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.

Pre-Visit Nerves

It’s no secret this go-around with ITP has been stressful. I thought by now I would be close to finishing another round of Rituxan, but since Dr. Ahn retired, no such luck.

Tomorrow I see my new hematologist again, he’s taking blood and after the results come back (within 10-15 minutes), a plan will be crafted. Why the nerves? I’m afraid of once again being told I need to “wait” for treatment.

Because my platelets can drop to zero pretty quickly, there is no sense in waiting around for it to happen. Unfortunately, ITP treatment varies by doctor and some are willing to wait until I get very low. I know my body well enough to know that will come quick, so acting now is best. After all, we wouldn’t wait until cancer reached a more advanced stage before issuing chemo. ITP should be treated no differently.

This is a short blog tonight, I need to try and get some rest, but I wanted to document my nervousness because I know there are other patients out there like me. I guess this is “normal”, but it shouldn’t be. I should be able to access the treatment that saved my life once before. I’m chasing that “new normal” I started blogging about almost five years ago. I want to go back to being myself, not living week to week between lab appointments.

Out of Remission, Back on Prednisone

Cue the long groan, I am out of remission and my platelets hit a startling 53,000 this week. This triggered swift action by my new hematology team, so I am taking 40mg of prednisone daily.

Before I get into that- let me tell you how much I love my new hematologist. Dr. Ahn retired February 2018, so I was nervous about seeing a new doctor. Friends of mine recommended Dr. Harrington at UM (who’s father happened to discover how ITP works). He’s brilliant, funny, and practical in his treatment of ITP, especially when it comes to steroids. I was so glad to hear him say he won’t keep me on prednisone for a long period of time.

One of those familiar “Purpura” bruises on my leg.

I had blocked out most of the negative experiences I had almost 5 years ago with steroids. After all the moon face, acne, weight gain, constant hunger, anger, poor temperature regulation, and insomnia were horrible. Well, that’s all back and I have been expressing my frustration about it. I was finally able to sleep last night, thanks to medical marijuana (more on that in an upcoming post). I wish I had that stuff back in 2014 when this journey started. I woke up this morning a bit more refreshed and less hungry. My goal right now is to not gain any weight while moving through this part of the treatment process.

Another sign, see that purple bruise in the 73? A simple pinch from my bracelets caused that.

I’m documenting bruises again and getting weekly CBCs. Obvious goal: using Rituxan again. But I run into the same problem I had in 2014, safety data is scarce. I’m leaning on my team at UM to trust that since it worked once, it will work again. Since I am traveling a lot this month, I am hoping by mid-May we will be able to get those infusions going.

If you have used Rituxan more than once for ITP and are willing to share your experiences with me (and your CBC data), please email me at [email protected]. I’m planning on doing something similar to what I did in 2014, creating a mini data set to show the efficacy of Rituxan for immune destruction of platelets.

There’s more to this story, I am still hard at work behind the scenes to get ITP patients access to a CD 20 inhibitor ON LABEL. Stay tuned.

A Healing Heart

I have not blogged in a significant amount of time. For those of you who followed Kona’s story on Twitter, Instagram, and Facebook, you know the magnitude of her death. In 13 months I lost Kona and Shaun, which was too much to handle. I felt the need to take a break from this blog and focus on myself for a while. I now finally feel ready to come back and start fresh.

Kona’s death signified the end of what was left of my old life before I got sick. She was my final connection to the old me, and my partner in crime. I fought with everything I had to save her, and still came up short. I am still haunted by the fact that I could not save her, even though I was able to figure out how to save myself. My hope is that Kona will live on in a variety of ways. The first being donating her tumor to research.

Kona’s tumor was divided into three segments: one for pathology, one donated to work on a treatment for Glioblastoma, and another is being stored at Purdue University for a later project. I wake up every morning and the first thing I think about is this. I comb Google Alerts on Glioblastoma, hoping that something will pop up with canines. Perhaps one day, that missing link will be discovered.

Kona with Dr. Embersics at Purdue, enjoying some chicken.

Kona’s story has grabbed global attention, from animal lovers to the rare disease community. As her Mom, it is therapeutic to have people approach me months after her death to tell me they followed her story. Now, Kona is being discussed on Capitol Hill, and I hope to introduce the KONA Act to the 116th Congress in her honor. Additionally, I will be requesting funding for the Purdue University College of Veterinary Medicine. Everyone there from Dr. Bentley, Dr. Embersics, and the staff did everything they could to save my girl. They deserve to have their research funded to the fullest extent possible.

Angel Kona at the National Institutes of Health

I now have a Kona angel that comes with me to all my meetings. It’s a reminder of her and the long road ahead to make sure we find effective treatments for glioma-based tumors. Her pathology report came back: oligodendroglioma. I promised her the night she passed that I would fight until my last breath to make sure they’re wiped off the face of the earth.

#KonaStrong

Our Journey to Translational Research

I’m writing this on the eve of the most important road trip of our lives.

On 6/15, Kona Bear my sweet Boston Terrier/French Bulldog mix was diagnosed with a 3cm Glioma tumor in the right frontal lobe. The day prior, she presented alarming neurological symptoms that her vet was ill-equipped to handle. Her diagnostic odyssey was similar to mine.

That fateful Friday afternoon, Kona’s neurologist pulled me in a room and showed me the MRI images before the entire scan had been completed. My worst fear was confirmed. My best friend was given 4-8 weeks to live without an intervention.

Fast forward two weeks, and we received confirmation from Dr. Bentley at Purdue University that Kona will undergo surgery to remove her tumor on July 5th. Three weeks after she first showed signs of something being wrong. In the time between diagnosis and our trip, my life has been filled with extreme highs and lows. It was topped off with Kona having a seizure on my face this past Friday night. I was asleep in bed with her when she walked over to me before it started. Thankfully, I had an idea of what to do. Before that, Kona had not had a seizure in my presence (if at all).

Once I began researching Canine Glioma, I was shocked to discover how closely linked dogs are to humans. We are the two species that grow primary brain tumors and our DNA is similar, making research on canines very valuable and easier to translate for human trials. Additionally, dogs and humans live in the same environments and are exposed to similar variables, so the documented side effects from clinical trials are more meaningful. Despite this, the outcomes for both canine and human glioma are grim.

I am dedicated to saving Kona and furthering science. I believe Kona holds data that can help researchers better understand glioma, the effects of surgery and chemotherapy for these tumors.

Tomorrow we embark on a new journey to change translational science.

To follow Kona’s journey, like her Facebook Page: Kona Strong.

 

Finally Found My New Normal

My mother, Judge Diaz and I after being officially sworn in.

I apologize for yet another lengthy absence from my blog. I’ve been diverting all my energy into a few big projects lately. Most notably studying for the Florida Bar Exam. If you know anything about the test, you have probably heard just how stressful it is! My last blog post was right after the exam. I needed a break from blogging.

I’m so excited to announce that I passed the bar exam and was sworn in on April 16th! It was a crazy morning, I barely slept the night before, Sjögren’s was causing a nasty flare, and I was nervous. When I saw I passed, I cried! It was great to be surrounded by friends, my professors and my family during the ceremony.

Since then, I have been busy establishing my new normal. I had the chance to chat with Marco Rubio and thank him for co-sponsoring the OPEN Act. He is enthusiastic about getting it passed, the next time I am in DC, we are going to make sure everyone is on the same page strategically to get it done.

As for my work- I continue to consult with pharmaceutical and biotech companies on various projects, ranging from patient advocacy plans to legislative strategy. I am excited to be using my legal education to continue the pursuit of making the world a better place for rare disease patients.

I look forward to rebuilding my life after all of these great things, and continuing the push forward. Thank you for being a part of my journey.

Achievement Unlocked: Law Degree

I did it. 

On December 7th, I graduated from law school with a concentration in health law. Last week, I took the Florida Bar Exam. Life has been pretty hectic since I last blogged, but I am excited to get back into it.

In June, I was awarded a scholarship to Seton Hall School of Law’s Healthcare Compliance Certification Program. It was a fantastic week in New Jersey, really fine tuning my knowledge of federal compliance regulations. I was also alarmed at how these laws can negatively impact the rare disease community. Expect more on this topic soon.

In August, I was invited to attend a special meeting with the Venezuelan dissident community in Miami, which was being held by Senator Marco Rubio, Governor Rick Scott, and Vice President Pence.

In September I had a chance to chat with Allen West, a great supporter of the rare disease community.

In October, I was invited to speak at the World Congress Patient Advocacy Summit. My speech was on the 21st Century Cures Act, and the role patient advocacy played in forming the legislation.

Of course, while I was in Washington, DC I made sure to meet up with Congressman Bilirakis to continue working on the OPEN Act.

In December, I met with Corey Lewandowski and David Bossie. We had a chance to chat about the OPEN Act, President Trump’s support of the rare disease community and how we can continue to push for more reforms to benefit the 1 in 10 Americans with a rare disease.

In January, I took a study break to meet former federal judge Ashley Moody, who is running for Attorney General of Florida.

It’s been a busy few months, and now I am back to work, focusing on reforming healthcare regulations and proposing legislation to assist patients and industry meet the end goal of getting treatments to market.

 

 

The Truth About OPEN Act

I was blessed to be with Congressman Bilirakis this week, working on the OPEN Act.

Since the end of last year when the OPEN Act was removed from the 21st Century Cures bill, I have spent many sleepless nights trying to understand why Washington was not comprehending the critical need for repurposing drugs. We are now working to convince Democrats that the OPEN Act is not a handout for pharmaceutical companies, as I am living proof that repurposing an FDA approved drug works wonders. Continue reading →