A split face randomized control study comparing the efficacy and safety of Carbon Dioxide laser treatment versus Radiofrequency ablation in the treatment of facial angiofibromas in patients of Tuberous sclerosis

开始日期2017-05-02

申办/合作机构

All India Institute of Medical Sciences (Bhopal)

NCT03140449 / Completed临床3期IIT

Study of Combination Therapy With Topical Rapamycin and Calcitriol for Cutaneous Lesions of Tuberous Sclerosis: A Double-blind Randomized Controlled Trial

The aim of the study is to determine the effect and safety of topical rapamycin or calcitriol and their combination for the treatment of TSC-associated facial angiofibroma.
Methods: A total of 52 TSC patients including 20 male and 32 female subjects were recruited, and 50 of them completed the period 1 study. In period 1, topical rapamycin (0.1%) or calcitriol (3 mcg/g) single-agent therapy versus their combination were applied twice a day by a left-right randomized, split-face comparison for 12 weeks. The primary end point was the reduction of facial angiofibroma severity index (FASI) for the grade of erythema, papule size, elevation and extension of the lesions at week 12. In period 2, the patients entered an open-label study and were reassigned to use the more effective ointment on both cheeks for another 12 weeks (week 13-24). A follow-up FASI analysis for recurrence after drug discontinuance for 12 weeks was also performed (week 36). The secondary end point was the reduction of Visual Analysis Score (VAS) evaluated by the subjects themselves at week 12.

开始日期2013-09-05

申办/合作机构

National Taiwan University Hospital

EUCTR2012-005397-63-ES / Not yet recruitingN/A

An open-label, multi-center, expanded access study of RAD001 in patients with angiomyolipoma associated with tuberous sclerosis complex (TSC)

开始日期2013-04-19

申办/合作机构

Novartis Farmacéutica SA

100 项与结节性硬化相关血管纤维瘤相关的临床结果

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100 项与结节性硬化相关血管纤维瘤相关的转化医学

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0 项与结节性硬化相关血管纤维瘤相关的专利（医药）

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项与结节性硬化相关血管纤维瘤相关的文献（医药）

2024-04-01·Annals of Pharmacotherapy

A Review of Topical Sirolimus for the Treatment of Facial Angiofibromas in Tuberous Sclerosis Complex

Review

作者: Feldman, Steven R. ; Akkurt, Zeynep M. ; Pixley, Jessica N. ; Dao, Diem-Phuong D.

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.

2014-12-01·Clinical and Experimental Dermatology4区 · 医学

Facial Angiofibroma Severity Index (FASI): reliability assessment of a new tool developed to measure severity and responsiveness to therapy in tuberous sclerosis-associated facial angiofibroma

4区 · 医学

Article

作者: Garnacho-Saucedo, G. ; Moreno-Giménez, J. C. ; Llorca, D. ; Salido-Vallejo, R. ; Ruano, J. ; Godoy-Gijón, E. ; Gómez-Fernández, C.

BACKGROUNDTuberous sclerosis complex (TSC) is an autosomal dominant neurocutaneous disorder characterized by the development of multisystem hamartomatous tumours. Topical sirolimus has recently been suggested as a potential treatment for TSC-associated facial angiofibroma (FA).AIMTo validate a reproducible scale created for the assessment of clinical severity and treatment response in these patients.METHODSWe developed a new tool, the Facial Angiofibroma Severity Index (FASI) to evaluate the grade of erythema and the size and extent of FAs. In total, 30 different photographs of patients with TSC were shown to 56 dermatologists at each evaluation. Three evaluations using the same photographs but in a different random order were performed 1 week apart. Test and retest reliability and interobserver reproducibility were determined.RESULTSThere was good agreement between the investigators. Inter-rater reliability showed strong correlations (> 0.98; range 0.97-0.99) with inter-rater correlation coefficients (ICCs) for the FASI. The global estimated kappa coefficient for the degree of intra-rater agreement (test-retest) was 0.94 (range 0.91-0.97).CONCLUSIONSThe FASI is a valid and reliable tool for measuring the clinical severity of TSC-associated FAs, which can be applied in clinical practice to evaluate the response to treatment in these patients.

2012-10-01·Journal of the European Academy of Dermatology and Venereology2区 · 医学

Sustained clinical effectiveness and favorable safety profile of topical sirolimus for tuberous sclerosis – associated facial angiofibroma

2区 · 医学

Article

作者: J. Ruano ; J.C. Moreno-Giménez ; M. Galán-Gutierrez ; G. Garnacho-Saucedo ; R. Salido ; A. Vélez ; I. Cuevas-Asencio

AbstractBackground Tuberous sclerosis complex (TSC) is an autosomal dominant neurocutaneous disorder characterized by the development of multisystem hamartomatous tumours. Facial angiofibroma appears in up to 80% of patients and has a considerable psychological impact. Various invasive procedures have been used, although they show limited effectiveness and potential adverse effects.Objectives To evaluate the sustained clinical benefits and safety profile of topical sirolimus applied to treat facial angiofibromas.Methods This study was a non‐blinded, uncontrolled case‐series comprising 10 patients with TSC‐associated facial angiofibroma that was treated with 0.4% sirolimus ointment 3 times a week for 9 months. Patients were clinically evaluated at baseline and at 6, 12, 24 and 36 weeks. Plasma levels of sirolimus were determined.Results A sustained improvement was observed in erythema and in the size and extension of the lesions as early as the first weeks of treatment. Sirolimus plasma levels remained below detection limits (0.3 ng/mL) in all cases. The formula was well‐tolerated with no local or systemic adverse effects.Conclusions Topical sirolimus seems to be an effective and safe medical alternative to surgery or laser‐based treatments in patients with TSC‐associated facial angiofibromas.