A single arm single center exploratory observational phase II study, aimed to detect long term changes in volume of port-wine macrocheilia and color of port-wine stain in patients undergoing surgery and laser therapy during sirolimus treatment. Quality of life and efficacy and safety of sirolimus will be assessed.
A port-wine stain is a reddish or purple birthmark caused by unusual blood vessels. Over time, it can cause nearby tissue, such as the lip, to swell and thicken. This study investigates whether a medicine called sirolimus (Rapamune), taken as a liquid or tablet, can help shrink lip swelling and lighten the birthmark. Participants will receive sirolimus alongside standard laser therapy and surgery. Doctors will follow participants for five years (60 months). They will measure changes in lip size using a gentle measuring tool, record birthmark colour changes, and track overall quality of life using standard health surveys. The trial is open to adults aged 18 and over with this type of blood vessel condition. Taking part is entirely voluntary, and your routine healthcare will continue regardless of your decision.
At a glance
What is this study about?
Port-wine stains are red or purple birthmarks caused by widened blood vessels under the skin. Over time, the extra blood flow can cause the skin and underlying soft tissues to thicken. When this happens around the mouth, it can cause the lip to become noticeably enlarged, a condition doctors call macrocheilia. This swelling can affect talking, eating, and how a person feels about their appearance.
Standard care often involves laser therapy to lighten the skin colour and surgery to reduce the extra tissue. However, swelling can sometimes return. In this study, researchers want to see whether adding a medicine called sirolimus makes these treatments more effective over the long term. Sirolimus works by calming the signals that cause blood vessels and tissues to overgrow.
Throughout the study, doctors will monitor participants closely over 60 months (five years). They will gently measure lip thickness in three dimensions, track changes in skin colour from deep purple or red to lighter shades, and assess safety. They will also ask patients to complete quality-of-life questionnaires to see how the treatment impacts their daily confidence and wellbeing.
By following patients over several years, the researchers hope to understand whether sirolimus provides lasting relief. The findings could help improve future NHS care for people living with complex vascular birthmarks.
Key takeaways
- Tests whether the medicine sirolimus helps reduce lip swelling caused by port-wine birthmarks.
- Combines medicine with standard surgery and laser therapy.
- Open to adults aged 18 and older.
- Follows your progress and quality of life over a five-year period.
- Participation is entirely voluntary and won't affect your normal NHS rights.
Who may be eligible?
This trial is open to adults aged 18 and over who have a facial port-wine stain birthmark that has caused significant swelling of the lip (macrocheilia). Participants must be planning to have surgery and laser therapy as part of their planned care.
Both men and women can take part. Because sirolimus is a prescription medicine that affects the immune system, the medical team will carry out standard health checks to confirm it is safe for you to take.
People who are pregnant, breastfeeding, or who have certain other medical conditions or medication interactions may not be able to join. Your hospital doctor can check whether this study is suitable for your individual circumstances.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am aged 18 or older.
- I have a port-wine stain (vascular birthmark) on my face.
- My birthmark has caused noticeable swelling or enlargement of my lip.
- I am scheduled or being considered for surgery and laser therapy.
- I am willing to take a daily medicine and attend hospital visits for five years.
What does participation involve?
Taking part in this study lasts for up to 60 months (five years). During this time, you will take sirolimus by mouth, either as an oral liquid or as tablets, as prescribed by the study team. You will also undergo your planned surgery and laser sessions. You will need to attend regular hospital appointments so the team can monitor your health, check for any medication side effects, and carry out blood tests. At set visits, doctors will measure the size of your lip using a small, gentle measuring device called a caliper and evaluate the colour of your birthmark. You will also be asked to fill out questionnaires about how your condition affects your daily life.
Potential risks and benefits
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Common questions
What is sirolimus?
Sirolimus (also known as Rapamune) is an existing medicine that slows down abnormal cell and blood vessel growth. In this trial, it is being tested to see if it stops lip swelling from coming back.
How is my lip measured?
Doctors use a small, pain-free measuring tool called a caliper to check the height, width, and depth of the swelling on your lip.
How long will I take part?
The study monitors your health and treatment results over five years (60 months) to see if the benefits last over the long term.
Will my birthmark disappear completely?
Treatment is aimed at fading the colour and shrinking the swelling, but it may not remove the birthmark entirely. The study will measure how much it lightens.
Can I stop taking part if I change my mind?
Yes. Joining is completely voluntary. You can withdraw from the trial at any time without your regular medical care being affected.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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