A survey of patients with facial angiofibromas associated with tuberous sclerosis complex: Short-, medium- and long-term efficacy and safety of topical rapamycin

IF 3.1 4区医学 Q2 DERMATOLOGY

Annales De Dermatologie Et De Venereologie Pub Date : 2023-12-01 DOI:10.1016/j.annder.2023.06.009

N. Sigg , J. Fouquet , D. Morin , D. Farges , S. Vrignaud , L. Martin

{"title":"A survey of patients with facial angiofibromas associated with tuberous sclerosis complex: Short-, medium- and long-term efficacy and safety of topical rapamycin","authors":"N. Sigg , J. Fouquet , D. Morin , D. Farges , S. Vrignaud , L. Martin","doi":"10.1016/j.annder.2023.06.009","DOIUrl":null,"url":null,"abstract":"<div><h3>Aims</h3><p><span>Topical rapamycin<span> is used to reduce facial angiofibromas<span> in patients with </span></span></span>tuberous sclerosis (TSC). In the absence of a commercially available preparation, numerous formulations have been tested clinically, although only in the short term.</p></div><div><h3>Methods</h3><p>The pharmacy at Angers University Hospital (France) produced a cream formulation that was administered to people presenting this genetic disease. We conducted a questionnaire-based survey among 79 patients with TSC about their perceptions regarding the short-, medium- and long-term efficacy and safety of a topical rapamycin preparation in relation to facial angiofibromas.</p></div><div><h3>Results</h3><p>This formulation was very well tolerated and its efficacy was sustained over the long term with a mean treatment duration of 33 months (extremes 1–60). Efficacy was rated ≥ 8/10 by 67.1% of patients while safety was rated ≥ 8/10 by 84.8% of patients.</p></div><div><h3>Conclusion</h3><p>This survey supports the safety and efficacy of topical rapamycin in the short-, medium- and long-term in the treatment of facial angiofibromas in a cohort of 79 patients with TSC.</p></div>","PeriodicalId":7900,"journal":{"name":"Annales De Dermatologie Et De Venereologie","volume":null,"pages":null},"PeriodicalIF":3.1000,"publicationDate":"2023-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Annales De Dermatologie Et De Venereologie","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0151963823000716","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"DERMATOLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

Aims

Topical rapamycin is used to reduce facial angiofibromas in patients with tuberous sclerosis (TSC). In the absence of a commercially available preparation, numerous formulations have been tested clinically, although only in the short term.

Methods

The pharmacy at Angers University Hospital (France) produced a cream formulation that was administered to people presenting this genetic disease. We conducted a questionnaire-based survey among 79 patients with TSC about their perceptions regarding the short-, medium- and long-term efficacy and safety of a topical rapamycin preparation in relation to facial angiofibromas.

Results

This formulation was very well tolerated and its efficacy was sustained over the long term with a mean treatment duration of 33 months (extremes 1–60). Efficacy was rated ≥ 8/10 by 67.1% of patients while safety was rated ≥ 8/10 by 84.8% of patients.

Conclusion

This survey supports the safety and efficacy of topical rapamycin in the short-, medium- and long-term in the treatment of facial angiofibromas in a cohort of 79 patients with TSC.

查看原文本刊更多论文

面部血管纤维瘤伴结节性硬化症患者的调查：局部雷帕霉素的短期、中期和长期疗效和安全性。

目的：局部雷帕霉素用于减少结节性硬化（TSC）患者的面部血管纤维瘤。在缺乏商业可用制剂的情况下，许多制剂已经进行了临床试验，尽管只是在短期内。方法：法国昂热大学医院的药房生产了一种乳膏配方，用于患有这种遗传性疾病的人。我们对79名TSC患者进行了一项基于问卷的调查，了解他们对雷帕霉素外用制剂治疗面部血管纤维瘤的短期、中期和长期疗效和安全性的看法。结果：该制剂具有良好的耐受性，其疗效长期持续，平均治疗时间为33 月（极端1-60）。对疗效进行了评级 ≥ 在安全性评定的情况下，67.1%的患者使用8/10 ≥ 8/10，占84.8%。结论：在79例TSC患者中，本调查支持局部雷帕霉素治疗面部血管纤维瘤的短期、中期和长期安全性和有效性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Annales De Dermatologie Et De Venereologie 医学-皮肤病学

CiteScore

1.40

自引率

0.00%

发文量

审稿时长

81 days

期刊介绍： Les Annales de dermatologie sont le rendez-vous mensuel incontournable de toute la dermatologie francophone, grâce à leur comité de rédaction qui assure une sélection rigoureuse des articles selon les normes de l''édition scientifique internationale. Une revue didactique, véritable aide à la pratique médicale quotidienne Pour compléter et enrichir la partie scientifique, la rubrique Formation médicale continue propose aux lecteurs des textes didactiques et interactifs (Cas pour diagnostic, Notes de pharmacovigilance, la Question du praticien, Dermatologie chirurgicale, la Sélection bibliographique du mois...) qui les font bénéficier d''une formation post-universitaire diversifiée et de qualité. La revue consacre également un espace pour la publication de questions des lecteurs auxquelles des experts apportent une réponse.