I started this blog for several reasons, one of them being to update my friends and family on my health issues. I haven’t done a great job with that, so here’s my attempt to start working on that. For those of you with no interest, feel free to skip it. It’s pretty boring if I do say so myself. Anyways, with PNH, there are four primary treatment options. Deciding which one is the right choice can feel a little like taking a shot in the dark depending on your symptoms and your history of blood clots.
Treatment option one: Watchful waiting. This was the only option up until a few years ago. This primarily consists of checking blood counts once a month or so, treating symptoms as they arise, and being on the constant lookout for anything that may be a blood clot. This was the option that we were going with, and the option our second PNH specialist recommended, until a year ago when we made a change in an effort to have a baby. We were pretty happy with it, my blood counts were decent and I had very few symptoms of actually having a disease.
Treatment option two: Blood thinners. We tried this for a couple months shortly after I was diagnosed after seeing our first specialists. She believed strongly that anyone with over 50% of their cells affected by the disease (70% of my cells are affected) should be treated in some way. She preferred soliris but as I was resistant, she settled for blood thinners. I was already borderline for blood thinners at that point because my platelets were at 70,000 so the risk for bleeding was high. After a couple months of struggling to find a dosage that worked, we came to the conclusion that I was probably at more risk being on it due to lifestyle (riding young horses, snowmobiling, lots of activities outlawed while on blood thinners) and decided to call it quits.
Treatment option three: Soliris. This is our current method of treatment, it is the only option that actually treats PNH, and it is the option recommended by most specialists. We started it because we wanted to have a baby and being on soliris was the best way to minimize the risks as much as possible. I’ve been on it for a year now and since starting it my blood counts have gone down considerably and so I have more symptoms. Because of this, babies are off the table. Technically speaking, it is working. My blood is not breaking down in my blood stream so from a clotting risk standpoint, it is a successful treatment. From all other points though, we are not so sure that it is really working. It could be a coincidence that my counts went down when I started it, in which case it is a very good thing that I am on it. If the drug is causing the lower counts though, its usefulness to me is debatable. Ultimately, it does provide a certain peace of mind, but it comes at a very high cost. The highest drug price in the world in fact. While I have been told repeatedly by everyone to not think about the cost, it is impossible not to. It is a factor, there’s just no way around it.
Treatment option four: Bone Marrow Transplant. I am no where near this point and I hope to never be there. This is reserved for the very sick, people who will die without it. The introduction of soliris has done a lot to make this a less common need for PNHers.
So the question now is, to treat or not to treat. Do I continue on the soliris or do I try stopping it and see what happens? I only started it to have a baby so that original need for the drug is gone. There is no way to know for sure how well the drug is working for me without stopping it, and that comes with a month or two of risks that my doctor and I are not too excited about dealing with. After that month or two passes, we could find out that I am just fine without the drug and that we can easily go back to watchful waiting, and hopefully have better blood counts as a result. Or, we could find out that the drug was doing much more than we thought and that I need to be on it. That would mean that all that risk had been for nothing. Soliris masks the disease in a way so we really have no idea how symptomatic I actually am as long as I am on it. I was slowly but surely having more PNH symptoms before starting soliris so I could be more symptomatic now and not realize it. The other issue is that because of my low blood counts, I am also at a point where I may have to start another therapy which comes with side effects that I would prefer not to deal with, as well as an increased risk of skin cancer, which is problematic since I just had melanoma so I am already at an increased risk. If stopping soliris would cause my counts to go back up on there own, then I could avoid all of that. So there it is, the thought that runs on replay through my head all day, every day. There are so many unknowns with this disease, so much grey area. It is tough to ever really know what the right decision is. For any of you interested in more information, here is a webcast about the current thinking and treatment of PNH by the first specialist I saw.