TOPIC:

Our 10 year old daughter was diagnosed with ITP a little over 2 months ago. IVIG treatment and Steroids briefly increase her platelet counts but once she is off the treatment the counts drop back down to under 5. Her Hematologists would like us to consider putting her on Promacta. Does any one have any experience with this or thoughts? Thanks!

I'm not a child, but I'd say give it a go.
There are some slight dietary restrictions but they can be virtually avoided by taking tablet last thing at night.
The aim is a count of 50 or above on the lowest dose possible.
From my own experience it may take a while to find a suitable dose but worth sticking at it.

I like your Hematologist and I've never even met them.

Thank You! Your links have been extremely helpful, especially the one 'ITP Types According to HAL'. One of the mysteries for us has been there was no identifiable sickness preceding her diagnosis in January. Reading through your table got me thinking more about that time. A couple weeks prior to her diagnosis a number of children in her school were out with the flu and a week after her diagnosis I came down with the flu (we all had our flu shots). At the very least it gives us another data point to go over with her Hematologist on Tuesday when we discuss Promacta and our options going forward. Thanks again for compiling and sharing this information -- it is so much appreciated and truly invaluable!

EDad, glad it helped. It's a bit of a coincidence that you arrived exactly when chronic versus acute ITP in children is being discussed.
The following may be of interest. Was trying to see if one particular strain of Flu is more problematic with ITP than another.
pdsa.org/discussion-group/7-treatment-general/30169-no-response-to-ivig-ack.html#65712

Flu viruses have what is called 'shifting' and 'drifting' mutations, as described here:
www.cdc.gov/flu/about/viruses/change.htm

I think large H1N1 shifts can cause pandemics. Similarly, I wonder if H1N1 is more capable of triggering ITP. Not enough data to know. Last I looked on the CDC website, H1N1 is the main strain going around this Flu season.

Just a few weeks ago, user 'th8899' son took 6 weeks on 75mg of Promacta before, shall I say, the ITP fever broke:
pdsa.org/discussion-group/12-newly-diagnosed/30194-11-years-old-son-just-diagnosed-with-itp.html?start=60#65692

Did your doctor try IVIG, and that didn't work, to begin talking about Promacta?

Thanks for the links Hal! Very interesting stuff.
A week after our initial diagnosis a blood draw showed our daughter's platelets to be very low so they tried IVIG. Below has been our experience...it tracks to your table...

Jan 22nd -- Our daughter was diagnosed with ITP...she started showing signs of bruising a few days before that progressively got worse. Her platelet count was 6 with no bleeding. They advised weekly blood draws and to call if there was excessive bleeding.
Jan 30th -- We went in for our blood draw and her platelet count was 3. Since she had had some minor nose bleeds that week her pediatrician sent us to Seattle Children's ER. At the ER they advised IVIG and we did it. She tolerated it quite well and we were discharged the next day.
Feb 5th -- We went in for a blood draw and check-up. Her platelet count was 45.
Feb 13 -- We went in for a blood draw and her platelets were >5 and she had had a small nose bleed a few days prior so our team prescribed a 5 day steroid course...80 mg per day
Feb 18 -- We went in for a blood draw and her platelet count was 75
Feb 25 -- We went for a blood draw and her platelet count was >5
Feb 27 -- Had another minor nose bleed so team prescribed a second 5 day steroid course...40 mg per day
March 4 -- We went in for blood draw and office visit and her platelet count was 52. At this visit our doctor brought up Promacta since the steroids and IVIG had not been very successful. She said that it can take awhile for the insurance to approve so we started the process.
March 14th -- We went in for a blood draw and her platelet count was 7.
March 21 -- We went in for a blood draw and her platelet count was 6
March 28th -- We went in for a blood draw and her platelet count was >5 again.
We have an appointment tomorrow. We had hoped her platelets would increase from 7 and wanted to give her body a little more time before we started the Promacta. Aside from the fact that her activity is so restricted the wait and watch approach has been OK for us...she looks and feels better than she has in weeks! The problem is that now that her platelets are back under 5 the wait and see approach doesn't work as well it seems. I'm going to ask her hematologist about H1N1 being a trigger for ITP...very interesting! Thanks again for the pointers and links!

EDad, thanks for the info. Parents don't often list what has happened and so data for children, here on the PDSA forum, is sparse.

It looks like your daughter's response is more like 'maria3132' son than it does 'th8899' son - who I referenced earlier. In Maria son's case he gradually improved over several weeks. In th8899's son's case it was more like a light switch. Going from low counts to high counts quickly, with nothing in between. It is this on/off thing that makes me suspect H1N1. It's just a theory on my part. I could easily be wrong. Another possibility is that any Flu virus 'shift' acts that way for ITP, and not just a H1N1 shift. Another possibility is that different people just react differently.

Here is Maria's graph of her son's response over time. Thank you Maria for the graph, and now the teaching tool.
www.pdsa.org/discussion-group/12-newly-diagnosed/30129-petechiae-but-no-bruising-in-3-year-old.html?start=60#64773

As you can see with a slow improvement, it is difficult to classify what the response is since it is always/often improving a bit. EDad what you've described is a partial steroid response and a partial IVIG response. If a child got ITP from the Flu then these partial responses would be consistent with an improving condition. For 'th8899' son, IVIG was a flat line. Steroids was a flat line. No real count increase.

So for your doctor, it looks like he is recommending Promacta because 1) there are possible bad consequences and overt symptoms with a sub 10 count and 2) neither IVIG or steroids provide a strong improvement in counts. The intention is that Promacta can lift counts and prevent symptoms, thus allowing one to return to a normal life. Gotta love that Promacta.

At this point it would be reasonable to expect 50mg of Promacta to get counts up in two or a few weeks. Then a gradual count improvement over time.

Fingers crossed.

I've found this article which may be if interest regarding H1N1 www.sciencedirect.com/science/article/pii/S1726490111002152#bib1
I hope Promacta proves effective for your daughter EDad

EDad, welcome to the group and you will find a lot of useful information here. I saw Hal already mentioned my son's case here. Just want to add couple points. First, I still believe that it is Lyme diseases triggered my son's ITP. And secondly, I still believe Promacta is not working for my son at all. His recovery is because of his immune system finally calm down from Lyme diseases. But our hemo is trying to be more conservative this time. He lowered Promacta dose to 50 mg and we will go back to his office by the end of the month to see if my son's platelet still hold. He has been on Promacta since last Nov. So far I did not notice any side effects. Wish you all the best.

We start 50 mg Promacta tomorrow. We will try to report on progress. At this point, getting our 10 yo's platelet count up to a level where more activities are allowed seems like a worthwhile goal, and Promacta is supposed to achieve that. We were actually warned about the danger of Promacta leading to too many platelets (counts over 350!), which right now seems like a problem we would welcome. Thank you to everyone who replied with their experiences and data points.

Just wanted to post an update! On 4/11 after a week on Promacta our daughter has a count of 9,000 platelets. We are hoping that things will keep going
in the upward direction. So far she is tolerating the medicine well -- no side effects. Fingers crossed.

Right. Promacta is pretty benign for most. I guess 5 is her baseline? It can take a few weeks. Steady as she goes EDad.

Thanks Hal! Yes, five is her baseline -- we are waiting, watching and hoping for the best!

So far so good...our daughter is able to do so much more and we are getting back into our routines with less disruption and worry. Her counts are going up slowly but we will take it! I've also included some dosing information I got from our doctor that I found useful.

Here are her counts:
Week 2 (4/18) - 16,000 platelets -- the count was not moving as quickly as our team wanted so they increased her dose of Promacta to 75mg daily
Week 3 (4/25) - 25,000 platelets
======================================================================================
Here is the Promacta dosing algorithm from Lexi Drugs...

“Chronic immune (idiopathic) thrombocytopenia (ITP): Note: Use the lowest dose to achieve and maintain platelet count ≥50,000/mm3 as needed to reduce the risk of bleeding. Discontinue if platelet count does not respond to a level that avoids clinically important bleeding after 4 weeks at the maximum daily dose of 75 mg.

Initial:

Children 1 to <6 years: Initial: Oral: 25 mg once daily (no dosage adjustment required for patients of East Asian ancestry)

Children ≥6 years and Adolescents: Initial: Oral: 50 mg once daily; for patients of East Asian ethnicity (eg, Chinese, Japanese, Korean, Taiwanese), reduce initial dose to 25 mg once daily

Dosage adjustment based on platelet response: Adjust dose to achieve/maintain platelet count ≥50,000/mm3 as necessary to reduce bleeding risk; maximum daily dose: 75 mg/day. Monitor platelet count weekly until stabilizes and then monthly; additional monitoring may be necessary based on response or any changes in product formulation (oral suspension/tablets).

If platelet count <50,000/mm3 (≥2 weeks after treatment initiation or a dose increase): Increase daily dose by 25 mg (if taking 12.5 mg once daily, increase dose to 25 mg once daily prior to increasing the dose amount by 25 mg/day; maximum daily dose: 75 mg/day once daily

If platelet count ≥200,000/mm3 and ≤400,000/mm3 (at any time): Reduce daily dose by 25 mg (if previous dose 25 mg once daily, decrease dose to 12.5 mg once daily); reassess platelet count in 2 weeks

If platelet count >400,000/mm3: Withhold doses; assess platelet count twice weekly; when platelet count <150,000/mm3, resume with the daily dose reduced by 25 mg (if previous dose 25 mg once daily, resume with 12.5 mg once daily)

If platelet count >400,000/mm3 after 2 weeks at the lowest dose: Discontinue treatment.”

Interesting to note that European guidelines differ slightly in cut off points for platelet count, see red below

If platelet count ≥200,000 150,000/mm3 and ≤400,000 250,000/mm3 (at any time): Reduce daily dose by 25 mg (if previous dose 25 mg once daily, decrease dose to 12.5 mg once daily); reassess platelet count in 2 weeks

If platelet count >400,000 250,000/mm3: Withhold doses; assess platelet count twice weekly; when platelet count <150,000 100,000/mm3, resume with the daily dose reduced by 25 mg (if previous dose 25 mg once daily, resume with 12.5 mg once daily)

Oh my. All these people on 75mg dose. I wonder if the doctor is similarly finding a lot of newly diagnosed folks on 75mg this Flu season as well.

EDad wrote:

...
Here is the Promacta dosing algorithm from Lexi Drugs
... reassess platelet count in 2 weeks ...

FYI, this is because it takes about 2 weeks for a dose change to take full effect. For example, going from 16 to 25 is an increase of 9. The expected count next week is (25+9=) 34. Proportionality is the guide. The time constant is 2 weeks.
Of course, if the immune system changes, all bets are off.

EDad
I spent over a year trying to taper steroids whilst simultaneously trying to establish an appropriate Promacta dose. At 3mg of Prednisolone a day I decided to put the taper on hold and try to find my optimum Promacta dose.
Courtesy of a virus my platelet count was 24 at that time.
I increased my Promacta from 25mg/50mg/50mg to 50mg/day.
10 days later my count was 41 and stayed at either 41 or 42 for the next 4 months.
It rose to the 90s over the next couple of months.

Thanks for the replies Hal and Mrsbo4! Do either of you know if the slow ramp up has any link to how successful Promacta might be for her? I know the nurses and the PA I speak with on the phone are disappointed with the counts so far. That said, it is improving her quality of life so we think it is helping.

EDad
All I can say is each of us has our own unique physiology therefore we each uniquely metabolise drugs taken.
Remember the aim of treatment is a platelet count≧50,000. Maybe the nurses and PA are unaware of this hence their disappointment.
Your daughter's quality of life is improving and her count is slowly rising. These are good signs. Personally I would stick with it and give it more time.

EDad wrote:

... Do either of you know if the slow ramp up has any link to how successful Promacta might be for her? I know the nurses and the PA I speak with on the phone are disappointed with the counts so far. That said, it is improving her quality of life so we think it is helping.

EDad, any improvement is good. IMHO, the 50mg response is disappointing for the average ITP population but pretty good for a row 2 response. A week or two flat line delay on 50mg would not be uncommon for them at all. In some cases, the lack of response is long enough, based on Promacta dosing rules, so that switching over to higher doses of Nplate becomes required to get counts out of danger.

For better or for worse, once the immune system has made a decision that something needs to be destroyed, it takes a long time to modify the decision. As adults, we rely on what our immune system learned many years ago during youth to fend off invaders.

I wouldn't consider NPlate yet. I'd stick with Promacta and give it time. My experience with NPlate was that counts swung all over the place . I wish I'd tried Promacta first then I wouldn't have needed to go on Nplate at all.

Another update --
We went in for our weekly blood draw on 5/2 and our daughter has 53,000 platelets!

Splendid news EDad

Disappointing news for us. Our daughter's 5/16 blood draw showed she is down to 17,000 platelets.
She is taking the medicine in the middle of night and has not missed a dose so we've ruled out user error for now. Our hematologist recommended staying the course and checking her platelets again in 2 weeks.

I agree EDad, Promacta can take a while to get going

Our blood draw on 5/28 got us back to 40,000. This sure is a bumpy, unpredictable experience.

Fingers crossed it stays there or climbs a little higher

If I've counted right, this is week 8 on 75mg. A 40 count is bit disappointing after this long, but regardless, it is a good count for sure. Well out of danger.