My N-of-1 Odyssey

On Monday 7/1, I’ll be repurposing Rituxan for Immune Thrombocytopenia for a second time. The goal is to put me back into remission for an extended period of time. When I used Rituxan at the end of 2014, I had a remission period over four years. I’d like to think we will be able to achieve similar results this time around.

Rituxan is a chimeric monoclonal antibody targeted against the pan-B-cell marker CD20. In plain English, it targets B-cells by going after CD20 which is expressed on the surface. Ultimately, after multiple blood tests and positive results from immunosuppression by steroids, we were able to figure out my B-cells were responsible for destroying my platelets. By shutting them down using Rituxan, I am able to maintain a normal platelet count.

The first test I received is Anti-Nuclear Antibody (ANA) which came back positive and indicates autoimmune activity. We also tested for Lupus, Rheumatoid Arthritis, and Sjögren’s Syndrome among others. I ended up also testing positive for SS-B antibodies, further confirming autoimmune activity. Another great marker: I respond very well to immune suppression with Prednisone. All of these factors collectively helped my doctor come to the conclusion that I had chronic Immune Thrombocytopenia and I was destroying platelets via my B-cells. I never had a bone marrow biopsy because when I am on high doses of Prednisone, my platelet count returns to normal. There is nothing to indicate that I have any issues with platelet production in my bone marrow.

Because of this, N-Plate and Promacta would not be the right treatment for me. I could stimulate platelet production, but my B-cells would destroy those platelets as well. Ultimately, the idea is to stop the process of platelet destruction. That’s how I came to the decision to use Rituxan in 2014, and why I advocated so hard to use it again this time.

So now I begin the process of making myself an N-of-1 example of how Rituxan can help ITP patients with B-cell led destruction of platelets. We will collect a CBC before each of the four infusions to track progress. I am currently on no medication for my platelets after I finished my Dexamethasone pulse last Monday (one week before my first infusion).

Besides chasing remission for a second time, I had an inordinate amount of trouble getting insurance to approve this treatment. We must see to it that Rituxan become an on-label therapy for Immune Thrombocytopenia, especially since patients with B-cell led destruction of their platelets do not have an FDA-approved treatment.

Acknowledging the clinical diversity of ITP is a must, especially with multiple drugs on the market and more in development. There is no one size fits all treatment for ITP, and what works for me may not work for another patient. Gathering treatment data and comparing it with ANA tests will be a great way to dissect some potential remission patterns in patients.

Calluses From Living Rare

Last week, I was forced to reflect on the last five years of my life. The diagnosis, the treatments, the risks, and reward of remission for over four years. The heartbreaks that came from losing people (and my dog) to things I could not save them from, no matter how hard I tried. It was the first time I told my story from start to finish. It was also the beginning of my self realization that I am a completely different person from who I was on April 30th, 2014, the day before my D-day (diagnosis day). 

Before ITP, I was a tough chick with a no nonsense attitude. But I had no calluses. That came to an abrupt halt on May 1st, 2014. Baptism by fire, taking on a dysfunctional body in a broken system that does little to help patients like me. There was no soft introduction into the world of rare diseases, I was simply dropped into the pit and expected to sink or swim.

I was forced to become my own advocate, to research, absorb, digest, and become the expert on ITP. My view of physicians changed from putting my implicit trust in them, to questioning everything. I no longer wore rose colored glasses when it came to my views on healthcare. I was being raked over hot coals, and that’s how I developed my calluses. 

There is a certain level of pressure to be gentle, warm, and kind as a patient. I’d like to think I have retained those qualities over the last five years, but they’re not as apparent now. It’s hidden underneath a hardened exterior, one I had no control over developing because I needed it for survival. It builds through every bump in the road, side effects from treatments, disappointing lab results, and just the overall way life can seem to come down on you during difficult times.

To every rare disease patient who takes time to reflect on their patient odyssey, don’t beat yourself up over a hardened exterior. While we do our best to navigate a system that constantly throws us curveballs, a roughed up exterior is part of the package. Wear your calluses with pride, you earned them like your zebra stripes.

Chasing Remission a Second Time

6/7/2019 my first IVIg infusion

It took 14 days for my platelets to plummet back to a level low enough where I was beginning to bleed again. I had my first IVIg infusion at the beginning of the month, starting off at 20,000 platelets. My vein blew during the CBC beforehand. My insurance played games with approval, causing a four hour delay that ultimately rushed my treatment. 48 hours later, I was suffering with the classic symptoms of aseptic meningitis (don’t worry, an adverse event report has already been filed and I am okay).

My platelets reached 98,000 three days after the infusion, then 78,000 and 14 days later, back down to 21,000. Given all I went through, this was a disappointment. However, notably absent is my Sjögren’s related pain, so I won’t say this is a total loss. I declined doing one more infusion because I don’t believe a 14 day window is worth it. Ironically, Dr. Ahn predicted back in 2014 that an IVIg infusion would last no more than two weeks, again showing how absolutely brilliant he is!

There are a few questions surrounding IVIg and how it will impact ITP patients with B-cell led destruction of platelets. I wonder if the prevalence of other autoimmune activity shortened the effectiveness of the infusion, or perhaps if I did two back to back, would it have lasted longer? I don’t feel the need to put myself in a lab rat position for this, simply because the scientific evidence is not compelling enough for a second look right now. I have my eyes set on the long term goal.

I ended up doing a Dex pulse for four days, and today is my last dose of 10 pills. This is not as bad as Prednisone in that it is quick, but it still comes with major side effects. Heartburn, insomnia, irritability, sweating, etc. Pretty much the same things I experienced before. Tomorrow morning I will have my platelets checked again, and I am curious to see how much they popped up.

Which brings me to the big news: we are trying Rituxan again. I have been waiting to hear those words since March, and I have dealt with a lot of miserable experiences to get here. This time is much different, I know what to expect, but the stakes are higher. I want to see Rituxan become an on-label treatment for patients with Chronic Immune Thrombocytopenia that have B-cell led destruction of platelets. That means it is not for people with T-cell issues, platelet production issues (who respond well to N-Plate and/or Promacta), and those on Tavalisse.

I launched the ITP Patient Driven Research Initiative last month to tackle this issue, and ultimately work toward better targeted therapies for patients with my rare disease. The research and drug development currently underway is a start, but we can do better. A solid data platform led by patients inputting their results based on a variety of treatments over extended periods of time will help us develop data sets needed for better treatment protocols. From there, we can acknowledge the clinical diversity of ITP and get pharmaceutical companies on board to run better clinical trials.

That’s the update for now. I have an early day at the hospital tomorrow with bloodwork and planning. I’m hoping to start infusing ASAP, especially since I have been through the platelet olympics these past few months.

New Therapy Jitters: IVIg

Thursday’s doctor visit was a rollercoaster. I ended up at 31,000 platelets and needing treatment, so he suggested we try IVIge. Cue major nerves, I have spent the last five years reading about the brutal side effects other ITP patients experienced, so I am worried about how I will feel after the infusion.

I’m curious to see how long my platelets will remain at a normal level after treatment. At the height of my platelet destruction in 2014, Dr. Ahn believed it wouldn’t hold for more than 10-14 days. However, this time around I caught it early, so the outcome may be entirely different. I am happy it is only one infusion, instead of four, and will hopefully have me back on my feet after the weekend.

So how am I preparing for it? First- I asked fellow patients. The hospital told me to hydrate, but I wanted to hear from people who have been in my position. Thankfully, everyone told me to load up on electrolytes, so I bought a ton of Powerade (Seminoles don’t drink that gator-garbage), Smart Water and Coconut water. I’m also planning to eat soft foods the day before, during, and a few days after the infusion in case I have vomiting. Because my teeth were weakened by past prednisone usage, I want to minimize any potential damage that can be caused by throwing up. Infusion clinics really should be giving out this advice when you schedule your appointment, but that is a fight for another day. I want no surprises, so I’m glad I asked because just chugging water the day before was not going to cut it.

The most difficult part about this infusion is the unknown. I did not experience this level of apprehension when I used Rituxan, but I was confident it was going to work. This is a sort of a shot in the dark, I expect for the treatment to fail after a few weeks and my platelet count to drop. However, we need the data and we need to see how I tolerate it. So I’ll play along, for science.

To everyone who offered advice, thank you.