The TSA gets lots of enquiries about topical sirolimus and we know just how important this is to so many of you. So, we are investing in providing the right resources for you about facial angiofibromas and topical sirolimus.
A facial rash, or facial angiofibromas, is one of the symptoms experienced by many people with TSC. One of the treatments for this is topical sirolimus (rapamycin) – an ointment that is applied directly to the rash. As topical sirolimus is not yet licensed by the European Medicines Agency, funding is not routinely available for it to be prescribed to patients in the UK.
Globally there is growing evidence of the success of using topical sirolimus. This has largely been based on evidence derived from case reports and case series. Although there has been some trial work in the USA, Australia and Japan, that has shown that topical sirolimus is safe to use in patients and resulted improvement of facial angiofibromas.
We are working towards starting a UK scientific trial looking at the effectiveness of topical sirolimus, within the next 12-18 months. In the meantime, some areas of the UK – the TSC Clinic at the Royal United Hospital in Bath, and, Great Ormond Street Hospital, London, have had some success in being able to prescribe the cream and are reporting good results using it with their patient populations.
Dr Lea Solman, Fellow in Paediatric Dermatology, Great Ormond Street Hospital, London shares her experiences of using topical sirolimus in the Summer 2017 edition of Scan, page 18.
And Dr Sam Amin, Research Associate in Tuberous Sclerosis Complex at Bath TSC Clinic, Royal United Hospital, Bath, explains how he has been prescribing the cream, and its successes to date, in the Summer 2017 edition of Scan, on page 19.
You can also watch a video interview between the TSA's Emma Damian-Grint and Dr Sam Amin and Dr Chris Kingswood about the forthcoming trial of topical sirolimus for facial angiofibromas HERE.
And here is a scientific research poster on the same subject.
Topical Sirolimus Poster (2651 KB)
If you are interested in taking part in the forthcoming trial, or would like to share your experiences to date in trying to access topical sirolimus for facial angiofibroma in TSC, then please email firstname.lastname@example.org
or talk to your local TSA Adviser.