.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1093\/bjd\/ljad243","type":"journal-article","created":{"date-parts":[[2023,7,17]],"date-time":"2023-07-17T00:17:20Z","timestamp":1689553040000},"page":"520-530","source":"Crossref","is-referenced-by-count":16,"title":["A novel rapamycin cream formulation improves facial angiofibromas associated with tuberous sclerosis complex: a double-blind randomized placebo-controlled trial"],"prefix":"10.1093","volume":"189","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3934-265X","authenticated-orcid":false,"given":"Phillip","family":"Aitken","sequence":"first","affiliation":[{"name":"AFT Pharmaceuticals Ltd , Auckland , New Zealand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6724-7185","authenticated-orcid":false,"given":"Ioana","family":"Stanescu","sequence":"additional","affiliation":[{"name":"AFT Pharmaceuticals Ltd , Auckland , New Zealand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7708-2401","authenticated-orcid":false,"given":"Laura","family":"Boddington","sequence":"additional","affiliation":[{"name":"AFT Pharmaceuticals Ltd , Auckland , New Zealand"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline","family":"Mahon","sequence":"additional","affiliation":[{"name":"Dermatology Department, Christchurch Hospital , Christchurch , 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Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1281-2009","authenticated-orcid":false,"given":"Ester","family":"Moreno-Artero","sequence":"additional","affiliation":[{"name":"Dermatology Department, Cl\u00ednica Universidad de Navarra , Madrid , Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Doris","family":"Trauner","sequence":"additional","affiliation":[{"name":"University of California San Diego Health Sciences, Department of Neurosciences , San Diego, CA , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven T","family":"DeRoos","sequence":"additional","affiliation":[{"name":"Helen DeVos Children\u2019s Hospital , Grand Rapids, MI , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2935-0798","authenticated-orcid":false,"given":"Jasna","family":"Jancic","sequence":"additional","affiliation":[{"name":"Faculty of Medicine, University 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N","family":"Price","sequence":"additional","affiliation":[{"name":"Division of Dermatology, Phoenix Children\u2019s Hospital , Phoenix, AZ , USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2303-1670","authenticated-orcid":false,"given":"Kinga","family":"Hadzsiev","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics, Medical School, University of P\u00e9cs , P\u00e9cs , Hungary"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kate","family":"Riney","sequence":"additional","affiliation":[{"name":"Neurosciences Unit, Queensland Children\u2019s Hospital , South Brisbane, QLD , Australia"},{"name":"Faculty of Medicine, University of Queensland , St Lucia, QLD , Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stacie","family":"Stapleton","sequence":"additional","affiliation":[{"name":"Johns Hopkins All Children\u2019s Hospital , St Petersburg, FL , 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