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Sirolimus Gel Treatment vs Placebo for Facial Angiofibromas in Patients With Tuberous Sclerosis Complex: A Randomized Clinical Trial.

Author: FujitaYasuyuki, FukaiKazuyoshi, HamadaIzumi, HioTaihei, MurotaHiroyuki, NagaiHiroshi, NiizekiHironori, OgaiMasaaki, OhnoYuuki, ShimizuKenji, Wataya-KanedaMari, YokozekiHiroo, YoshidaYuichi

Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

Tuberous sclerosis complex (TSC) is a genetic disorder that causes tumors to grow in various organs, including the skin. Many people with TSC have facial growths (angiofibromas) that can be disfiguring. Because effective treatments that are less invasive than surgery or laser therapy have been lacking, many people have gone untreated.
Paper Details　
Original Abstract of the Article :
Most patients with tuberous sclerosis complex (TSC), an autosomal-dominant disorder that is caused by the constitutive activation of mammalian target of rapamycin, experience disfigurement caused by skin lesions involving facial angiofibromas. Many have been left untreated because of a lack of thera...See full text at original site
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引用元：
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6128500/

データ提供：米国国立医学図書館（NLM）

A New Approach to Treating Facial Angiofibromas

The [tuberous sclerosis complex] is a challenging genetic condition, often causing facial angiofibromas, benign tumors that can lead to disfigurement. This study explores the potential of sirolimus gel, a [mammalian target of rapamycin inhibitor], to treat facial angiofibromas in patients with tuberous sclerosis complex. The researchers conducted a [randomized clinical trial] comparing the effects of sirolimus gel to placebo. The results demonstrate the effectiveness of sirolimus gel in reducing the size and prominence of facial angiofibromas, offering a less invasive alternative to surgery and laser treatment.

A Promising New Treatment Option

The study reveals that sirolimus gel significantly reduced the size and prominence of facial angiofibromas in patients with tuberous sclerosis complex. This finding is significant, offering a less invasive and potentially more effective treatment option for these challenging skin lesions.

Navigating the Desert of Tuberous Sclerosis Complex

This study offers a glimmer of hope for patients with tuberous sclerosis complex, providing a new approach to managing facial angiofibromas. Sirolimus gel, with its less invasive nature, may offer a more patient-friendly and effective treatment option. As always, it's essential to have a thorough discussion with a healthcare professional to determine the most appropriate treatment for each individual.

Dr.Camel's Conclusion

Sirolimus gel, like a camel who navigates the desert with ease, offers a new approach to managing facial angiofibromas in patients with tuberous sclerosis complex. This study is a testament to the power of ongoing research in finding innovative solutions for complex medical conditions. As we journey through the vast desert of medical knowledge, it's essential to continue exploring new avenues for treating challenging conditions and improving patient outcomes.

Date :
  1. Date Completed 2019-09-16
  2. Date Revised 2019-09-16
Further Info :

Pubmed ID

29800026

DOI: Digital Object Identifier

PMC6128500

SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

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