Sirolimus

Symptoms & Treatment
1

Hi all!

I have a large, diffuse GVM (glomuvenous malformation) in my left leg. I’ve recently visited Dr. Mulliken and his team at Boston Children’s, and Sirolimus was mentioned as a possible treatment for my pain (as the gvm is so large and some of my larger spots have thrombosed, so sclerotherapy is difficult).

I know this is a relatively new-ish treatment, so I’m wondering if anyone has had any experience with it and might be willing to share. I’ve been looking at the Sirolimus for Vascular Abnormalities channel on facebook, as well.

I am particularly interested in the experiences of those who do NOT have a lymphatic abnormality (as many of the facebook posts seem to). I’m concerned about the possibility of permanent edema as a side effect (I’m very lucky in that my gvm does not make my leg noticeably larger than my right) and my vanity and wardrobe would like to keep it that way.

Have you experienced edema, either in your affected limb or other parts of the body?
Did you stop taking Sirolimus once this was diagnosed?
Did your swelling go away after you stopped taking the medicine or reduced your dosage?

Relatedly:
Are the positive effects of Sirolimus worth the hassle of regular bloodwork/side effects?

The doctor I spoke to the most mentioned more than once that most side effects (stomach issues, cholesterol/liver enzyme levels) could be controlled with dosage adjustments.

I appreciate any insight that you all might have!

Christina

2

I see that you haven’t had any responses. I did do a search on the site, and this is the search results. Hopefully you can get some additional help this way.

Sharon from ModSupport

3

Hi Christina,
Sorry for the late response. I have to warn you I’m no authority here. My medical knowledge and awareness of my condition is very limited. However, I did take Sirolimus for nearly a year. I have avm on my face and pinna as you can see in my picture. I didn’t have pain over the avm; just the swelling. The tab was suggested to me as a new treatment after all other options seem to have exhausted. I was 21 then. Though we felt it was helping initially, I stopped taking the pill after I started feeling that it wasn’t that effective. One thing I noticed as a student was that I was feeling more tired those days. I’m not sure if it has anything to do with sirolimus. I also took propranolol along with it so maybe that. But I don’t think I had edema. Anyhow, I cannot say if it could work for you just because it didn’t do much for me. Our avms are at two different places. I’m not sure if this is going to help you at all, but that’s all I know.