Highly Refractory ITP

Hi Everybody!
My doctor informed me today that my ITP is classified as "Highly Refractory", meaning I do not respond at all to any treatment. He said I was an extremely rare case, yet I still can't win the lottery. I was diagnosed 5 months ago when an unrelated trip to the emergency room found my platelet count to be 6 and they admitted me for a week. Since then I have been on six different treatment plans and my platelet count has never been as high as the 6 I was admitted with, and has been as low as 2. I am starting on Danazol this week so we will see what happens with that.
I have made peace with the fact that I might just have single-digit counts for the duration, so any positive moves in my count I would consider a bonus. I am for the most part symptom free except a bit of fatigue, and I feel like I can still do everything I was doing 6 months ago. It's hard for me to wrap my brain around the fact that this thing that I had never heard of before I got it and really don't feel any symptoms from has put me out of the game, but the disability checks start in March, so I guess it's real.

Now to the question I had set out to ask before this somehow turned into a soul-searching autobiography. Anybody else out there have or know of a similar situation or heard of any treatments a Refractory patient has had success with? My doctor is reaching out to several colleagues since he has never had a case like mine so I figured I would reach out here.
Thanks for reading,
Brian

What are the different treatments plans you have been on so far. 6 in 5 months seems a lot ?

I was given IVIG and steroid shots in the hospital, followed 4 weeks of prednisone, 4 weeks of Rituxan 4 weeks of 50mg Promacta then 4 weeks at 75mg then 4 weeks of Nplate , oh, and Flintstones chewables

Hi
I didn't respond to anything except
Ivig- and that only lasted a few days. I was on nplate for 6 weeks at the max dose (10mcg/kg) before I saw any improvement. I believe you are supposed to increase your dose and give the highest dose at least 2-4 weeks before giving up. It suddenly worked for me and I'm even down to 9 mcg/kg now. Hope this is helpful- good luck!

Welcome Brian. Did your doc mention to you that Flintstones chewables, err ahh I mean, Rituxan can take 12 weeks to kick in?

Also, has he mentioned Danazol to you yet?

Sounds like a combination treatment would be next (after more NPlate?). Goo-Foo Research Master 'Rob16' should have some recommendations for you...

Hi Brian,

Combination therapy can work when single treatments to not. The most effective ones use either Nplate or Promacta to boost production combined with an immunosuppressant like mycophenolate mofetil (Cellcept) or cyclosporine to suppress platelet destruction.

I posted some scientific literature on combination therapies here:
pdsa.org/discussion-group/6-general-itp-discussion/27398-8-oct-2016-my-platelet-count-is-168.html?start=210#56024
That post included a link to a patient called drbean7218. That link no longer works, so please look here for it:
pdsa.org/discussion-group/6-general-itp-discussion/27398-8-oct-2016-my-platelet-count-is-168.html?start=60#34306

Bholm wrote: Hi Everybody!
My doctor informed me today that my ITP is classified as "Highly Refractory".

Now to the question I had set out to ask before this somehow turned into a soul-searching autobiography. Anybody else out there have or know of a similar situation or heard of any treatments a Refractory patient has had success with? My doctor is reaching out to several colleagues since he has never had a case like mine so I figured I would reach out here.
Thanks for reading,
Brian

Interestingly I am a "Highly Refractory" (63yr old male ITP since 2011) and have just started (at my request) 200mg three times a day of danazol. I currently have 50mg IVIG weekly to keep me from zero! We tried 100mg fortnightly but I was ending up with counts of 1,2 or 5. Now having gone back to weekly I can get into double figures.

Nplate worked for me for two and a half years (not without side effects) then suddenly tapered off. Coming off Nplate altogether I believe has placed me in a worse situation than I was in before taking it.

Danazol can take months to work (or not work) and I will be continuing weekly IVIG for the time being.

Please keep us informed of any progress as I will. They always do a FBC prior to my infusion so it's going to be interesting!

11th January 2016 count 15 started Danazol.

Thanks for the information rysmyth. My doc mentioned trying Danazol to me yesterday. I did 4 weeks of Rituxan in December but have not seen any benefit yet. Last 4 weeks my counts have been 12, 14, 15, 17 (yesterday). I am glad that at least they are going up, although so slightly it is in the error band of counting, but they could have been going down the same trend. I see my doc again on February 1 to decide if we go the Danazol route.

I will be watching for your progress on this, thanks for sharing.

Bholm:
Sometimes N-Plate, Promacta and Rituxan take more than a few weeks to work. I see far too many doctors give up on a treatment too soon.

Hang in there....most people respond to something eventually. How are your symptoms?

Symptoms are not terrible, bruises and some mouth blisters. I do bleed more than I used to when cut but I am still clotting without heroic efforts.

You can try your ability to respond to splenectomy by plugging in the numbers. Main parameter is your age.

jamanetwork.com/journals/jamasurgery/fullarticle/396191

Just a study doing a multivariate analysis.

Splenectomy prediction score = 0.039 + {[Age (Years) × 0.047] – [Preoperative Platelet Count × 0.018]}.

All patients receive a score between –4 and +4. A negative score predicts a successful outcome after splenectomy, and a positive score indicates an increased risk of a failed response. Patients are subsequently classified into 3 categories. Patients with a negative score (<0) (n = 10) had a 100% success rate following LS. Patients with a moderate score (0-2) (n = 26) had a 65% rate of success, and patients with a high score (2-4) (n = 31) achieved a successful outcome in only 40% of cases. For example, a 50-year-old patient with a preoperative platelet count of 15 × 103/µL would have a score of 2.11, correlating to a 40% success rate.

Trouble is you can have your spleen out and get great counts for six months only to find your liver takes over destruction duties. Growing accessory spleens is another possibility.

None of the above are predictable.

Agree, RJ. A person could also develop other antibodies. Long-term follow-up is needed in these studies. Some do, but some only rely on post-splenectomy counts. Those numbers can also be skewed by treatments used prior to splenectomy which raise counts short-term. "Success" can be a misleading term.

Hello RJ,

If you are in your 40s or younger and do not achieve remission or low stable counts with no medication after 1 year of treatment then splenectomy is a valid alternative.
Surgery is risky but having weekly ivigs for the next 20-30 years is not good alternative either.

Define low stable counts please

I would take 20-30k count with no medication as a stable low count.
I cannot achieve it, sometimes I wonder what would happen if I take the risk and live with low platlets no matter what.

Jay, some people do because they have no choice. Nothing works or they decide that the meds are doing more harm than good. If it's at all possible to find a balance, like using a medication at the lowest possible dose to maintain a safe count, that may be a good option for as long as possible. Most people eventually choose to do what they can tolerate, whether it's constant meds or low counts. I've seen many, many remissions that I never thought would happen, so there is always that hope.

Depending on your life style and job - one can live with counts between 20k - 30k without treatment (some even lower) - you should have a game plan in place with your Hemo should you need to treat - but it is doable if you have no symptoms - I have done it for the last couple years.

I found the long term side effects of treatment were much worse than not treating.