Wednesday, August 17, 2016

Autoimmune Patients And The HSCT

I have received an influx lately of questions regarding my current treatment plan to receiving an Autologous Hematopoietic Stem Cell Transplant (HSCT), also known as a Bone Marrow Transplant. Obviously, this is a less traveled rout for autoimmune patients, although it is becoming a more recognized treatment within the community. I thought it would be helpful to my family, and readers, if I took a post to explain the reasoning behind this decision, and what the process will look like if I am accepted into the HSCT trial.
What is an HSCT?
First I will start off by explaining what the name means: autologous means the transplant will be preformed with my own cells, and a hematopoietic stem cell is a cell derived from the blood or bone marrow that creates blood and immune cells. The name literally means my own stem cells will be transplanted to myself. The entire point of a transplant is having the ability to give an incredibly dangerous amount of chemotherapy to a patient without causing death. The dose will obliterate all their bone marrow and immune/blood producing cells; the stem cell reintroduction is simply to save the patient from the brink of death. These new immature cells start regenerating blood/immune cells the body cannot on its own. It can be compared to wiping a hard drive. Everything is deleted, and then you must hit the reset button. The stem cells will reset the body by creating new immune cells that are hopefully disease free. How can they be disease free if they are MY OWN cells you ask? They are immature stem cells and have not been "activated" yet. Remember, my old cells only activated when I was 20 years old. These new cells were just 'born' and have not been told they need to attack me, yet! lol This is a widely used practice in many cancers and blood disorders. The high dose of chemo successfully eradicated all the diseased cells -- leaving a clean slate. You can see how this process would highly benefit someone with autoimmune disease, as our primary malfunction is overproducing immune cells.

Who receives it?
Now that you know a the purpose behind the transplant, you're probably wondering why they are not a more widely used treatment? The truth is this treatment is extremely dangerous, and does carry a heavy mortality rate. The human body is not meant to withstand that overwhelming dose of poison, and a variety of patients are unable to survive the process.
The use of HSCTs in autoimmune patients is fairly recent, only coming into play withing the last fifteen years. This process is solely preformed on severe, therapy-refractory autoimmune patients with poor prognosis' that lack any form of alternative treatments -- It is more considered a rescue strategy than an actual treatment in the eyes of medical science. Studies have shown that Systemic Sclerosis patients seem to have the most difficult time with treatment due to the existing heart, kidney, and lung issues. Our already fragile organs give us less chance of success when compared to other autoimmune, or cancer patients who may not have internal organ damage. The process is currently being studied in severe Multiple Sclerosis, Lupus, Systemic Sclerosis, Myasthenia Gravis, ITP, CIDP, and a growing list of others. While patients may have much to gain form this treatment, it is only considered if all other options have been exhausted due to the evident possibility of serious side effects, including death. Currently the HSCT for autoimmune patients is a clinical trial, and is still considered "experimental" by the US. In order to receive treatment you must be accepted into detailed criteria placed by the researching physician. Very few centers around the country take part in these trials, and only accept a limited number of patients.

What is the process?
Once accepted into the trial by the completion of detailed evaluations, the patient will undergo "harvest." The patient will receive a weeks worth of medication that forces stem cells to overproduce, as well as move out of the bone marrow into the blood stream. You can imagine what a very uncomfortable and painful process that would be; it has actually been compared to child birth. Once the immature stem cells have migrated to the blood stream a central access catheter is placed and the cells are harvested through an apharesis machine. The stem cells are then frozen and stored for the patient. Next comes the chemotherapy conditioning. Patients are given anywhere from 15 - 30 grams of a cytotoxic (cell-killing) agent called Cytoxan over a four  - five day period. (For reference I only had one gram a month when I received it earlier this year.) This powerful drug will obliterate any cell left in the blood or bone marrow. The stem cells are then reintroduced to the patient which will save them from their now desolated body. Slowly, the stem cells will graft and the patients blood and immune cells will start to reproduce; this process can take weeks, or months. The patient must take heavy precaution from any possible infection risk during this time; fresh flowers, fruit, vegetables, plants, and human contact could cause infection the patient would never recover from -- Isolation is a standard precaution. Once the patient leaves the hospital the recovery is far from over. The body will continue to adjust to its new immune system over a period of about two years, through the first hundred days poses the most risk for the patients recovery. There is a long lists of "do's and dont's" the patient must carefully follow once returned home.

Lingering Questions?
Q: What is the difference between this, and a Bone Marrow transplant?
A: They are the same procedure. In recent years the phase Stem Cell Transplant gained popularity since the hematopoietic stem cells are now more often harvested peripherally. Saving the term BMTs for hematopoietic cells harvested directly through bone marrow aspiration. However most treatment centers still chose to use the overarching Bone Marrow Transplant terminology for both extraction methods. So HSCT/BMT are interchangeable - unless you want to get specific with extraction methods.

Q: Is there a difference between using a donors cells vs your own cells?
A: Yes! Autologous is using ones own stem cells, and allogeneic is using a donors cells. The allogeneic transplant poses a much higher risk of mortality due to the high possibility of Graft vs Host Disease. Both have actually been preformed in autoimmune patients, although there is almost no data published regarding the allogeneic transplants since it has only been preformed a handful of times. In theory donor cells would be a better option as they would not be previously diseased, however; medical journals that can be found regarding allogeneic transplants state, "clinical evidence in favor of allografting is sparse and the risks are considerable."

Q: How can your new stem cells be disease free if they are your own cells?
A: The medication given prior to harvest causes your body to produce brand new, immature stem cells. Think of it this way, I was healthy for 20 years before my adult cells "activated" and autoimmunity began to take over. These new cells haven't been "activated" to attack my body yet. They are little clean slates.

Q: Is a Stem Cell Transplant the same as a Stem Cell Treatment (Therapy)?
A: NO. NO. NO. An outpatient clinic, or treatment center, claiming to use your own adipose (fat) stem cells to help everything from arthritis to T1D is a completely unrelated therapy. Please be aware, a HSCT is preformed by a medical doctor within a hospital and involves high doses of chemotherapy to eradicate an immune system. A 'stem cell treatment' is typically not preformed by an MD, is at an outpatient clinic, and does not involve chemotherapy or irradiation of ones current immune system. The stem cells they use are not from the blood, but from your fat, and are typically injected via a shot or infusion.

Q: Are you killing babies?! (I have actually been asked that..)
A: No. Stem cells are found many places besides umbilical cords.

Q: How long does all of this actually take?
A: If I am accepted into the trial I will live in Chicago roughly two months if everything goes smoothly. However the process could be longer, or shorter.

Q: Does insurance cover this treatment?
A: Yes and No. HSCTs for autoimmune diseases are considered experimental by the FDA and therefor are not covered by most insurance companies. While often, the researching physician can persuade your insurance company to pay for the procedure, some patients are stuck with a 125,000+ dollar bill if they want this treatment -- and that doesn't include travel expenses. Some patient fight their insurance company for weeks, while others fight for years to attain coverage.

Q: Is it considered a cure?
A: No. The HSCT doesn't not cure autoimmune patients. While some patients may find themselves in a blissful remission of disease, they are not without the effects of their disease previous to the transplant. While this is a successful treatment for many, some do relapse after treatment as a consequence of their own stem cells being diseased beyond repair, or the donor cells not grafting properly.

Q: Why would you do it then?
A: As stated before, this is more of a rescue strategy for those with a poor prognosis than an actual treatment. In many cases the patients undergoing this torturous ordeal are left with the two options: 1. Die or 2. Playing their luck with a HSCT. Again, this treatment is for serve disease unresponsive to any treatments currently available. Given the choice, I want to go down fighting rather than just accept my death.

Q: Where can I get more information?
A: To check availability head to and simply search your illness, or hematopoietic stem cell transplantation. It may take some digging, but there are trials still recruiting patients. For medical journals on the HSCT and autoimmune patients simply Google "Autoimmune and HSCT."

Disclaimer: Please remember every patient is different. Some patients bodies may handle a transplant better than others. The disease the patient is fighting also makes a drastic difference in their HSCT experience. I am not an HSCT expert! All of my information is based of information received from my physicians, patient experience, and medical journals I have read. 

Thursday, August 11, 2016

A Towering City Of Disappointment - Health Update

This week I made my promising trek to Los Angeles to visit a prestigious HSCT (Hematopoietic Stem Cell Transplant) doctor at City Of Hope.  A few weeks ago I had contacted this physician by email and he became extremely interested in my case. He asked if I could fly down and meet him in person for in depth consultation. I received a call from a new patient coordinator the next day, received my appointment date, and booked my flight immedietly! Fast forward to Wednesday. I traveled to COH alone because it was a very last minute ordeal. Everything moved so quickly that it didn't give my family adequate time to save for the trip, request work off, or find pet sitters. So, I waltzed into the giant campus by myself, ready to hear some good news from this physician I was so very ecstatic to meet. The paper work said to arrive at 930am for an 11am appointment, so I figured there would be some sort of blood work, testing, or pre-consultation. I arrived promptly and was given a very kind patient advocate, but told there was nothing happening with my care until I saw the physician at 11am.

Wait what?! They expected my already anxiety ridden self to arrive two hours prior to an appointment and just wait?! Why they do that to patients is beyond me...

So I waited, and waited, and waited. By 10:30am I was extremely nervous and antsy, and by 11am I was almost in tears. By 11:20am and still no appointment I was having a full blown anxiety attack and trying not to hyperventilate. I know that all seemed to escalate quickly, but when your life is riding on an appointment and you have had nothing but blank time by yourself to think about it, the mind goes to bad places. I finally went up to a desk and asked what was going on and they just said, "they will call you back shortly." By 11:30am I was called back to a room with a physician who obviously wasn't the one I had emailed. RED FLAG #ONE. This physician told me he was on the same HSCT team and that the other physician I flew all the way to California to see, said this doc could see me instead. Not cool. Since I wasn't about to get up and leave I started asking questions. I wanted to know about their former Scleroderma patients and how they fared during the HSCT. He expressed they had only done three and every single one had ended up in the ICU for upwards of 2-3 months, half dead, drowning of blood in their lungs. RED FLAG #TWO He told me that they were able to pull every patient back from the brink of death however, and that they were doing okay now -- Those aren't good odds if you ask me. 

With in the first few minutes he told me I was a great candidate and fit all the criteria! He actually also expressed that my body wouldn't make it another year waiting for a transplant, and that we needed to move forward as soon as possible. It was an overwhelming YES! However, he then asked when my last appointment was with Doctor X (name withheld) at UCLA -- "Um never." I replied "Why?" He then went on to explain that I cannot have a transplant at COH without being a patient of Doctor X first; that I would have to become her patient if we wanted to move forward. RED FLAG #THREE

....You mean to tell me I was supposed to get an appointment with another physician at a totally separate hospital before coming to see the doctor who I didn't even get to see at COH?! And no one told me that?! 

So even though I was a yes from their transplant team, I couldn't do jack because no one on the team bothered to tell me I needed to first get an appointment with a physician at a totally different hospital. Why the hell did I fly to California then?! The doctor who I was "consulting" with basically just apologized for "wasting my time." and that if I wanted to fly down again in a few weeks to see that other doctor at UCLA I could come back and re-consult the same day at COH. Probably the most unorganized visit I have ever experienced in my life. The entire orchestration was not up to my standards for a hospital that will literally be holding my existence in their hands.

So lets look at these red flags a bit close:
1. I never even got to see the physician I specifically booked a flight to meet.
2. Everyone of their scleroderma patients have been in the ICU on the brink of death.
3. They weren't even organized enough to realize I wasn't a patient of the physician I had to see before I came to them -- even though they had all my medical records. Did they even read them?

For these obvious reasons I am ruling out City Of Hope for my HSCT. Don't get me worng, the physician I saw was lovely and kind; he genuinyl wanted to help but, I need a hospital that doesn't have their head up their bum if I am being frank. I am of course disappointed I wont be able to have the transplant closer to home, but I am also very grateful I had this experience to rule out the poor choice. I will not wonder "what if?" I know that this door is shut so another can open. Thankfully I have been accepted at Northwestern Memorial Hospital in Chicago for a consultation. I should receive a call next week to schedule my tests/appointment. Luckily, it's pretty comon knoledge that that if you are a candidate by STAT trial standards you will most certainly meet criteraia for the ASSIST trial! I of course have to get the absolute yes from the physician, but I don't see how I wouldn't be accepted. Woohoo! I am finally moving forward and am ready to bump up my fundraising goal to assist with transplant costs! Your assistance will help fund travel expenses to and from Chicago, housing for the duration of the transplant process, and medical expenses not covered by insurance. As I am not 100% sure of the complete cost currently, the goal may change in the future. 
Please visit the link to help save my life!

Wednesday, August 3, 2016

Swallow Study - Health Update

Today I had a Fluoroscopy Esophagram, also known as a Barrium Swallow. Basically, it is a giant video x-ray that watches barrium move through your esophagus. I have had many, many, many of these studies over the last four years since my esophageal motility is at such serve end stage involvement. All the radiologists find my imaging incredibly intriguing! lol Today I had the pleasure *insert sarcasm* of having yet another one of these lovely tests done to asses my current situation as I seem to be aspiration more frequently then normal. The radiologist and his team got me all ready for the study and explained the procedure, I expressed to him that I wouldn't be able to do the laying down part of the test and he asked me why. I told him my esophagus has absolutely zero peristalsis to which he chuckle a bit saying "I am sure that's not true! Everybody has some! We will get you better numbers than zero miss." I just shook my head and asked them to start the test standing up. We started imaging with a tiny teaspoon of barium -- then they asked me to swallow. "Swallow honey!" They yelled again, "Swallow!" Well, I was swallowing. "Ohhhh," I hear from the radiology room. "Wow, her esophagus doesn't work at all, are you seeing this?!" I watched as all the interns huddled into the room to view my apparently very exciting failed organ.
They asked me to attempt to swallow another tablespoon of barium for those who missed it -- that was a mistake. By the second teaspoon I started coughing, burping, and grabbing my chest. "Oh wow, we have to stop," I hear from the other room. My bum food pipe which has no muscle tone was unable to empty that barium into my stomach fast enough and it was now all backed up into my esophagus. We stopped the test and the radiologists and physicians had me sit down to rest while we waited for the ounce of fluid to move slowly into my stomach. About ten minutes later, I finally had passed the fluid from my esophagus to the stomach -- which should happen instantaneously.

This explains all my recent aspiration; anytime I consume more than a few ounces of fluid my esophagus backs up almost all the way to my upper esophageal sphincter. All the doctors in the room suddenly handled me with 'little kid gloves' and puppy dog eyes, expressing how sorry they were for the "torture." I just kind of laughed in between coughing fits because I  had already told them how this would end, they just refused to believe me. After that they sent me on my way telling me to never have more than a few tablespoons of anything at one time or I would backup my organs and aspirate. Awesome. Somehow my esophagus has gotten even worse then it was two years ago. I had no idea you could get worse than zero. Urg. So if you're wondering why I am tube fed, it is because my esophagus sucks and cannot handle more than an ounce at a time without ruining my lungs -- and they are already pretty good at ruining themselves! lol

On a happier note I have a date for my first Stem Cell Transplant Consult! August 10th I will meet a physician at City Of Hope in California to evaluate my candidacy for the STAT trial. This isn't a guarantee of acceptance, but simply a meeting to evaluate my condition. If I am accepted I will have to fly back down for rigorous testing to be sure my organs can handle the stress of the transplant.
I am also happy to share that another physician has taken interest in my case! A doctor in Chicago from the ASSIST Trail wants to evaluate me as well. This isn't my first choice due to its lengthy travel, but I do believe the physician is extremely skilled at his craft -- and I would like to see what he has to say about my case. I haven't gotten a date for my consult yet, but I did submit all my new patient paperwork this week. I even received an email from his nurse that stated the physician was "eager" to see me. Hopefully that means soon!

I am extremely grateful to have two physicians interested as I now have the ability to compare experiences and make an informed decision on what I feel will be best for my health. I will keep you all updated as things unfold! Thank you to each and every person who shared, or donated to my You Caring Transplant Fund! You have all made it possible to receive the second consult by funding 600 dollars over my goal! Having two options will insure the best possible outcome for my complicated situation. Thank you from the bottom of my heart!