The Importance of Advocacy

It’s been a roller-coaster of a year, so no wonder I have not visited this site since August. But I do hope to fix that in the coming months (I hope I didn’t just jinx myself). 

I tell you what: it pays off to be stubborn!

I wanted to write a word about the importance of advocacy and most importantly, the importance of advocacy for yourself. I have written quite a bit about this. In fact, if you do a simple search for “advocate” or “advocacy” on this blog, you’ll be able to find quite a bit on this subject.

There is no one in the world that knows your body and you better than yourself. You should always advocate for you. What I have found out after 41 years of being an FH patient is that doctors will fall into roughly three categories:

1. They will have no clue what to do with you. They will shrug and tell you, “well, this is genetic, so there is not much we can do to fix this. It is what it is. Go about your life as you normally would.”

2. They will have a god complex and declare that they know exactly what to do about it and they will apply all treatments “by the book”. Or so they say: they will start with changing your lifestyle, your diet, your exercise routine, then they will give you the drugs they have available, start with the least “scary” ones perhaps (like a bile sequestrant or a Zetia - both that trap the cholesterol you get from your bad diet into your GI tract), then with statins, if you’re lucky, and maybe they will add some other drugs when those won’t work. They will expect each drug to work according to its literature and with what they learned in school. They will expect each drug to have exactly the side effects listed in the paperwork that comes with them. If you bring on your own side effects, and if your numbers don’t match the predictions on the prospect, they will ask you if you’re taking them according to the right schedule, or if you know how, in fact, to inject your own drug (in the case of injections), etc ... They will just assume you’re doing something wrong, instead of having the wisdom to know that you are unique and each drug should work uniquely to every individual, instead of knowing that any drug is not a “one size fits all” experience.
   I call this category of doctors,  “doctors that treat me by the book, and never look at me for the unique individual that I am with a unique make-up and history". They think they know, but what they know is a template. This is the most dangerous category of doctors, I think, and they can do the most damage. 

3. Finally, there will be the doctors who both know what they are doing and listen to you as an individual. They look at your unique case and treat you according to your type of elevated lipids, according to your body type, family history, and medical history and sensitivity to drugs and other things. This category is rare and you’re lucky to find them. 

In all these cases, however, the specialist remains the same: you! Although they can be  the guide, you should be the specialist and you should work as a team of equals. You know how your body responds to new medicine. You know whether your body takes lower doses or is slower to respond to a medicine. You know how much you can take of what. And you should feel empowered to guide them, as well. 

Here’s my case. I was diagnosed with FH when I was 8. I have seen all the specialists you can see that (typically say that they) can manage this disease (endocrinologists, lipidologists, cardiologists). Since 1983, I have been on almost all medications that have been available on the market since then till now. No matter what the drug paperwork says, I can tell you with mathematical accuracy that: 

• Niacin absolutely makes me nuts. The flushing is so intense, I literally feel like I am about to die burning in flames. And no, there are no secrets or tricks for me that work to actually make it possible for me to stay on it. Not for me. 
• Bile-acid sequestrants make me gag and give me terrible stomach acid. I do believe this has caused and even chronicisized my GERD, although I stopped taking this type of medicine in my teenage years.  
• Zetia gives me gas so painful I want to punch something. When I first started taking it, I cried myself to sleep at night, it was so intense. I got used to it over time, but it took years. 
• Nexletol upsets my liver enzymes and elevates my uric acid. The elevated liver enzymes are only casually mentioned in the literature and for a relatively small percentage of patients. They also say they will come down over time. I took Nexletol just fine for a couple of years and only after that, they started climbing.
• Outside of the cold-like symptoms that Praluent gave me in the beginning, it is virtually side-effect-free now, after 8 years and it did the most good for me until recently when a yet newer drug worked even better. But not before I had to fight for it with two cardiologists. 

This year, as you might remember, I changed my cardiologist (who also manages my cholesterol). The old one took over two years to figure out how to give me access to Evkeeza, a new infusion drug especially approved for Homozygous FH patients. In the end, he failed, and although I had a long history of seeing him, I left and found a new doctor. 

The new cardiologist describes himself as not only a lipid, but also an FH specialist. He has lots of patients on LDL Apheresis at the only LDL Apheresis center in my state (I believe). When I walked into his office my total Cholesterol was 307 mg/dl and my LDL was 238 mg/dl. At the time, I was taking Lipitor, Zetia, and Praluent. I came to him because I had wanted to try Evkeeza (https://www.evkeeza.com/s/) since 2021. 

The cardiologist told me he would prescribe Evkeeza for me but that will not be enough. He insisted that I need to wrap my head around apheresis (which I have refused to do even before my open-heart surgery and before getting a mechanical aortic valve which are making it more hard now to get a port) and I will absolutely need to only take Evkeeza to supplement a weekly apheresis treatment if I want to see my LDL anywhere near 70 mg/dl (which is my target). He said with Evkeeza alone I could maybe get as low as 110, but never around 100, much less lower than that! 

He told me that approving Evkeeza would be lengthy, because of the prior authorization process, but in the meantime, he could schedule me for apheresis right away. I think I spent an hour in his office trying to explain to him that I will not, under any circumstance, do apheresis - it’s a matter of personal choice and I have said “no” to it for close to 30 years. He insisted that he will be the first doctor who will crack this stubbornness and will get me to do it. I left on a promise to think about it, but only after he would help me to do the minimum of 6 months of Evkeeza treatments and proved that it won’t bring my numbers low enough. 

His office was better than he gave them credit for, and they approved my Evkeeza almost right away. I started the infusion almost a month to the date from my first appointment with him. The drop in numbers was immediate. After just one month of treatment, my LDL dropped to 100 mg/dl. (remember when he insisted that even after a full treatment of 6 months it might just get closer to 110, but definitely never lower?). After 6 months, I am at 75 mg/dl. And this is with no Ezetimibe (which he discontinued because he now believes Evkeeza works better than expected for me) and with half the strength of Lipitor that I was taking before (I was taking 80 mg when I saw him first, and I am taking 40mg now). 

At my last appointment he said: “You convinced me. I am not ashamed to admit that you were right and I was wrong. You proved me wrong. And I thank you for it. No more talk of apheresis. You proved that this new drug works.” 

I, of course, have no merit in this, other than I just wanted to try a new treatment. I know that drugs work for me. I have seen them working at various efficiency levels. I have met a lot of folks on Evkeeza who describe LDL levels of 50 mg/dl. Even people with HoFH with higher levels than mine describe unbelievable results. I didn’t know if it would work this well, but I knew I wanted to try it. I am glad I didn’t let my guard down and I didn’t sign up for apheresis like the doctor so strongly pushed for it.

There is nothing wrong with apheresis per se, of course, but it is just not my personal choice. There is a certain trauma and invasiveness that I associate with apheresis and I would rather avoid it at all cost, if possible. There is a certain commitment to it, and I travel too often and too much to commit being tied to a hospital bed for at least half a day every week. Now that I am very concerned with clotting and infection (because of my mechanical valve and my blood thinners), I do not want a port implanted anywhere in my body, unless there are literally no other options. And I truly wanted to try something less invasive (if you can call a once a month infusion “less invasive”) that could actually work better for a longer period of time than a week. I just knew my limits and what I can and cannot commit to. And I knew my body. Praluent worked so well for me, from the beginning, that I knew another “new” drug might too. 

The mechanism of each of the drugs I was taking at the time is completely different, but I have just seen that newer drugs do work better for me. So, I wanted so badly to try it. And I am glad I pushed and held my ground. 

And of course, I am lucky and grateful that I found a doctor who would actually listen to me. He did dig his heels a bit in the beginning because of his many years (probably as many as my whole life) of what he perceived to be a tried-and-true treatment that he is very familiar with and with which he has seen results, but my perseverance won out. It’s a two way street - and I am glad we met in the middle. 

My new cardiologist has a favorite phrase when we discuss treatments and numbers and drugs. He often congratulates me for my knowledge of FH and treatment options (most of my knowledge comes from self-teaching since the internet is such an exhaustive source of information now), but also from following and advocating for the Family Heart Foundation (https://familyheart.org/) - their webinars and knowledge sharing of the newest findings in treatments is invaluable) and then he says “we’re on the same page”. 

I am glad that about this treatment we finally managed to meet  ...  on the same page. 

My most recent numbers, after 6 months of Evkeeza