Fostamatinib trial for ITP

I've decided to start a new topic rather than post on others as I have been doing.

Have been on the clinical trial for 5 weeks now. Am optimistic this is working as for the first time have had 2 consecutive counts that have climbed, but not getting too excited just yet as previous meds have initially increased my platelet count only for it to drop again on next count

Mrs . B
Thanks for posting. How did you get involved in the study. I found the list of sites, which includes one in my area. Do i just call the contact to get more information? I have only been diagnosed for a short time so i am not sure that i meet the criteria or want to go that route yet as ther are approved treatments i have not tried, but am interested in how you became a participant. I am thrilled for you that it seems you got the drug and it is having a positive impact. Your decision to be a subject int the study may help everyone in the future have another option for treatment. Thank you!

Hi Emily

I'm in the UK..I was diagnosed last July, plus I was diagnosed with osteoporosis a few weeks later ...massive doses of Pred worked but sent me to hell and back..so very keen to get off it asap or down to the lowest dose possible as steroids are contra indicated in OP.

Tried Azathioprine that worked initially but had dreadful side effects and stopped working fairly quickly.
Next tried mycophenolate same story.
Not keen to take any other immunosuppressants as all increase risk of malignant melanoma unless one stays out of the sun..I need sunlight for healthy bones.

One week my consultant was on holiday so swapped to a different one and asked her for an indium scan which showed splenic sequestration of platelets. I don't want a splenectomy unless absolutely essential.

The new consultant is leading the trial & asked if I would like to consider it as ideal candidate.Needles to say I jumped at the chance.

So far 5 weeks in I have had no side effects at all. Of course I could be on placebo but as platelets are climbing I'm cautiously optimistic I'm not In the UK all trail drugs are free so an added bonus.

Maybe just ask you haematologist to refer you to the site in your area.

Very useful explanation on a video below, here is web address I cannot seem to hyperlink it unfortunately.
www.youtube.com/watch?v=ov2JUHDpt58&feature=youtu.be

Let me know how you get on

Anne

And then it appeared in the post after I'd submitted it

Thanks for the information. I did go to the website, but thanks for the link. I will contact the local person on monday just to get information. I was just diagnosed in january and just finished rituxan so i do not think i am a viable participant, but i like to know all options. Plus there might be another clinical trial in the future if this one is not appropriate. Keep us posted and again thank you for being a subject in the study.

I go to london on friday fro my Indium scan, but also am not sure i would ever consider surgery.

mrsb, I see that it's being trialled at Kent & Canterbury and Colchester. Which hospital do you go to? I used to be seen at the K&C.

The little video is interesting and explains it well. I wonder what the side effects are and how immunosuppressed it makes one.

I'm in Leicester..my mother used to be a renal patient at K&C. Quite a few of my family members live in Kent.

I finally got to watch the video. Interesting concept and definitely a different way to treat ITP. It will be interesting to see the long term effects and efficacy.

No side effects at all so far The trial dose was increased by 50% a fortnight ago as had not reached target count of 50. Platelets were then 39..this week they are 33 so looks like I'm on placebo. Which to be fair I knew was a one in 3 chance. Now got to sit tight for another 6 weeks then I can swap to the open label trial and be guaranteed to get Fostamatininb.

Trial tablets are dispensed every 2 weeks...Platelets rose from 33 to 144 in a fortnight. New pack of tablets 3 days ago retested today and down to 79...beginning to wonder if I was given the real drug by mistake last time as it was thought I was on placebo. Next retest in 3 days

I like the video kind of keeps it at a level everyone can understand and I am glad it seems or seemed to be helping your counts. I share Sandi's outlook and hope that long term it shows promise because the more options for treatment there are the better it is for all of us

Totally agree Mark, Video has been very useful for explaining ITP & trial to family & friends....Platelets down to 24 today so pretty convinced I'm on placebo..Will go onto open label extension trial in 3 weeks if they stay below 50. This last for 3 years to look at safety & long term efficacy. Anne

Latest update... Am deemed to have failed the randomised trial as it looks as if I was on the placebo. With the exception of 1 'spurious' reading of 144 (see previous posting) my platelets have averaged 30. I have now been transferred to an open label trial and commenced on actual fostamatinib 100mg twice a day. If my platelets aren't 50 after 4 weeks dose will be inceased to 150mg twice a day.

2 weeks on Fostamatinib 100mg twice daily...no side effects* at all so far but no appreciable rise in platelet count. Started at 20 now 25. Back to clinic in a fortnight if they aren't 50 dose will be increased to 150mg twice a day.

* commonest are nausea, diarrhoea & increase in blood pressure. Also need to have liver function & neutrophil count monitored.

Next update in 2 weeks

Thanks for the updates. I hope you respond soon.

4 weeks into the extension trial and still not a single side effect. Count climbing slowly, now up to the dizzy heights of 36!! As per trial protocol dose now increased by 50%, next count due in 4 weeks.

Looks good so far!

Mrs. B,
thanks for the updates. I am thrilled for you that there are no side effects and so far there is at least some effect on the those platelets!! Keep us posted.

I bet I'm not the only one here taking great comfort in the fact that the experts are continually investigating new treatments to add to the arsenal.

Am very glad it appears to be helping you, mrsb!

Week 8, I'm covered in bruises and count is 9 ...Hopefully due to the cold I've just had. Weekly bloods...watch and wait..may have to up Pred as a temporary measure if they go any lower. 4 weeks to go until decision is made as to continue trial or not.

Sorry to hear that. It's not good news when someone is failing a trial for a new drug.

Fingers are crossed for you Mrsb04. I am really keen to hear how you go. I am desperately waiting for the trial to be approved here (Australia) as after 15 months of sitting constantly lower than 10 I have pretty much run out of options for other treatments other than trying combinations of meds that haven't worked on their own. My fingers are definitely crossed for you!

116 this week..hopefully it was the cold as that has now virtually disappeared. Repeat count next week.

That's good! I hope it continues.

So do I Sandi but not getting too excited just yet. x

Rpetzel
What treatments have you had so far? I live in Sydney and had failed all the other drug treatments until I was lucky enough to obtain a free supply of Romiplostim (NPlate) from the drug manufacturer Amgen and the PBS.
As this drug is new to Australia I was wondering if you had had it as one of your treatments.
Thankfully I responded very well to it.

HI Robert,
Yes I did manageto get permission to try that after I tried eltrobopag with no reponse. I had the best response from the romiplostim that I have ahad for anything (got up to 18 at highest point and was feeling a lot better) however it was considered a repsonse according to the guidelines so I couldn't keep going on with it. Other than those I've retuximab (pre splenectomy), high dose of pred, imuran, and most recently Dexamethasone as well as having had several doses of IVIG and platelet transfusions. I was on dapsone/pred mix for four years and had resonably good counts during that period but haven't been able to get anywhere near back there since this last crash. Really glad to hear N-Plate is working for you. It certainly had the least amount of side effects of everything I have tried.
cheers Raegan

Thats Great. Hope it continues heading up.

That's a real shame that you weren't able to continue with romiplostim. I have a friend who was on it when I was and her counts would never go above 20 but that was deemed to be okay as was better than her usual under 5. Several years later her count gradually went up and now she's off all treatment with counts around 100. It's the first time she's not been on treatment for about 30 years. Nplate needs lots of patience, I mean years of patience. It's a shame when the doctors give up too soon.

Thanks Ann, that is really useful to know. I will definitely take this back to my specialists and particularly if the fostamatinib trial doesn't happen or it doesn't work for me maybe there's a way we can keep pushing for extended treatment. They currently have pretty strict guidelines around it here but I'm sure they will improve over time. It offers me some hope at least and I feel for your firend who was this low for so long, it is quite restrictive and I'm very frustrated with not being able to do my normal level of activities and having to give up the sport I love.
thanks again. Raegan