Update - ITP Fostamatinib trial

Hi - i was diagnosed with ITP 18 months ago and so far nothing has worked....I am currently on the trial drug Fostamatinib. I started the trail 6 months ago - it was suspected i was on the placebo for the first 3 months as nothing happened and i had to have several IVIG transfusions to get my levels up. I have been on the 2nd phase of the trial since January starting on 100mg and now up to 150mg. Jan and Feb nothing really happened....levels hovered in the 20's but in the past 2 months levels have gone from 24 to 31 to 72 to 35 and now currently at 59. Apart from IVIG nothing has kept my levels up and they normally plummet into the teens very quickly. Having had no side effects at all either in the past 6 months i have now also been experiencing very nasty gastric problems, it is more settles now but has not been fun! Anyway what i would love to know is if there is anyone out there who has been on this trial and if so how it went for them. I am back in hospital this Friday to check my levels and am just praying they have held and are in the 50's or higher.

I did both phases in the UK and it didn't help at all , here are links to my forum posts

pdsa.org/forum-sp-534/7-treatment-general/28623-fostamatinib-trial-for-itp.html?limitstart=0

pdsa.org/forum-sp-534/7-treatment-general/28869-life-after-fostamatinib-trial.html#51038

Hi - i thought i would update you on how I am doing on the trial. My platelets have now been in the 50's for the past 2 months which for me is a record and they have never held like this since i got ITP nearly 2 years ago. I started the trial in September 2015 and am glad i stuck with it as in February when it did not seem to be working i did nearly give up on it all. Apart from having the gastro problems this has been fine to take and hopefully my platelets will remain stable now and stay in the 50's which i will be very happy with. Hopefully this drug will become available to everyone soon and that it helps other people too. Will keep you posted.

Great to hear

Hi everyone
I thought I would update you. I am still on the trial, am actually the last person where I am being treated on the trial as the others could not deal with the gastric side effects which are not fun but this drug is the only one that has kept my platelets up - last two blood tests they were 52 and then 63 last week. Am very glad I have stuck with it and despite the side effects I am going to stay on it. I just cannot face coming of it and then starting a new drug which might not work and then going back to the weekly blood tests and constant IVIG transfusions. Better the devil you know! Will keep you posted - the trial is not now taking new patients but hope it does become available to everyone in the future and I am glad I have done my bit to hopefully help others with ITP.

sachmo16 wrote: Hi everyone
I thought I would update you. I am still on the trial, am actually the last person where I am being treated on the trial as the others could not deal with the gastric side effects which are not fun but this drug is the only one that has kept my platelets up - last two blood tests they were 52 and then 63 last week. Am very glad I have stuck with it and despite the side effects I am going to stay on it. I just cannot face coming of it and then starting a new drug which might not work and then going back to the weekly blood tests and constant IVIG transfusions. Better the devil you know! Will keep you posted - the trial is not now taking new patients but hope it does become available to everyone in the future and I am glad I have done my bit to hopefully help others with ITP.

Well those counts sound great! I'm glad that the trial is working for you and kudos for sticking out the nasty gastro stuff. I don't blame you for sticking with something that seems to be working when everything else wasn't!

Keep us posted and good luck.

Interesting about the gastro side effects maybe the fact that I was taking Ranitidine prevented me getting any

Hi - I have now been taking Fostamatinib for nearly a year and am glad to say that it is still the only drug that has worked for me and is keeping my platelets in the 50's. I think my body has now adjusted to the drug and the side effects are so much better and the gastric issues are significantly better, they flare up again sometimes but nothing like they were when i started. The trial has now ended and hopefully the drug will be licensed and other people with ITP can get to try it for themselves, i nearly gave up with it countless times due to the side effects and how long it took to finally work but glad i stuck with it!

Interesting. Glad it is working for you.

Have they been adjusting dosage up and down to get to this happy compromise between stomach issues good counts? That is, were you having the more severe stomach issues at a higher Fostamatinib dose?

Glad it worked for you it didn't for me. Sachmo16 you might be interested in this www.pmlive.com/pharma_news/rigel_shaken_after_fostamatinib_misses_phase_iii_trial_target_1173284

Hi - I started on 100g for 3 months and then dose has stayed the same since - 150g, it has just got gradually better and my Consultant thinks it must just be down to my body adjusting to the drug. I am wondering that when it does get bad that it is linked to something i am eating so trying to keep a food diary. I was one of 3 people on the trial at the hospital and the others all gave up because of the gastric problems. I so nearly did but just could not face trying another drug....they all seem to have side effects so just stuck with it and prayed it would get better!!!

thanks - very interesting

Hi - I started on 100g for 3 months and then dose has stayed the same since - 150g, it has just got gradually better and my Consultant thinks it must just be down to my body adjusting to the drug. I am wondering that when it does get bad that it is linked to something i am eating so trying to keep a food diary. I was one of 3 people on the trial at the hospital and the others all gave up because of the gastric problems. I so nearly did but just could not face trying another drug....they all seem to have side effects so just stuck with it and prayed it would get better!!!

Hi - just thought i would send a quick update - i am now over in a year into the trial and so far so good. My platelets have reached a record 92! They have been gradually increasing over the past 4 months, 40's then up to the 60's and then the 90's last week, could not believe it! Side effects very are now very manageable.

Hi Sachmo: I just had a failed Splenectomy surgery (11/13) and my Hemo is considering prescribing me Tavalisse. Can you give us an update on how you are doing on this med, and a little more detail on what you experienced regarding the GI side effect? Thanks and I hope you continue to do well!!

Hi -sorry I have not logged on for ages. I am still on it and my platelets got up to a record 180. The dose has now been reduced and I am on 100mg twice a day. Platelets still holding and side effects fine. I have now though got other issues and potential Lupus. My hands and fingers started swelling up and I was in serious pain in my hand joints. I am now taking hydroxychloroquine which has worked but all very frustrating as I have been fine on the fostamatinib and all is now need is lupus!

Thanks for the update; so sorry to hear about the Lupus symptoms, but great news about your counts! Since my failed splenectomy, my counts have ranged from 6K to 172K on Promacta, with the variability due to modest changes in dosage (e.g. I dropped dosage from 50 mg daily to 37.5 mg and my numbers dropped to 68K to 6K in one week--go figure!). Anyway, I'm hoping that Tavalisse might serve as a "backup" option should the efficacy of Promacta wane.

As for your 180K counts, is your Consultant concerned that the numbers are possibly too high; especially with the "sticky platelet" question (i.e. my Hemo says that the TPO's stimulate brand new "sticky" platelet production, which can create a risk of possible clots at higher counts...) ?

Tavalisse isn't a TPO but works by preventing the destruction of platelets so doesn't have the problems that the TPOs have with clotting.

Good point; thanks for the clarification.

sachmo! The only one here to have had a good/strong response to Fostamatinib. Nice.

Sorry to hear about Lupus. Let me ask a question on that. Do steroids help with symptoms? I guess I'm wondering if steroids helps all those with Lupus.