Four Years with No Answers

Hi Everyone,

I wanted to reach out to this support group to see if I can get some help/advise for what my Doctor seem to be deeming ITP.
I want to give a very quick background that will prolly not be as quick as I wish but will carry the pertinent information needed (I think).

In 2012 (I was 20) My wife (girlfriend at the time) and I went on a date to SF and came home to have some desert with friends, I was fine until I got home, and all of a sudden I was super tired, and freezing cold, I took a shower to try and get warm it didn't help so I curled up in the blankets. I woke up an hour later sweating and still freezing, took my temp and was running a high fever, long part short, ended up in the ER. They did the work up and decided I had pneumonia and that my labs were abnormal including a low platelet count and some other things, anyway they sent me home told me to return the next day and they would do another scan and more labs which then they found no pneumonia, but my labs were still bad. So they referred me to a Hemonc Dr. I saw that Dr. once and he said it was ITP and that i just needed to start Prednisone and it would fix it. Well for 6months we tried 20MG up to 60MG down to 40MG up to 80MG being the highest and with all of that there was no cure, so for we went and saw him locally again and asked if there was anything else we could do or if he would refer us out and he said no he didn't believe there was need for more testing or anything my platelets weren't low enough for him to be concerned, but if I started noticing petechiae that I should come back.

Well for a year I was fine my wife and I got married but then it just went down hill fast. I started noticing the petechiae and getting really tired, I was having shots of sharp pain around my lungs that would take my breath away and then Id be fine. So one night these symptoms got so bad I went back to the ER and they did a whole workup on me again. This time it was really bad lab results, WBC was low RBC was off, Neutrophils were off, I mean everything was not normal. So I talked to the ER Doc and told him the above info and he decided to do the referral to UCSF for me to get seen. This would take about 4 Months for me to get in, and so during that time I went on vacation with my family. Well while on vacation the petechiae got really bad and I got really sick again, I was always short of breath and at this time so sick that I couldn't eat and if I did it just came right back out. Well the week we got home from vacation was the week of my UCSF appointment and so thats where I went. But I was not expecting the results.

Not only was my platelet count to 2,000 but I was Neutropenic with absolutely no Immune system. So they told me I had to be admitted immediately and I would then spend 14 days in the hospital. This was in 2014 6 months after my wife and I got married. They did all kinds of tests, to basically find nothing so they did my first round of 100 grams of IVIG, boy were the side effects awful, I wasn't allergic to it but I got sever migraines from it so this did this 2 days in a row and then also a platelet transfusion to try and increase my count so I could have a lymphoid removed and tested clear other than the puss that was inside of it. So they ruled out all cancers and with the IVIG working at the time they allowed me to go home but two weeks later I had a relapse and had to return for another week with same symptoms and treatments, they then sent me home increasing the treatments of initially and then putting me on a weekly dose of 100 Grams this seemed to maintain my platelets at around 80,000 but after six months it was no longer working, Well with everything going on they decided to send me the Immunologist to get a diagnosis of CVID, however they found that not to be true either and put me back a square one.

We then decided to try 120MG Prednisone every day for almost 5 months and during that time it worked but it too came to an end, So we mixed the two treatments and came to the conclusion that worked as well for a short time, however that also came to an end. Then came the NPlate which for the most part has worked, however I had a doc that only would give me 3 times the original dose so because of that I ended back in the hospital but this time it was because of hemolytic anemia and 6,000 platelets. Well they game me 100gram dose of the IVIG again and steroids and a platelet transfusion but they finally realized the platelet transfusion was pointless as my body just devoured them anyway. So out of the hospital I came this was in 2015 and we continued with NPlate dosing changed until Feb 2016 where we started Rituxin, and I had 4 transfusions, it has now been 3 months and I found out yesterday my Dr. is saying was unsuccessful as I am still needing the NPlate shots every other week with my platelets falling to around 20,000.

Now I want to take a moment and saying that during all of this, I have had very strange labs in my opinion but my doctor seem to thing it is all part of the ITP. I have low counts HGB, HCT, MCHC. MCH. My RBC changes now and again along with the WBC. Now along with this all of my cell morphology is abnormal too. I also developed a CVT (Blood clot) with 27,000 platelets.

My Doctor is now requesting that I do a splenectomy, but all i keep thinking is that there has to be something else causing all of this. I have done some research and feel like I just don't quite match up completely to ITP.

It's hard to say what is going on since it could be numerous things. You gave a lot of information but some of it wasn't specific enough.

ITP is only low platelets. It does not include low white cells or low red cells. When those are low too, it is sometimes diagnosed as Evans Syndrome. Reds and white can be off on a CBC but not be low enough or high enough to be considered a problem. My CBC's are never normal, but it's generally not a concern.

Many people treat with N-Plate and since it is a maintenance drug, you have to keep using it to get a safe platelet count. The dose should be adjusted to maintain counts around 50k since blood clots can be a risk. Hopefully, your doctor knows that and is dosing you correctly.

I'm not quite sure what you are asking. Splenectomy is not a cure and since you've had a clot, I'd consider that very carefully before doing it. Splenectomies can raise the risk of clotting, especially when using N-Plate. If the splenectomy does not work, you'd need to keep using an ITP treatment.

How are symptoms when your counts are down? It looks like you have had some over-treatment going on with all of those high doses of Prednisone.

Have you had a bone marrow biopsy? A few months ago I had very low counts for platelets hemoglobin and neutrophils. I had a biopsy and was found to have under productive marrow. However going on 75 mg promacta daily has stimulated the bone marrow, increased my hemoglobin and neutrophils to near normal and kept my platelets at a low but above bleeding level. I previously had a diagnosis of just ITP for 3 years and benefited more platelet wise from Promacta but I am happy with the levels I have at present.
Rituxan has not worked for my platelets either.

According to the National Organization of Rare Disorders:

Evans syndrome is a rare disorder in which the body’s immune system produces antibodies that mistakenly destroy red blood cells, platelets and sometimes certain white blood cell known as neutrophils.

My comments assume you have Evans Syndrome or some similar autoimmune cytopenia.

A good general article:

emedicine.medscape.com/article/955266-overview
Medscape: Evans Syndrome

I would steer clear of splenectomy:

emedicine.medscape.com/article/955266-treatment#d9
Medscape: Evans Syndrome Treatment & Management
Splenectomy does not have a clearly established role in the treatment of Evans syndrome, but it may be considered in refractory cases. Splenectomy may improve the CBC and reduce steroid dependence; however, relapses are common and, in most cases, occur within 1-2 months after the procedure.
According to a national survey, splenectomy provided a reported duration of response that ranged from 1 week to 5 years; however, the median response duration was just 1 month.[15] The risk of postsplenectomy sepsis appears to be increased in children with Evans syndrome, especially those with pancytopenia.

Because of its rarity, information on treatment for Evans Syndrome is limited, but there are many treatments for ITP left to be tried. Some may work when others don't, including dapsone and danazol.

One that you won't find on any list is Sirolimus. A very recent study showed Sirolimus (Rapamune or rapamycin) may be effective in cases of multiple cytopenias, e.g., anemia + neutropenia + thrombocytopenia. The following study is small, but it does show a strong effect:

www.bloodjournal.org/content/127/1/17
Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial

@ Sandi, I'm sorry that all the info was not as detailed, Originally I was treated by my now retired local hematologist who only would use up to 3 times the NPlate, high doses of prednisone and just in general poor treatments now I have had all kinds of doses of the NPlate with the new Dr. that took over his practice. My biggest question was if I truly had ITP, because like I have read it seems the only thing that should be happening is the low platelet counts. I personally really do not want to do the splenectomy and that is good to know about the clotting factors. My symptoms are relatively minor, petechiae and migraines and very tired. I rarely have any actually bleeding but it has happened. I personally have noticed the symptoms get worse with stress.

@Rob, thank you for that information, I will do some research

@Margaret, I have had a bone marrow biopsy at UCSF and they said it was fine.

Well, how are your counts now with N-Plate?

Do I understand that your platelets are at 20,000 with treatment every other week? Dosing is normally done every week!

Also, is there any reason not to continue with Nplate, dosing EVERY week, and increasing the dose to give safer counts closer to 50,000. Are you having side effects? Is your insurance complaining? Nplate is a maintenance drug, not intended to cause remission (although sometimes it does). If weekly injections are a problem, is Promacta a possibility? It is a similar drug in pill form. Sometimes it is treated differently by insurance.

What was abnormal about your cell morphology? Was it elevated MPV (mean platelet volume)? Large platelets are very common with ITP. I have large corpuscles - elevated MCV. For me it is hereditary. Abnormal morphology does not always spell trouble.

With my dose of NPlate at 5 times the start dose and finishing the Rituxin treatment almost 3 months ago they drastically vary, one week after the shot they go up to 150 or higher, and then the next week they are stable and the week after they crash to 23 at least those are the number this time.

This was my most recent lab results:

WBC
6.9 K/uL

RBC
5.14 M/mm3 Date:

HGB
13.4 gm/dL (Low)

HCT
40.6 % (Low)

MCV
78.9 fL

MCH
26.1 pg (Low)

MCHC
33.1 gm/dL

RDW
14.5 %

PLT
23 K/uL (Critical)

PLT Estimate
Decreased (Abnormal)

Auto Neutrophil Percent
59.0 %

Auto Lymphocyte Percent
28.7 %

Auto Monocyte Percent
7.6 %

Auto Eosinophil Percent
3.7 %

Auto Basophil Percent
1.0 %

Auto Neutrophil Absolute
4.1 K/uL

Auto Lymphocyte Absolute
2.0 K/uL

Auto Monocyte Absolute
0.5 K/uL

Auto Eosinophil Absolute
0.3 K/uL

Auto Basophil Absolute
0.1 K/uL Date:

RBC Morphology
Abnormal (Abnormal)

Poikilocytosis
1+ (Abnormal)

Anisocytosis
1+ (Abnormal)

Microcytosis
1+ (Abnormal)

Hypochromia
1+ (Abnormal)

Ovalocytes
1+ (Abnormal)

I am not on NPlate, but others have reported that it can take several months for the platelets to be more stable even with the same dose of NPLate weekly. Hopefully that happens for you.

And if not, Promacta is an option and can produce more stable results.

Hey Everyone, I just wanted to show you all my lab results after treatment with N-PLate.

WBC
6.4 K/uL

RBC
4.95 M/mm3

HGB
12.9 gm/dL (Low)

HCT
39.5 % (Low)

MCV
79.8 fL

MCH
26.0 pg (Low)

MCHC
32.6 gm/dL

RDW
14.5 %

PLT
199 K/uL

Auto Neutrophil Percent
59.0 %

Auto Lymphocyte Percent
28.0 %

Auto Monocyte Percent
8.0 %

Auto Eosinophil Percent
3.9 %

Auto Basophil Percent
1.1 %

Auto Neutrophil Absolute
3.8 K/uL

Auto Lymphocyte Absolute
1.8 K/uL

Auto Monocyte Absolute
0.5 K/uL

Auto Eosinophil Absolute
0.3 K/uL

Auto Basophil Absolute
0.1 K/uL

That is a nice increase in your platelets... a little too nice, because you don't want to be too much above 50k, lest you get a blood clot.

Are you having Nplate weekly now? You should be getting it every week to stabilize your count. You don't want it to be too high or too low, so you should get it every week, unless your count gets too high, >400k, then you skip a week.

Please read the following dosing instruction carefully, and be sure that your doctor is following them closely:

The initial dose for Nplate is 1 mcg/kg based on actual body weight.

Dose Adjustments

Use the actual body weight at initiation of therapy, then adjust the weekly dose of Nplate by increments of 1 mcg/kg until the patient achieves a platelet count ≥ 50 x 109/L as necessary to reduce the risk for bleeding; do not exceed a maximum weekly dose of 10 mcg/kg. In clinical studies, most patients who responded to Nplate achieved and maintained platelet counts ≥ 50 x 109/L with a median dose of 2 mcg/kg.

During Nplate therapy, assess CBCs, including platelet counts, weekly until a stable platelet count ( ≥ 50 x 109/L for at least 4 weeks without dose adjustment) has been achieved. Obtain CBCs, including platelet counts, monthly thereafter.

Adjust the dose as follows:

If the platelet count is < 50 x 109/L, increase the dose by 1 mcg/kg.
If platelet count is > 200 x 109/L for 2 consecutive weeks, reduce the dose by 1 mcg/kg.
If platelet count is > 400 x 109/L, do not dose. Continue to assess the platelet count weekly. After the platelet count has fallen to < 200 x 109/L, resume Nplate at a dose reduced by 1 mcg/kg.

So I was told not to get a shot this week, because my platelet count was 199, but based on the direction you posted that was the wrong information. This is how my treatment has been going for the last year. So I am very concerned. I am being referred to Dr. Gernsheimer based in Washington, to try and get a second opinion before my spleen is removed. Im hoping to come back with some definite answers.

Remember: it is your decision, not the doctor's decision, whether your spleen is removed.

Your last comment shows how knowledge empowers you to take charge of your healthcare. Good for you!

Your body responds well to Nplate. I do not see a reason here for splenectomy.

Regarding Nplate, the dosing instructions say leave the dose the same this week. It would be reasonable to reduce the dose by 1 mcg, but simply skipping a dose when your count is <400k is against everything I have read.

It looks like your second opinion doctor is an exceptionally fine choice:
depts.washington.edu/hemeweb/research/gernsheimer.htm
Terry Gernsheimer, M.D.
Professor of Medicine, Division of Hematology
University of Washington School of Medicine
CURRENT CLINICAL INTERESTS: Bleeding and Thrombotic disorders, platelet disorders
CURRENT RESEARCH INTERESTS: Platelet transfusion and platelet immunology, autoimmune platelet disorders
She co-authored the 2010 International consensus report on the investigation and management of primary immune thrombocytopenia, led by Dr. Drew Provan who is arguably the best in the world.

Edit: ... and Dr. Provan does not recommend splenectomy at all!

I know you will find Terry Gernsheimer, M.D. to be a great choice for a Hematologist, She has been my Hemo for the last 3 years and I find her extremely knowledgeable and not willing to "over treat". She is great at listening and working with you to form a game plan.

She has also been a Medical Advisor here for quite some time.

Hi All, I just wanted to update everyone and talk some things through.

Its been 2 yrs since I posted, and I am still in the same boat but with more side effects of all the drugs, I think. My insurance denied me going to see Terry Gernsheimer, M.D. and we couldn't afford to do it on our own, but I was able to see my old Hematologist, who is now at UC Davis. This was after I had another set of Rituximab treatments with no improvements. So when I got to see my doctor. We went through the treatment plans I was on, and the plan of having splenectomy. HE WAS FURIOUS. He stated that due the way the my local Hem/Onc doctor was dosing the N-Plate, that it could be causing me more issues due to the inconsistencies, missing weeks specifically. He even threatened to call the drug reps to have them go to the office and teach them how to dose and use the drug. It was crazy. He also stated that the splenectomy was not going to do me any good and that they are not sure its worth trying to do that. So he decided to start Promacta, this was almost 8 months ago or longer, and I have yet to start a dose. I am so scared of just taking another drug when nothing seems to work. I do understand dosing wasn't right, but with the IVIG, Rituximab, N-Plate and Prednisone. I feel like mentally these med have started taking a toll on me. I forget things, like clock-in at work, words in the middle of sentences or I just go completely blank on my train of thoughts, I'm also always exhausted. I don't sleep well at night, and then I drained all day long and now I have floaters in my eyes. While not of these may be related, I'm only 26. It was so bad my work asked for me to get a note from my primary care asking if I am able to do my job. Both my provider and I both feel like I am, but that we may need to make some accommodations. My fear though is that as of yesterday, My work isn't sure they will be able to accommodate. I work for a hospital and run our TeleMed (Virtual Care) Program. I have been doing it for almost 2 years. I feel like my health is going to cause me to not be able to provide for my family. Which brings me to my biggest fear, permanent disability and what that means for me. Is ITP able to be diagnosed as a permanent disability. I honestly don't know what do, but I can't keep going in and out of work, I use all my sick time and pto for Dr. Appointments and am getting ready to have to go out for a week for a procedure. I don't have time. So back on disability, but how long will this last? Do I need to look at this as a long term thing? I mean we are going on 6 years since this started with still no clarity.

I'm sort of confused. What med are you using right now?

Have you had a general check-up to see if you have anything else going on? There may be other reasons for your symptoms....don't just assume it's ITP/treatments.

These are disability guidelines for ITP:

These are the SSD guidelines for ITP. Once you file, there is a six month waiting period until you hear whether or not you are approved. If you are approved, there is a year waiting period until you can receive Medicare. It is hard to get approved for ITP.

The Social Security Administration (SSA) discusses disorders of the blood and circulatory system under Section 7.00 Hematological Disorders, and defines “chronic” as lasting at least three months. You must have been examined at least twice in that three-month period. Chronic Thrombocytopenia, regardless of the cause, is considered under Section 7.06. To meet the requirements of this listing, you must have a platelet count that is repeatedly below 40,000 AND have at least one of the following symptoms: at least one spontaneous hemorrhage which required transfusion within five months prior to filing for disability benefits OR you must have had intracranial bleeding within twelve months prior to filing for disability benefits.

www.disability-benefits-help.org/disabling-conditions/chronic-thrombocytopenia-and-social-security-disability

I've been prescribed to take Promacta, but haven't taken in yet. It may be against the standard way. But I felt like we just kept pouring medications into my body and never really let there be time from one treatment to the next so I made a decision to wait to take Promacta. I will however be starting taking it as my numbers have been all over the place over the last 6 months. I still get labs weekly. I have ranged basically from 30's to 90's. I have had primary care follow-up at which point I was asked to go see: Neurology for Migraines; Dermatology; Sleep Medicine for possible sleep apnea; They even talked about Rheum but are holding off for now.I can't be in the sun, I get severely exhausted with heat and exertion and my cheek and under my eyes get red as if I were sunburned. I have anemia as well, but I'm not iron deficient and I have to take 50,000 iu's weekly of Vitamin D, because my body doesn't created enough. It seems like my health goes in cycles. The sumers get really bad and the winter/spring I mend. But yes because of all this, I am meeting with my primary care monthly.

Rheumatology might be a good place to start based on your symptoms.

If your counts are ranging 30's to 90's, that is above treatment level and most would not treat with those counts.

I agree. If my count was above 30 I wouldn't take any meds.

I will ask for a Rheum Referral as well. I didn't know there was a "treatment rage". I mean I kinda of just assumed that over 50 was good but not as low as 30. I have been told that my platelets must be at least 50 for surgery. Thank you for the information!

It depends on symptoms. Do you have bruising or bleeding with counts in that range?

If you would end up needing a surgery, you'd deal with the counts at that point. Some surgeries can be done with low counts and some need counts over 100k.

Presence of new floaters necessitates a trip to an ophthalmologist. You could have retinal bleeding. New floaters meant a retinal tear for me, requiring a laser repair surgery. Gook luck with all of this.
Norma