I have become very familiar with this board over the past couple months as my mother has a truly baffling low platelet disorder going on right now. I have read through many posts here and appreciate everyone who has made community what it is. I'd like to tell you a little about my mother's condition. She is 71 years old, healthy and active up until December. She has indicators for lupus, but never had any lupus symptoms. She had guillian-barre syndrom in her late 20s or early 30s, has had a thyroid removed, but otherwise nothing of note. She doesn't drink, smoke, and eats healthy (not perfect, but nothing egregious) . She has never had a sign of platelet disorder.
In July of 2017 she went to the doctor for a routine check and came back with platelets around 545. Mid-August her platelets had climbed to 608. By the end of August they were down to 245. By mid-September they were 45 and then progressively dropped into November when she had 9. They gave her Dexamethasone and Prednisone (which she has been on a decreasing amount of since), along with a transfusion. These treatments did not work. Mid-November they gave her Rituxan twice to no effect. They checked for H. Pylori and it was negative. They tried a third round of Rituxan and it did nothing. End of November, they did a bone marrow biopsy and there was nothing to indicate issue. Beginning of December she was admitted to the ER and admitted. They tried IVIG and it did nothing. She proceeded to stay in the hospital for 7 weeks to mid January while they tried to figure out what was causing the low platelets. They brought on another hemotologist, did every work up on her blood, brought in a rheumatologist as they could not determine if it was a blood disorder, immune issue or what. They think ITP, but they did a 2nd bone marrow biopsy and they used that to diagnose her with amegakarocytic thrombocytopenia, a very rare disorder. They started Nplate in December and I think she is up to 8mcg/kg but have seen nothing.
Most recently they tried apheresis to clean her blood and try to knock out the immune response. They tried some immunosuppressant as well as Danazole.
Through this entire time her platelets would only increase with transfusions (up to 40) and then aggressively drop over 3-4 days to 7-8 where they would level out. They would sometimes drop to 3-4.
In mid-July they sent her home because they had exhausted all options. She was still getting the Nplate, Danazole and immunosuppressant. Over the next week she started to feel really cruddy and as of this last weekend went to the ER due to pneumonia. She is in ICU now and recovering well, hopefully she will be taken off the ventilator soon.
It's been a rough last 2 months for her, she was in amazing spirits at the hospital through her treatments, but nothing is working and as you can see we feel we have tried everything. At home she became hopeless and depressed and thinks her quality of life is going to go down hill quickly. Hoping you all gave give some words of encouragement along with experiences to help guide us in the right direction. So, on to my questions:
- Any one have any ideas beyond what we have already tried?
- I spoke to the hematologist about trying Promacta if the Nplate doesn't work, and he said if one doesn't work, the other won't. Any experiences where Nplate didn't work, but Promacta did?
- I was thinking of getting a functional medicine doctor involved to look at her wellness (eating, exercise, etc) to see if there are other options. Anyone have success with this?
Thank you again for all of your help up to this point, so happy to have this as a resource.
I'm sorry walter to hear this about your Mother. A couple questions come to mind.
You mentioned she is on an immunosuppresent. Do you know which one, or ones, it is?
Also, I looked up 'amegakaryocytic thrombocytopenia' and see that it is a congenital condition. If that is the issue, one would think her blood ANA level would be normal or close to it. Do you know what her ANA test result is? An example would be '1:80' with a qualifier of 'homogeneous'.
That is the first I've ever read of someone's platelet level going up before crashing. I assume the doctors have no comment on that as well?
The immunosuppresent she is on is called Hydroxychloroquine.
I am not sure what the ANA test result is, I don't seem to have access to her biopsy report.
Doctors had no comment on her platelets going up dramatically then down.
Diagnosed in 1998, currently in remission. Diagnosed with Lupus in 2006.
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Hi Walter - I'm happy you found us. I'm not sure how much help we can be though.
My first question is: how are her symptoms? Does she have any bleeding or bruising? Also, did she start any new medications (including supplements) or have a vaccine prior to the start of ITP?
Hydroxychloroquine isn't really an immunosuppressant. It is also known as Plaquenil and is a very common Lupus treatment. I've been taking it for 11 years (for Lupus). I'm not quite sure why they would start her on that one, unless they feel that she may have Lupus and controlling that might help to control ITP. ITP can be a symptom of Lupus. Do you know what tests they ran for that? The ANA is just one of them...it's blood work.
I disagree with the doctor about Promacta not working if N-Plate doesn't. They are similar, but work a bit differently. At this point, really, what's the harm in trying it? We've had people here who have responded to one and not the other.
As far as the depression and quality of life: most people find that it's not ITP itself that causes those things, the treatments do. Both Prednisone and Decadron can cause a lot of nasty side effects, including depression. There is no reason to believe that ITP will cause poor quality of life unless bleeding becomes a problem. Low counts are a bit more serious in elderly patients because the blood vessels are not as strong as they would be in a younger person, but that does not mean gloom and doom.
I've seen this happen before....many treatments are tried and they all fail. Eventually, something kicks in and counts go up. This is not a hopeless situation yet by any means. Tell your mom to hang in there. ITP is strange, it sometimes just comes and goes for no apparent reason. Please keep us posted.
Holy frigging smokes mrsb, what a find. That study describes row 4 in my table almost perfectly. Poor steroid and IVIG responses and Cyclosporine as very effective treatment. Even talks about (T cell) antibodies to c-Mpl (TPO receptor) as being the problem - just as the
4 ITP types study
walter, as far as I know Sandi is correct and the doctor is mistaken. It would be appropriate to try Promacta when Nplate doesn't work. Some folks start producing antibodies to Nplate, thus causing it to be ineffective. In a related note, there are no reports of antibodies occurring against Promacta. But a small percentage of folks don't respond to Promacta apparently because of physiological differences.
Another question for you walter. The response difference between row 2a and 4 in
my ITP treatments table
is very small. When you say IVIG did nothing for your mother, does that mean her count went up for a day, or, for a week, and then fell back down? Which best describes the situation?
I stumbled across this study on 'cyclic' thrombocytopenia and noticed that counts can go really high (600) during a cycle. Suggested treatment was with Danazol. So perhaps your mother is doing well now since the doctor is treating her with that.
I am happy to report that after a very trying couple of weeks, my mom should be discharged from the hospital today. She still has a battle with low platelets, but she is on her way to recovery in regards to the pneumonia. Really appreciate the support everyone.
She got 10mg/kg of Nplate and they are going to try that for 3 weeks. If that doesn't do anything, they may try the Promacta.
She also has an appointment with Dr. Liebman, who focuses on ITP patients. Interested to see what he has to say.
Thank you Hal9000 for sending this study, I will pass it along to the doctors.
Studies like this and the one that mrsb04 linked to are very helpful. I think it is all a matter of trying things out to see what sticks. The doctors pretty much all agree that while there are standard treatments that work, there are tons of one off studies and anecdotal stories that show people going into remission from atypical treatments. In my mom's case, I think it is important to not bombard her with too many treatments at once, but rather line them up and try them all out. The treatments can sometime cause more hardship than the low platelets themselves.
I am also trying to convince my mom to look at here diet and environmental factors as well. Has anyone tried a functional medicine doctor? I personally subscribe to the belief that a lot of health issues stem from out lifestyles. My mom does not live an unhealthy life, but who knows is some random food, cleanser, or hairspray is contributing to her condition.
You might be interested in reading about
and her husband's ITP experience. He had no steroid response, no IVIG response, did NOT respond to Nplate, but did respond to Promacta. Also along the way, splenectomy did not work. Eventually he went to Mayo Clinic which recommended adding steroids to Promacta, or, trying Cyclosporine.
IMHO, Promacta and Pred works well for row 2 and Cyclosporine works well for row 4 in my ITP treatments table. Promacta/Nplate works for row 4 too but the highest doses are required.
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