Making the decision to start Sirolimus

If you or a family member has CLOVES, then you know that there aren’t many options available to help treat it. Invasive methods are out there, like de-bulking surgery or sclerotherapy, but many people may not want to jump right into difficult medical procedures like those.

Instead, there is another relatively new alternative available. A recent discovery that has proved effective in those with CLOVES is a drug known as Sirolimus. Sirolimus is an immunosuppressant often used in individuals who have had a transplant to help prevent organ rejection. However, a study done in 2016 concluded that Sirolimus is actually quite effective in individuals with vascular anomalies. Basically (to skip over the medical jargon), this drug is able to soften the tissue and actually lessen the size of the mass of the vascular anomaly. Based on this Phase II trial, Sirolimus was actually quite effective in the patients studied.

Now, this drug treatment is often used in patients with CLOVES, but some may have a hard time making the decision to go on it. I sure did when I first heard of its use.

Going on medication is a hard choice to make for anyone, especially if you’re making this decision for your child. No one really wants to have to rely on taking pills every day. It took a long time for me to really make this decision. When I visited Boston Children’s Hospital last summer to meet with the CLOVES team, my doctors highly recommended I start Sirolimus to help treat the vascular anomaly on my back that had been getting more swollen. For a little while, I had a difficult time saying ‘yes.’ With this drug being an immunosuppressant, I would be at a much higher risk for infection and getting sick. I tried to balance the pros and cons, but that didn’t really help either.

The pros were, it could possibly make my vascular anomaly smaller in size, but it’s not 100% guaranteed. Everyone’s reaction to the drug is different, as CLOVES is such a unique disease. The cons, on the other hand, seemed to have a more extensive list. I would be more at risk for infection, I would be more at risk for cancer, and the everyday side effects didn’t seem appealing to me at all. Acne and mouth sores are the most common side effects in people taking Sirolimus. Did I really want to have to endure all of that just for the possibility that maybe my vascular anomaly might lessen in size? All of these thoughts are the reason I said ‘no’ at first.

However, as more time passed, I thought about my other treatment options. And guess what? There were none. The fatty tissue in my back couldn’t be de-bulked through surgery because it’s actually lying under my muscle. Turning to that option would be a lot more risky—I could lose some function in my left shoulder if I took that risk. Obviously, because of that, I wasn’t going to turn to that option. So I started to really think about actually starting the Sirolimus. You know what’s good about just trying a new medication? You can stop it at any time. I would only be taking it to see if any positive results occurred. Honestly, for me there wasn’t much to lose at that point. If the side effects were too much for me, I could stop the medication at any time. If anything happened on the medication that I didn’t like, I could always stop it. That’s what is nice about just trying something new.

So I asked myself, what do I have to lose? There is a new treatment option out there that used to never exist. I had so little options in my past when CLOVES wasn’t even a diagnosis yet. The more I thought about it, the more I realized how incredible it is that there is actually another treatment alternative available. I’m so used to there being only one way to treat my disease—surgery. That’s how it’s always been. Now, there is actually a medication that can possibly reverse the effects of my disease (somewhat). How crazy is that?!

So that’s how I made my decision to go on Sirolimus. I’ve been on it for almost 4 months now, and I don’t regret it. The way I did it is my doctor eased me into the full dose, which is what they do for all CLOVES patients I believe. I first started taking 0.5mg for 2 weeks (1 pill), then increased my dose to 1mg (2 pills) for 2 weeks, increased the dose to 1.5mg (3 pills) for 2 weeks, and then I got to my full dose, 2mg. At the full dose, I received a new prescription so that I was only taking one pill at 2mg, not 4 pills (that would be insanely annoying). You have to take the medication at the same time every day, so it’s helpful to pick a time of day that’s easy to take it. I take mine at 8:00am with food so that I’m usually taking it with breakfast. If you miss a dose, it’s not a huge deal, but sticking with that ‘same time every day’ instruction is important for it to be effective.

Being on Sirolimus does involve having to get blood drawn often. For me, and I’m assuming for most anyone on it for my same reason, I got labs drawn every 2 weeks when I increased my dose to see how the drug was interacting with my body. After I got to the full dose, I’ve been getting labs way less often, so it’s not too much. And there’s one more instruction to follow while on Sirolimus—you can’t eat grapefruit. Super random, I know, but apparently it can lessen the effect of the drug.

About a month or so after I first started, I did notice some mouth sores forming and a few breakouts on my face. They aren’t fun, that’s for sure, but they don’t last long. And there are ways to help ease these side effects as well. My doctor prescribed me Nystatin, a medicated mouthwash that helped get rid of those mouth sores quicker. The mouth sores only last for a few days to a week at a time. As for my face breakouts, I visited my dermatologist to help me with this, and she prescribed me Sodium Sulfacetamide Sulfur Cleanser to use on my face once or twice a day in addition to the Tretinoin gel I’ve already used for a while for spot treatment. And to be honest, this really helped control those side effects. Also, what I’ve noticed recently is that these mouth sores and acne only happen sporadically. It’s not a consistent breakout—they’ll last for about a week and then everything will be fine and dandy for a while. No mouth sores, no acne for weeks at a time. And oddly enough, when I break out, I also get mouth sores—they happen simultaneously. I have no idea why; it’s probably different for everyone, but that’s been my experience. The good thing though is that these side effects can be treated.

My doctor told me that I shouldn’t see results of the Sirolimus until I’ve been on it for about 6 months. So far, I haven’t noticed anything, but I’m committed to staying on it until I notice something—if anything. The side effects aren’t nearly as bad as I thought they would be so I’m really glad I decided to go on it. As for the immunosuppressant dilemma, I haven’t gotten unbearably sick while on it either. I’ve had a head cold here and there, as usual in the winter, but I never got to the point where I had to visit my doctor.

Overall, I’m glad that I decided to go on Sirolimus. It felt like a huge decision to make, but it actually wasn’t as monumental as I had built up in my head. And why not try something new? With how rare this disease is and how little treatment options there are available, trying something new is not a bad idea. I’m hoping that sharing my decision to go on Sirolimus and my experience on it so far will be helpful in some way to others trying to make the same decision. With how little options there are for us with CLOVES, a new treatment is huge. For me, I decided to try something new, and if it doesn’t work, I can just stop it at anytime. That’s all there is to it, really. The doctors in Boston strongly suggest it, and that’s saying something.

Trying something new can be scary. And it’s every individual’s own personal choice on whether to start this drug or not. In my opinion, I decided to take the risk since trying Sirolimus had the possibility of lessening some symptoms of CLOVES. I’ve exhausted my efforts throughout my life and done what I could, and I’m always open to exploring new options. For others, it depends on your situation. Weigh the pros and cons, take a while to really think about it, and decide if it’s the best choice for you. I personally gave it a lot of thought before jumping into it.

But to help anyone make their decision, I haven’t had a bad experience with it yet. I’m hoping this may have eased the minds of anyone contemplating Sirolimus for you or your child!

Information on Sirolimus retrieved from:

1. Efficacy and Safety of Sirolimus in the Treatment of Complicated Vascular Anomalies​ 
2. Drugs.com/Sirolimus

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