Anything new out there or on the nearish horizon?

I've been away awhile, the Nplate worked so damned beautifully for about 8+ years, but over the last few months it's been slowly crapping out on me. I'm hospitalized since Fri., when my count was 4. Where a REALLY low dose of 110 worked for nearly a year, Fri. I got 1000, PLUS IVIG (70), PLUS 125 of solumedrol. Sat. I hit 14 but with no hematologist on call, they kept me one more night with no more meds. Today I was at 5. IVIG had not worked for me in the past, nor had steroids, so the only ones surprised were the docs, but now what? Splenectomy failed, promacta failed, rituximab failed, danazol failed, cytoxan failed, I think I even tried Winrho but I'm not positive, but if I did then it must've failed too or I'd remember. Is there anything else? Or am I screwed again, and back to life in the single digits? If it's that, I'll try to inspire with my vim, if not my vigor. But DAMMIT, I had rollercoaster plans this summer.

There are a three things that come to mind. None of them are particularly enticing. Nevertheless, here goes.

From what you've described, you response pattern appears to match row 2a in my treatments table . This table was created by reading and grouping together PDSA user accounts of what did and did not work. As you can see, row 4 isn't that different than row 2a. You can make your own estimation of which applies, or, if none applies.

1. A new drug is Fostamatinib. It appears to work on either a row 2a response or a row 4. One, and only one, of the two. Don't know which yet, not enough user reports. It is now available for 'compassionate use' as described here:
pdsa.org/products-a-publications/e-news/2018-enews/item/1480-pdsa-enews-013118.html#compassionate-use-treatment

2. As far as your Nplate response, it is possible/likely that you have developed antibodies to it. It doesn't happen often, but it does happen. There is one user report who has suggested Rituxan can be used to rid oneself of antibodies attacking Nplate. This was 'lucidawn' and her son Tim. Note that she reports that Nplate must be given simultaneously with Rituxan. Here is one relevant comment. If you read all her comments what she was right about was that the two had be administered at the same time.

3. There seems to be some evidence that 'Tamiflu' can raise counts (no remission) with row 2a or 2b folks.

If row 2a is actually the case then AFAIK Imuran, Cellcept, and Cyclosporin will likely not work. If row 4 is actually the case then AFAIK Cyclosporin will likely work.

Hope this helps.

When I click the links I get a blank new tab, no table. It may be because I'm on an oldish notebook but it's all I have at the hospital, but wow Hal, it sounds like you've created quite the valuable resource, thank you for your work! I'll ask the hubby to bring my laptop tomorrow.
I have insurance, will that affect any "compassionate use" requirements? Is compassion even permitted anymore, I live in Virginia and we seem to be the "die in the street" state sometimes. (Most times.). Hell, when I called them to find an in-network therapist, they told me "Greensboro" (NORTH CAROLINA- 147 MILES away)- wha...?!??!!!?!?!? Oohhhhh kayyy... compassionate much? Apparently not.
But I digress, sorry. Your summation is, um, helpful (VERY!), but a touch disturbing. I suspect I'll have to get myself sent to UVA to find a doc willing to try anything even slightly unconventional, I'm at the Centra hospital in Farmville, VA and let's just say that one rarely hears the words "Farmville" and either "progressive" OR "compassion" in the same sentence. Prince Edward County is where they effing CLOSED THE PUBLIC SCHOOL SYSTEM DOWN in '64 rather than integrate. The whites in the county started a private school and the black children who did not have family elsewhere that they could go stay with had NO SCHOOLS for, I think it was 2 years. Anyway, more digressing, but going "off label" in any way makes 'em all nervous as a cat in a room full of rocking chairs, sheesh. Dr. Bussell used to be my "ace in the hole" but with him retired I don't know where to turn. Here. So I'll reply further once I see the table, but thank you for the resource no matter what.

I was enrolled in the UK Fostamatinib trial. Did absolutely nothing for me. My co 'guinea pig' had a successful trial but it failed her too after she had been on it a year.

I'm feeling torn. I've done "it works/it no longer works" enough, I honestly think I'd rather just not do anything than get back on that ride- it is NOT what I meant when I said I had roller coaster plans this summer, arrgghhh. I think it's been 5 years since Joan Young first told us about the progress in gene therapy, can they not yet run simulations on our genes and find out what kind of odds we've got for a given therapy? I've had this damn disease more than 32 years, I am so OVER the whole thing. When I had my brain bleed almost 11 years ago, I did a bunch of research on how this disease impacts life expectancy and seemed to determine that I'd lose 20 years, based on dx. at age 30 with no meds working and having a brain bleed. So then I did the websites that give you your life expectancy based on lifestyle and family history, and came up with 72. Minus 20=52. I'm 61. I've beaten the crap out of the odds but I'm TIRED. Tired of ITP, tired of fighting and failing, tired of my three year hunt for a therapist (so far), just plain TIRED.

Oh, poor me. Pity party over, sorry. I should be happy, they just told me my count hit 22 and they're releasing me. I can practically hear them- "She's over 20, get her out of here quick before she falls again!". I'll see my hematologist tomorrow and find out if it's gonna last, but I also just got another dose of solumedrol so it's hard to say, IF I still have platelets, what did the job.

Not a happy girl. A sad, sad girl. Dang it. I hate sad, I love happy. Poop. OPM (Oh, poor me).

Not sure. I thought 'compassionate use' might mean they, the drug company, will give it to you if nothing else works. Maybe all the doc has to do is fill out the paper work? I dunno.

Between a brain bleed and Tamiflu, I think I'd take the Tamiflu without reservation. Like other ITP drugs though, best to try it when there isn't an emergency to know if it will work.

Yea, finding an open minded doctor that would be willing to administer Rituxan and Nplate together might take some investigation. If it worked that would be instant 'happy girl' wouldn't it?

Not sure what would be blocking the link to my table. Maybe doing a right-click over the link, then 'copy link', then paste it into a new tab would work.

Oh my gosh - you're THAT Liz! Liz from way back in 1998 when the PDSA began! Well, hello woman!

Sorry that you're back, but I did miss you!

Before you jump on that Tamiflu train, I've read some horrible things about it this winter.

Yep, it's me. I'm baaaaack!! Dang it- nice to be remembered though. So, Tamiflu? Huh? Talk about "off label", what's the deal with Tamiflu?
I'm at the hema now, my count is up to 33, I'm getting a final dose of IV solumedrol, then 4 days of 100mg/daily pred (EF me, dammit), and back here Fri. for Nplate again and to learn the pred taper schedule.
Gah, what a nightmare. I've got to get my brain back to a place of gratitude, I griped too much about weekly doc visits and the cosmos listened and said "OK, we can put an end to them, how about if the med stops working. Happy now?". My life is largely so good, I have a loving husband and a good, good support system of friends and family, lots of 4-legged love from kitties and dogs, that's the Stuff that matters. Gotta get back there. I think this summer might be a nice time to see Cleveland!

I think the last time you were here was around the time you got married. Glad to hear that everything is going well.

About Tamiflu, I've been hearing about horrible psychiatric side effects. People become suicidal, some have killed themselves after only one or two doses. Some people have become horrible ill (severe vomiting). I'm pretty sure they took it off of the market in Japan.
psychoticdisorders.wordpress.com/2012/12/03/abnormal-behavior-and-tamiflu-psychosis/

Well that doesn't sound fun at all. I've really been struggling with depression for the last several years, and I get abdominal migraines already, so neither one of those side effects sound like a good fit. Reckon I'll look at some other options first if I have to. I'm still hoping the Nplate will rebound, any stats on how often that happens? That it craps out and then starts working again?

Yes, it happens. Have you started any new medications in the past few months? Anything different?

When did your counts start to go below what you think are acceptable? What was your N-Plate dose?

Just my two cents-- I did have some experience with Promacta 25mg working for me- then NOT working at all, nothing. And then a year later it worked! My counts came right up within a week- I was surprised! Its so complicated how these TPOs work. I think its not necessarily anti-bodies, I think there could be other reasons a drug quits working, but I'm no scientist.

I have been on Nplate for several years. I have crashed on it quite a few times- down to 1K 3K, 5K. Sometimes I have come back up by leaving the dose the same (I'm at 408mcg) Other times by raising the dose. But when the dose is raised, it takes several weeks for my counts to come back. Good luck! You are a good writer, very funny- keep your humor up!

Geez, what's NOT different? I was on topiramate for something else and after a year-ish realized that what I'd thought was some dry eye was in fact the vision in my left eye trashing, from 20/150 like my right eye to 20/375, effing blinder than sh*t! Called the eye doc, when he got the med list he flagged the topiramate (AKA Topomax) and I stopped it but he also THINKS I have glaucoma in that eye, so one follow up a month ago and another in 4 months, and weird uneven vision that looks to be forever.
Stress beyond words, I won't even start. LOTS of stress. TOTAL FAIL in finding a therapist, even my insurance co. says the nearest in-network is in another state, "147 miles from you". WTF? Wow, welcome to Virginia, the "Die in the Street" State. Sheesh.
None of these things exactly corresponds with the Nplate fail, but I'd been on a really low dose, 110 when I weigh 200, for about a year and it was keeping me consistently above 50. Sometimes 52, sometimes 240, but good and my goal has always been to be on the LOWEST POSSIBLE EFFECTIVE DOSE, so 110 was good, but then I crashed to under 20 and we began incrementally raising the dose, at first by 50-ish and then by 100/WK. til finally we went from 750 to 1000 in a week, last week, and I should find out tomorrow what that did. Nothing FAST, which for me is very unusual. I usually respond to a dose change quickly, like overnight.
When this all stops so have some of the biggest stressors, so that part is good now. And I'm chillin', whether I want to or NOT (DAMN pred!!), so that's probably good too, but man, I hate this part of the ITP ride. After 31 years diagnosed you'd think... I don't know what you'd think. That I'd be used to this sh*t? NOPE. That ITP would be over me? NOPE. That I'd have more sense or patience maybe, but a BIG NOPE on that one. Grrrr. You'd THINK they'd have a better treatment, a cure or something, but that's pretty ambitious. Dang. OPM.

Liz:

I wouldn't give up on it just yet. Rebounds have happened. Let us know how it goes tomorrow.

As far as life expectancy, ITP itself doesn't take years off, but the treatments can. You are right to always take the lowest possible dose to maintain counts.

Hey there- Jeff got me back on as "me", Sandy can you change the Lizabeth1 posts to libelder or shall we just not worry about it? I'm fine with the latter, it's all me either way.

So, today- count was 54 which we both think is steroids and not Nplate. He said I can stop taking the pred., he wants me in on Tuesday (their first day of the work week) at 8:30am for a Rituxin drip which he's willing to give simultaneous to the Nplate, so we held off on todays shot- giving me the weekend to figure out from you guys, do I stick with the most recent and highest dosage I've ever used, 1000? I weigh 200 so it's not THAT high, and by itself it's also not working, but up until this little crapout, 110 worked so for ME, it's massively high. Do I risk hitting 800? I mean I know it is a risk that I may hit 800 if the Nplate/Rituxin combo kicks the Nplate back into gear, but is it a worthwhile risk or should I ask them to cut the Nplate dose at the outset? My inclination is to take the full 1000 on Tues. and see where it goes from there, not put the cart ahead of the horse as it were. But y'all are the ones who may have some experience with mixing the two meds so you tell me, is there a precedent for lowering the dose?

He wants me to do the Rituxan for 4 weeks and he's leaving it up to me if I do the Nplate the whole time too, or not, but either way- if it works then groovy, crisis averted, Lib's a happy gal again, BUT if the Rituxan fails, it's unlikely there is a drug study within the state of VA and with all the meds I'm doing and trying, even more unlikely I'll be eligible for one if we find it, SO- next up in doc's mind is immunosuppressive chemotherapy. He seems puzzled by my reluctance. (BONE HEAD) Anybody got anything good? I ended my "Yeah, maybe NOT" with "but we'll cross that bridge if we get to it, it's too soon to go down that road" and that calmed his feathers, but no foolin', I'm not going down that road and if I turn out to be the first ITP patient he loses well then doesn't it just suck to be HIM, but no. NO. Not going back down that failed, awful, tried and failed path- no immunosuppressive has ever worked for me for management and I'm not going through that sh*t again to prove it to him. VERY short term crisis intervention is one thing, but management? With steroids or worse? Yeah, NO. I'll start carving on my hand hewn coffin tonight, NO.

So there's that. Thanks for the forum, for letting me vent and rant, my husband is of course fearful but trusts me to know best because he is ALL NEW to this, and I'm not. Ditto friends, and family know better than to try and tell me what to do so they just send loads of love and I am grateful for it, but it does help ME, to be able to voice these angry reactions and I appreciate y'all allowing it. I'll be fine- right now, nap time. AGAIN. O, PM!

Lib, I can't change the name. I don't have the capability to edit that as far as I can see.

I'm not sure what to tell you about the treatments. It's always confusing when two treatments are done at the same time because you don't know what is working. There is also the possibility that counts will go too high which can be risky while using the TPO's. Have you ever done Rituxan before?

I did do Rituxan, but I think not four times... two? Jesus, after 31 years I'm not sure- if I did try it though, it was a fail or I'd have remembered. Hubby will drive me in and pick me up so I don't have to worry about if I feel crappy, although doc say "Oh, no, no side effects at all"- do they ALWAYS say that? I do usually do alright with side effects though, except pred of course.

No worries on the name, I'll be posting as libelder now and I'm sure folks will figure it out.

I'm off to nap again. Love that sleep number bed, I'd be a mess without it!

Lib:
Usually Rituxan goes pretty well for most. You feel sleepy from the pre-meds, but that's about it. When I had my fourth treatment, I had a fever during the night but was still okay to work the next day. I did have a rare side effect (serum sickness) which was horrible. If a person is going to have it, it usually hits around the time of the third infusion (right before or after). It's a delayed hypersensitivity response.

I would have known you right away as Libelder....didn't catch the Lizabeth name.

libelder wrote: So, today- count was 54 which we both think is steroids and not Nplate. He said I can stop taking the pred., he wants me in on Tuesday (their first day of the work week) at 8:30am for a Rituxin drip which he's willing to give simultaneous to the Nplate, so we held off on todays shot- giving me the weekend to figure out from you guys, do I stick with the most recent and highest dosage I've ever used, 1000? I weigh 200 so it's not THAT high, and by itself it's also not working, but up until this little crapout, 110 worked so for ME, it's massively high. Do I risk hitting 800? I mean I know it is a risk that I may hit 800 if the Nplate/Rituxin combo kicks the Nplate back into gear, but is it a worthwhile risk or should I ask them to cut the Nplate dose at the outset? My inclination is to take the full 1000 on Tues. and see where it goes from there, not put the cart ahead of the horse as it were. But y'all are the ones who may have some experience with mixing the two meds so you tell me, is there a precedent for lowering the dose?

He wants me to do the Rituxan for 4 weeks and he's leaving it up to me if I do the Nplate the whole time too, or not, but either way- if it works then groovy, crisis averted, Lib's a happy gal again, BUT if the Rituxan fails, it's unlikely there is a drug study within the state of VA and with all the meds I'm doing and trying, even more unlikely I'll be eligible for one if we find it, SO- next up in doc's mind is immunosuppressive chemotherapy. He seems puzzled by my reluctance. (BONE HEAD) Anybody got anything good? I ended my "Yeah, maybe NOT" with "but we'll cross that bridge if we get to it, it's too soon to go down that road" and that calmed his feathers, but no foolin', I'm not going down that road and if I turn out to be the first ITP patient he loses well then doesn't it just suck to be HIM, but no. NO. Not going back down that failed, awful, tried and failed path- no immunosuppressive has ever worked for me for management and I'm not going through that sh*t again to prove it to him. VERY short term crisis intervention is one thing, but management? With steroids or worse? Yeah, NO. I'll start carving on my hand hewn coffin tonight, NO.

So there's that. Thanks for the forum, for letting me vent and rant, my husband is of course fearful but trusts me to know best because he is ALL NEW to this, and I'm not. Ditto friends, and family know better than to try and tell me what to do so they just send loads of love and I am grateful for it, but it does help ME, to be able to voice these angry reactions and I appreciate y'all allowing it. I'll be fine- right now, nap time. AGAIN. O, PM!

Hi Lib,

I live in Northern Virginia, so I will wave at you . It is never fun when something that has been working seems to start crapping out on you. I'm sorry to hear that NPlate isn't giving you the response that you once had.

Regarding a path going forward, my advice would be to not mix medications. I'm currently doing treatment with Promacta (the other TPO-RA medication), and am an "ITP Lifer" like it sounds like you are. Been dealing with ITP for 34 years or so, and have done various treatments here and there (including being given a splenectomy as a child when there weren't many options beyond IVIg, steroids, or splenectomy). Because of my splenectomy, I am very concerned about the higher risks of blood clots (thrombosis) with higher counts on TPO-RA medications. I like my counts on the lower end of the range, between 30K - 50K to try to absolutely minimize my risk of stroke/clots as well as to use the minimum amount of medication possible. If you don't want to give the NPlate more time to work or for your counts to perhaps creep back up towards 50k (maybe you could get some kind of a response, even if it isn't your usual quick, high response?), I would definitely stop treating with NPlate before starting Rituxan. Once you start mixing things, there are two complications. One, you may experience a significant count increase with Rituxan. This would be great on its own, but not great if you are simultaneously treating with NPlate which has greater risks of strokes at high count numbers. Two, the risk of developing some kind of a response but not knowing exactly what is causing it would be frustrating because it will lead to you doing more treatments than necessary (and as we know the treatments are the primary danger with ITP, not the bleeding). You won't know if it's Rituxan or NPlate kicking back in.

One other thing of note about Rituxan, make sure that you consider the fact that you won't be able to receive any immunizations for 12 months after treatment. Immunizations will not take because of the damage Rituxan will cause to your immune system. For most people, this is probably not something to consider, but if you like international travel to places that may require immunizations, it's something to think about.

I'm getting the impression from your posts (and I could be wrong!) that your doctor is a little bit pushy or in a hurry to do things. All too often, hematologists are in a big hurry to treat and meddle with things before giving you time to process and make a decision you are comfortable with. If your current count is in the 50s, there is no absolute urgency to hurry hurry and start Rituxan immediately. It's all about the symptoms and if you aren't having bleeding, take your time to make the best decision about what you want to do next without unnecessary pressure.

I don't remember where you said you are in VA, but my awesome hematologist (Dr. Kessler at Georgetown) is running multiple trial studies for ITP patients who are "tougher cases" and don't respond to the common treatments. There are options for different kinds of trials if they are accessible to you and you don't want to go with the traditional heavy immunosuppressive treatments.

Good luck with your decision!

Oh my. Where to start.
First, I do tend towards what others have suggested. When I see the 54 count I'm not quite so convinced there was only a trivial contribution from Nplate. A safer route would have been to wait a bit and see if the Nplate response has had a major change. You and your doctor are vastly more familiar with your responses and so this is water under the bridge. LOL, 'mostly'.

Ah, 'libelder'. You know when I stumbled on your comments during my PDSA forum readings months ago and saw this count record and this other count record , it didn't look like ITP. That's some crazy extreme rollercoaster numbers. It might look like having two ITP antibodies working against you, but more extreme then that. I wondered about an outside influence. Maybe lyme disease or something else not yet diagnosed.

Anyway. Now that I see these I wonder why you haven't considered trying Promacta again. Was the dose so high back in 2011 that it caused liver problems? When your Nplate dose went down to 100, I wonder if the Promacta dose would have gone way down as well. So, I have to wonder if a low dose of Promacta would work for you now. Fill me in.

Back to Rituxan and Nplate. This is no known answer to how much Nplate to take. It's entirely up to you. Since your previous history includes 180 to 250 that dose range sounds pretty safe. Getting back to 'mostly'. One option might be to go to 1000 for just the first treatment. The chances of Rituxan fully kicking in the first week are small.

Since you didn't get a Rituxan remission on your previous try (and you don't respond well to steroids), there isn't any reason to expect one now. One would think only a remission plus Nplate scenario would lead to really high counts by your next count check and Nplate injection one week later. Or, am I wrong and have overlooked something?

Where to start? Promacta- it never worked at all, old doc started me on it before a 4-week trip to China when it first came out, I took it for... 12 weeks? Maybe 16, but showed no response. I basically said "I'm going to China anyway, so how about I go WITH Promacta rather than taking nothing at all" and doc agreed but I got into other medical trouble there & my count was bad, and on the flight home it got very bad & ended in hospitalization back in the US on my arrival home.

Regarding treating now when I have an OK count, docs concern is that when I do crash, I crash FAST so he's afraid to wait. I am of several minds, a HUGE part of me says "EF it, no" but I've been battling such serious depression for so long that I don't entirely trust my judgement. Hubby and friends are all brand new to this and terrified, so not as helpful as... as if they were not scared, I guess.

Lyme's, huh? Had it, quack doc refused my requests to test for TWO YEARS before finally testing and saying "Oh. Guess what?". I was treated with 2 rounds of 10 days each of whatever the antibiotic is, but of course now I have the antibodies so they SAY I will always test positive, even though "it's been treated/cured". Grrrrrr.

Sadly, here in Central Virginia healthcare STINKS and options are oh, SO limited. ONE hema office within 2 hours, literally ZERO therapists "in network" with Humana, I drive an hour+ to get to my primary care doc... At least my current hema listens, and if I say "no" or balk I don't THINK he'll flip out too badly- but we have not yet conflicted, so I don't know for sure.

So if it were y'all, would you wait and not do the Rituxin til I crash again, or find out now if it's going to avert crisis? Or possibly even just plain work? Just opinions, I know. Thanks.

Ahh, ok. That clears things up a lot. After re-reading I now see that Promacta was only for a short time.

The Doc makes a strong point. A 'crash' could be very confounding. Makes me wonder if a 1000 Nplate dose should be continued and the Nplate dosing rules should be followed normally while taking Rituxan. My two cents.

Have you cut off the Pred, or are you quick tapering?

Hi libeldr~ I guess from your story I don't really understand why your doc would say to stop the prednisone if you both think thats what brought your counts up to 50? It does make one crazy so maybe that high dose is intolerable.

I certainly see the problem! You've had a brain bleed, so don't want to crash again. You said that steroids never worked for you and it seemed like the solumedrol did nothing when you were in the hospital. hmmm... So it would be my guess that the steroids are not working? of course, not sure about that!

If it were me though, I would stay with what you are doing right now- prednisone + Nplate. Something got your counts to over 50, so its probably one of the two treatments. Or maybe the combination. My policy usually is if something is working, stay with it and see how it plays out. I don't like to jump around with treatments. The TPOs can act very strangely. Also you said you have "an inclination to take the 1000 Nplate". I usually go with my intuition, then if its wrong at least it was my decision.

For me and Nplate the counts rise a bit slowly. My counts go up the second week after the dose increase. So if these were my numbers, I would think the Nplate is just getting going and next blood draw the count will be around 100+. Just my thoughts- hope whatever you decide is successful!

Well I did the first Rituxin drip yesterday, took 5.5 hrs. Didn't love it, it made me feel very flu-ish yesterday and last night but I feel OK today. I opted to just do the Rituxin and not Nplate too, when I go in for my final drip if it hasn't shown a response I'll probably add it then, we'll see. Doc allowed me to stop the pred last Fri after 3 days at 100mg., my count then was 54, but it was back down to 12 yesterday so he had me restart at 60, took that yesterday and today and then tomorrow I drop to 40 and stay at that til at least the next drip. I'm not optimistic any of this is going to work. Doc says next up is "aggressive immune suppressive chemotherapy"- anyone know exactly what THAT is? My initial response is HELL NO, I lived with single digits for nearly 20 years, I'll chance it, but I need to find out exactly what he's suggesting- when I told him I doubted I would agree to that he started telling me all about the only ITP patient he almost lost and all the horrific stuff they did before she went into surprise remission where she has remained... But A) he made it sound like he doesn't know WHY she's in remission or even if it's from one of the treatments, and she went through HELL. I've been through hell already and I am TIRED. I'm tired. Death has no sting, but stroking out DOES, so, tired, and scared. Crap. I've got four weeks til the final drip and by then I need to have a plan. Ugh.