My Promacta Journey

This week's labs

3/29/17 plt 21,000
dosage 50mg promacta daily

The roller coaster continues. The nurse was hinting that she might like me to increase the dosage, but I said no. I had discussed with the new doctor that I don't want to chase the numbers and continue to have no symptoms. Still hanging out at 50mg and will see what happens next week.

This week's labs

4/5/17 plt 23,000
dosage 50mg promacta daily

Almost the same count as last week. Still absolutely no symptoms. I will call the two week consistency a win. Lots more data points needed to establish a pattern, but honestly I'd be thrilled if I hang out around 20 on 50mg. For my sense of risk of treatment versus risk of adverse event, this feels like it might be a sweet spot.

However, sadly, I live in the real world where I don't get to use the lab to make my own choices. After all bloodwork, I have to see a nurse in the office. Apparently they won't let you get counts below 20,000. Last week she had tried to convince me to increase my medication to 75mg as a result of the count of 21. I demurred saying that I wanted to stay at the same dose for a while to see what my current trend is. There wasn't any more pressure at that point. This week, she tried to convince me to alter the dosage again, and again I expressed my wish to stay the course to establish a baseline on 50mg. She went on to say, "Well, if you go below 20 we will have to transfuse you. It's all fun and games until someone gets a cut." OUCH! Not cool nurse, not cool at all. Does she really think I haven't been dealing with this my whole life and am just messing around? I made some vague comments about how platelet transfusions should only be rescue treatments to stop bleeding in certain circumstances, and certainly not something to use to increase the count in an ITP patient. I'm pretty sure they actually speed the rate of the immune system eating the platelets (right? am I crazy here?) I didn't want to get into it with the nurse about it so I left, but I'm pretty down about that encounter.

I feel like it's hard to find a situation where doctors/nurses trust you to know your body, or are even willing to let you express your wishes about what you are comfortable with. Completely asymptomatic low counts are not a freakout occurrence in my mind. Maybe I err too much on the side of not treating, I don't know. I despair of finding a hemo that will let me have counts 15-30 without panicking and rushing in to throw more medication and treatment at me!

Anyway, waaah. All of you google masters please feel free to pass along pubmed or research articles on why it would be stupid to do a platelet transfusion on an ITP patient who isn't bleeding out. I'm going to have to go back armed with some research. Again, this was all with a nurse, and I hope that my doctor would know better, but I'm seeing lots of red flags.

Mom, you stick to your beliefs, Ask for the evidence base that this nurse is working from. If it's over 5 years old then question it. I'm a nurse (albeit a renal specialist) who thinks the attitude displayed to you is extremely unprofessional. Treating ITP is about treating the symptoms not the platelet count. Anne x

ugh Sorry you had to deal with that. The nurse sounds uninformed and very insensitive! "fun and games?!" omg how irritating. But you had an EXCELLENT response! So you shouldn't feel bad about speaking up for yourself and offering accurate important information. You can only try to educate someone, if they aren't open to it then theres not much you can do. Along with the platelets being destroyed sometimes in just a few hours, I think I read that theres a platelet shortage. ? So many good reasons for using platelet transfusion only in an emergency.

I have refused platelet transfusions several times. Once when my counts were 7K and hemo was out of town. The nurses called me at work and were quite hysterical saying I needed to come straight to the hospital. I said I'm fine, no problem, I'll take a bit more prednisone. They kept calling at least 3 times trying to frighten me. But I kept refusing treatment. THEN the hospital called saying my platelets were ready and when would I be coming in? Ai yi yi I was at work and other people could hear me talking so I had to speak in a very polite calming voice, like "no I won't be needing that order today, thank you" haha

Anyway, you sound very well-informed and sensible. Trust yourself. I totally agree that we have to weight the risk of low counts with the risk of treatments! So true! I have had counts of 1K, 2, 3K and never had a problem. So in my case, I'd like counts of around 50K but if it means more drugs, more side effects, I am comfortable with lower counts. And as I've learned from others and my own experience, when dosing the TPOs- Pick a dose, stay with it, give it time to work.

Thanks Anne and poseymint!
I appreciate the support and confirmation that I'm not just a difficult patient

I am looking around in my area to see if I can find someone who is more specialized in ITP or thrombocytopenias in general. I think I found a doctor in Baltimore through Johns Hopkins who at least lists thrombocytopenias as a specialty. It will be a bit of a hike (I'm in the DC area), but for a one or two time consult it would be worth it. I just want to find someone knowledgable, but I hate how in this country we just have one big lump group of "hematologist/oncologist" and no good way of finding out each doctor's particular interest. Someone who is primarily an oncologist isn't going to be up on the finer points of treating ITP. Lots of hemos may treat ITP but primarily deal with patients who are very responsive to steroids and aren't refractory. Ugh, it's frustrating.

Thanks again for the support! I appreciated reading it after my depressing visit! I'm going to stay the course with what I want to do and just go doctor shopping if my current doctor doesn't listen to my concerns, or talks crazy about platelet transfusions.

LOL, I used to score '12' often. The first time it happened they told me to go get a platelet transfusion. I said I don't see this as a medical emergency. End of conversation. Realize that I've had 'easy bruising' for at least the past decade (before being diagnosed), and, that I have read it is indicated only for emergencies with ITP. On my second '12' score I went ahead with it, but only because I wanted to talk to a doctor because of the ulcer I got from my first and only Dex pulse. Since then, they've never even suggested to get a transfusion. They know where I stand and I know where they stand.

Keep in mind that the nurse has instructions from the doctor about what to do with a low count. IMHO, let her do her job. You are the patient/customer and are the ultimate decider in all treatment decisions. Both the doctor and the nurse, despite some scare tactics, should realize this.

LOL, on the other hand, you are pushing them with a non main stream idea - a count less than the normal 50 target with Promacta - right off the bat. As an old boss used to explain, you have to be really slow and gentle about things when you set out to change someone else's mind.

Not a count update, but an interesting discovery I had as a result of poking around on the PDSA website. It turns out that one of the Medical Advisors (Dr. Kessler) is based out of Georgetown in my area. I'm going to try to see if I can get in with him (I'm guessing there will be a crazy wait for an appointment as a new patient, but it's worth a shot). Surely one of the PDSA advisors will give me better advice, so I'm really hopeful about that. I'm going to find out tomorrow if seeing him is an option.

Fingers crossed that my count on Wednesday isn't technically below 20, otherwise I will be triggering the nuclear option at my new office and have to fend off their desire to throw platelets at me! How arbitrary that a 19 will sound the alarm, but a 20 means I go another week in peace (both with no symptoms!). Argh, it's maddening.

That's a great idea! Even if it takes a while to get in at least you'd get another opinion from someone who knows what they are doing. Keep me updated!

The experiment's afoot!

Today's lab results (using fingerprick blood)
4/12/17 plt 45,000
dosage 50mg promacta daily

They also used venous collection for a sample which will be sent out and I should get that count in a few days. I'm pretty psyched with a 45 via fingerstick after the last two counts in the low 20s. I am guessing that fingerstick results are just all over the board depending on clumping and how quickly they get blood out versus how quickly you start the clotting process. It would be just my luck if the time I am testing both I get comparable values, but we'll see. The phlebotomist did make a comment that "you know that the two values will be different, right?" I had to smile while I was thinking to myself, "uh yeah, that's kind of the whole point of this exercise. I don't want to get threatened with treatment with incorrect numbers!"

I somehow didn't have to see the nurse today. Maybe my weird lab request has somehow marked me as a difficult patient. The lady in the lab seemed a little bit scared of me :huh:

I'll keep you guys posted about the results of The Great Bloodwork Comparison, Part II!

(Oh, and in other good news, it looks like I might be able to get an appointment with Dr. Kessler. We are waiting on records and then I should be able to make an appointment. Fingers crossed!)

Two sticks - ouch. I'd actually love to see the difference between a finger stick, an instant count and a sent-out lab count. I think the counts are not quite as accurate when they sit around as opposed to the instant analyzer count. The blood starts to clot when it sits, even with the EDTA tubes. For a while, I couldn't get a count at all when it was sent out because of clumping and had to start getting instant counts.

Good news on the second opinion!

Today's lab results (using fingerprick blood)
4/20/17 plt 49,000
dosage 50mg promacta daily

Slight uptick from last week's count of 45. Lots to update for me this week! I got the lab work back from the venous collection performed last week and, wouldn't you know it, it was the same number (45). Sandi, you got me thinking about clotting since it was a "send out," and I'm really missing my old office's in house instant CBC machine that used venous collection. If only I could compare them all, lol! I actually had my appointment with the doctor today, and discussed my concerns about the "platelet transfusion" discussions I had with the nurse. My doctor said that no, there wasn't any worry about my being sent for a platelet transfusion at any arbitrary number, particularly with no symptoms. I'll give the nurse the benefit of the doubt, even though she was snarky, that she wasn't paying attention to the fact that I have ITP, or was in a bad mood that day or something. As long as she can't influence treatment decisions, I really don't care. I may request venous collection if I get any crazy low counts in the future, but frankly my experiment was more about me not being forced to alter my treatment over low numbers. My doctor seems reasonable about not being hasty in changing treatment, so I'm good with that!

I had some funky pains in my left leg, that started as weird pains in the lower leg and have also developed a Baker's cyst in my left knee. After doing some reading, I was getting a little bit paranoid about DVTs and the potential for pulmonary embolisms (thanks for the fears promacta!), so I went in and got an ultrasound performed on both legs to check. Since I've been doing promacta for about a year now (right? I need to look back and see when I first started posting about it, lol), I figured getting a check was good since I was having funky symptoms. No clots spotted, yay! I went to an orthopedics guy and got x-rays, which were fine, and will need an MRI to look at all the soft tissues to see why I've developed this Baker's cyst in the knee.

Final medical update (I'm writing a book this week, whew!), I got an appointment with Dr. Kessler! I'm really excited. He reviewed my records and is willing to take me as a patient, so I'm going to have an initial consult with him in June. Apparently he is really thorough and takes a long, long time with his patients, so this sounds great! Even though things are going well for the moment at my new office, I'd love to have some expertise thrown into my case and treatment. Really good news all around here from me. Lots of platelets (but not too many!) to all of you.

That all sounds great!

It IS good news! Perfect counts, new doctor! I can't wait to see what he says.

This week's lab results (using fingerprick blood)
5/3/17 plt 24,000
dosage 50mg promacta daily
Low-ish number today, which I still don't totally believe because of my continued suspicion of fingerprick blood.

Still asymptomatic, so I'm staying the course. I had to show my results to that same nurse who didn't like my treatment plan before and got some passive agressive behavior, so I'm going to steer clear of her in the future! I'm so thankful that she doesn't influence my treatment decisions

This week's lab results (using venous collection blood that was sent out to a lab)
5/16/17 plt 64,000
dosage 50mg promacta daily
Still mostly asymptomatic (had a random blood blister or two in the middle there, but obviously things popped
up by the time I had my blood work done). I was having blood drawn for a yearly physical, so I had them do a CBC
at the same time to save me a trip to the hematologist. Count is perfect for Promacta, and I'm sticking
with my 50mg daily no matter what happens to see if there is a trend or stabilization. Oh, and I got my new
primary care doctor to do a liver check for me as well, since my new hematologist hasn't been on top of managing
my Promacta (I don't think she has any other patients on it) and I haven't had a chance to bug her that I'm
due for a liver panel. All good with that, so good news from me this time around.

Waiting on my appointment with the specialist at the end of June

Great news!

That is a great result! I'm really pleased for you

I can't wait to hear how your visit with Dr Kessler goes!

I didn't know they could do finger pricks... the places I go to take 3 vials and analyze them in the office right then and there, so presumably they're fairly accurate. I would be skeptical of the finger prick instant analyzers too. You always hear about the potential for false negatives on stuff like that, whether it's strep, or tests for UTI, etc.

Congrats on the latest count!

This week's lab results (using fingerprick collection)
5/31/17 plt 61,000
dosage 50mg promacta daily

Still doing fine here and just waiting for my appointment with the specialist.

This week's lab results (using fingerprick collection)
6/14/17 plt 13,000
dosage 50mg promacta daily

Yikes, that's a low one for me. Still no symptoms of any kind, and I feel like the lab technician was VERY slow in collecting the blood after the stick. Again, I don't know that I believe all of these fingerprick numbers, and this feels like an outlier. If anyone at the office sees this count they will probably try to hunt me down and mess with me. It's ramadan and I've been fasting which has been a shock to the system in terms of eating/sleeping schedule as well as when I take my medicine, so I will probably have to cut that out. Even if the count isn't really 13k, it's surely low.

Just biding my time until the appointment with the specialist (1 week!), so hopefully I can avoid the current office. Coincidentally my new hematologist is having a baby and is probably in the process of dealing with her delivery right now. Is it bad that I'm hoping she won't notice the count?! :lol:

If I trusted my labs, I'd be more concerned I guess, but I think I can hold out for 1 week to get better advice and hopefully an instant read venous collection count. Oooh, the rolls royce of collection methods. I'm a bloodwork snob now!

Yea, ensuring the bio availability is a real weak link. What I've been doing is drinking a good amount of water over the 4 hours before taking it. Make sure everything is washed down. No pesky 'cat' ion to neutralize it...

I will write an update since I got to have my initial appointment with Dr. Kessler.
It's a whole new world out there! I've never had a doctor's appointment that went as well. It is such a pleasure to finally find someone who so fully understands the condition and someone that I don't have to watch out for to harm me with bad treatment decisions. Relief!!

He was AMAZINGLY thorough (there was quite an initial wait because he is apparently famous for taking a long time with each patient), and I would say that I was probably in the exam room discussing things for at least 45 minutes, maybe even closer to an hour. Going through my full history and current treatment plan. We are going to start from scratch and check for other autoimmune conditions and things like Von Willebrand's Type 2, etc. Since ITP is a diagnosis of exclusion, we'll do a thorough check to make sure that we know what we are dealing with. Nobody has thought to double check my diagnosis from when I was 8, so this seems like a great first step to make sure no new autoimmune things have popped up in the interim!

We talked about things that I should have been doing all along in splenectomy management (always taking antibiotics prior to getting dental work done. Oops, missed that memo when I had the surgery done as a child!) And he will do a blood smear to check for evidence of any accessory spleens that may have generated over the years. We also talked about how the splenectomy may have affected my ability to be successfully vaccinated and ways that we can determine if I have the right signs of having had the Pneumoccocal vaccine and others in my system. Ugh splenectomy, boo! So many factors to consider.

He agreed that my current fingerprick data set isn't of value (I was right!) and I am going to go back today to get a whole world of bloodwork done. We spent so long in consultation that the lab closed while I was there. I'm still amazed how thorough he was, I've really never had a doctor spend more than 15-20 minutes with me. He coordinates a lot of the research that they do at Georgetown and he was hinting at some really exciting findings and new directions in ITP management in the work that he's doing. I'm sure everyone going to the conference will hear lots of amazing things. I really wish I could go and hear him speak along with other experts in the field. I just didn't know how much I was missing in dealing with my previous hematologists.

So, I'll be ditching my current office (even their bloodwork doesn't give me any benefit), and I'm going to do my initial few data points over at Georgetown to make sure we can move forward with the best information. I will follow up with him in about a month, and then we can make some decisions about moving forward. I will probably not be continuing Promacta because of my erratic count history on it (considering the results I was getting back at my previous office, which are not suspect values). I am going to stay on my 50mg for the moment so that coming off of it doesn't unduly affect my results for the next month. I am also going to follow up with an ophthalmologist (which apparently I should have done prior to going on the medication) to evaluate my cataract situation. People on promacta apparently develop early cataracts that are different in nature from normal cataract development, and I need to get a baseline now so that if things are going on with my eyes (I'm highly suspicious because of my weird eye symptoms from when I was on 75mg) that would also be a contraindication for future promacta use. Interestingly, his office is always running trials for new things, and they were one of the places that did the initial promacta trials. So I definitely felt good that he knew what was going on with that, lol!

Counts after I achieve some kind of stability can be done with an order at my local LabCorp. No more need for extraneous hematologists.

So overall, absolutely amazing. I feel validated in all of my previous concerns, and I finally feel like someone has my back in dealing with my condition and treatment. I don't know that I've ever had that feeling. HIGHLY recommend anyone to follow up with any of the PDSA medical advisors. They really do know what they are doing, and ITP isn't like cancer (the specialty of so many hematologists)

I'll update my thread when I get my counts back from bloodwork today regarding where I stand now.

Wow -sounds great! Since you were diagnosed as a child, it makes sense to look at other disorders.

Some exciting research being done!

momto3boys wrote: [Dr. Kessler] coordinates a lot of the research that they do at Georgetown and he was hinting at some really exciting findings and new directions in ITP management in the work that he's doing. I'm sure everyone going to the conference will hear lots of amazing things.
... Interestingly, his office is always running trials for new things, and they were one of the places that did the initial promacta trials.

The only recent publication I found co-authored by Dr. Kessler regards a TPO similar to Eltrombopag, but without the dietary restrictions, called Avatrombopag:

www.bloodjournal.org/content/123/25/3887
A randomized trial of avatrombopag, an investigational thrombopoietin-receptor agonist, in persistent and chronic immune thrombocytopenia
James B. Bussel, David J. Kuter, Louis M. Aledort, Craig M. Kessler, Adam Cuker, Kelly B. Pendergrass, Shande Tang and Joe McIntosh
Blood 2014 123:3887-3894; doi: doi.org/10.1182/blood-2013-07-514398
Key Points
Once-daily oral avatrombopag dose-dependently raised PCs over 28 days, with stable counts maintained over a 24-week extension.
Low rates of severe AEs and study drug discontinuations due to AEs occurred despite dose increases in maintenance.

The product has been spun off to a private company, which has just announced an IPO:
www.heraldsun.com/news/business/article154663674.html
Durham-based company files for IPO worth up to $75 million

I also found a very intriguing clinical trial (Phase I/II trials) being run partly at Georgetown for a drug that appears new and different for treating ITP. Drew Provan is involved in the UK arm of the study . The drug is identified as BMS-986004 (Bristol-Myers Squibb) and elsewhere online as Letolizumab.

www.cancer.gov/publications/dictionaries/cancer-drug?cdrid=773819
anti-CD40L Fc-fusion protein BMS-986004
A dimeric fusion protein composed of the C-terminus of the domain antibody (dAb) BMS2h-572-633 targeting the CD40 ligand (CD40L or CD154) linked to a modified Fc fragment of immunoglobulin G1 (IgG1), with potential immunomodulatory activity. Upon intravenous administration, the peptide moiety of anti-CD40L antibody BMS-986004 specifically targets and binds to CD40L expressed on T lymphocytes. This prevents the binding of CD40L to its cognate receptor CD40 expressed on B lymphocytes, macrophages, and dendritic cells (DCs). This prevents T-cell mediated proliferation and differentiation of B cells, and prevents the production of antibodies. By inhibiting both the production of anti-glycoprotein (GP) IIb/IIIa antibodies by B cells and GPIIb/IIIa-dependent T-cell proliferation, BMS-986004 may prevent platelet destruction and may increase platelet counts in idiopathic thrombocytopenic purpura (ITP). The direct binding of BMS-986004 to CD40L on platelets further prevents CD40L/CD40-mediated destruction by macrophages and DCs in ITP. The modified Fc domain prevents the binding of BMS-986004 to the Fc receptor FcgammaRIIA on platelets, thereby preventing FcgammaRIIA-dependent platelet activation and anti-CD40L-induced thromboembolism. CD40L, a transmembrane protein of the tumor necrosis factor (TNF) superfamily, is primarily expressed on activated T cells, but is also expressed on eosinophils, basophils, natural killer (NK) cells, mast cells, platelets and activated endothelial cells. Check for active clinical trials using this agent. (NCI Thesaurus)

Thanks for doing the searching Rob! I'm going to go try to decipher the second article, it sounds interesting.

We are so spoiled with your amazing research googling skills; I really appreciate it!

You out did yourself on that one Rob. Pit bulls may not take days off, but I'd take a day off after that one.

The first sentence is quite interesting.
"
A dimeric fusion protein composed of the C-terminus of the domain antibody (dAb) BMS2h-572-633 targeting the CD40 ligand (CD40L or CD154) linked to a modified Fc fragment of immunoglobulin G1 (IgG1), with potential immunomodulatory activity.
"
Sounds like they were sifting through IVIG components and found one ingredient that naturally corrects one's immune system in regards to "anti-glycoprotein (GP) IIb/IIIa antibodies". Oh my, very cool. LOL, I bet there are all kinds of stuff in there that helps a good many of the auto immune 'disorders'.

Notice that this is from the National Cancer Institute. Perhaps this effort is being funded by cancer research - which one would think has a vastly larger budget than a drug company developing break through drugs.

Mom, this will be great. You can tell us about all the cool new studies you are going to participate in, errr ahhh I mean, all the cool new studies the doctor is working on.

Nice find Rob..anything without the dietary restrictions of Eltrombopag is worth looking at. Having said that I have got to grips with them and am coping but would love to live without them

I finally got my previous lab results when I went in for more bloodwork this week after returning from some travel
6/22/17
plt count: 98,000
eltrombopag dosage: 50mg daily

So that's a lot different range of number than I was ever getting at the fingerprick office. But as we all know, platelet counts bop all around, so it could also be a blip. When I go back in about 2 weeks to visit with the doctor again, I'll find out what my numbers were from this week and that may tell me more if the recent count is also high. (If that is the case, I will never trust fingerprick numbers again, lol!)

Since they gave me the printout of my previous lab results, I can see all kinds of results for different antibody testing IgG1-4, IgA, IgM, S. Pneumo IgG Type 26, 1, 12, 14, etc. etc. so many interesting things were being tested! I have to google around and see what all of this means (some out of range high numbers on those antibody tests, so definite autoimmune issues from what I understand in my initial 5 minutes of searching).

I'm excited to follow up with the doctor and really dig into the meat of what to do about me and what these results indicate. It's very exciting to have so much data to work with. I just need to get myself up to speed on some of these measurements so I can better understand the conclusions the doctor may come to.

I had some of those tests years ago, but didn't pay much attention. Some were elevated. I'd love to do them again.
It's good that you had those tests run and maybe something will come of it. Were you tested for the MTHFR gene mutation?

Just came across this regarding finger prick testing
www.ncbi.nlm.nih.gov/pmc/articles/PMC4785771/