Hospital appointment on Friday

It was supposed to be next Monday, but the nurse phoned today and asked whether we could make it Friday instead because the trial nurse for Elthrombopag (sp?) was only available on Friday. So it sounds like we might be going ahead for some treatment - Dougie was really pleased, even when I reminded him that it probably meant another bone marrow test. Still not comfortable with it, but I've come round to thinking that Dougie's comfort should probably take precedence over mine at this point - its been three years nearly and he's not a kid anymore. Just got to try and ensure that he is well enough informed about the risks and side effects etc to make a good decision for himself.

So, to anyone who has taken elthrombopag/promacta - are there any side effects or drawbacks?

Thanks, Ali

Tim took it for about 6 or 8 weeks I think. He did not respond...but, we found out in the end that he was hiding the pills and not taking them because he didn't like the way they made him feel. He also was having some black-outs (not fainting but loss of knowledge of time that had passed). It stopped when he stopped taking the drug. its not listed as a side effect, so it may have just been Tim...he is hyper reactive to most things. I don't know what he meant by it making him feel funny, but it was enough to hide the pills and lie to me about taking them..

BTW, Tim is at 2k today...4mcg/kilo last week. They kept the same dose (ugh), so he won't likely be any higher next week. Good news is, no bleeding.

Hope Dougie gets a good response to this drug and doesn't have to have a bone marrow. Tim's never had one.

Hi Donna

I'd completely forgotten about Tim having taken Promacta. Now you've reminded me I remember about the strange episodes too. As I said, I am not 100% convinced by it, but I think I need to take a back seat at the moment. IVIG was clearly quite a traumatic experience for Dougie because its becoming clear that he doesn't want to do the other alternative (rituximab) because he is so worried about getting a drip again. I had thought that overall IVIG was still a positive experience, but he is VERY adverse to having more drips, so I think thats why he wants to try Elthrombopag instead. The doctor has already said that the trial involves a bone marrow test prior to starting, so unfortunately that looks like it will happen.

Hopefully Tim will get sorted out on N-Plate soon again; the poor lad seems to be in a bit of a dip again. Its very strange that it would stop working like that when it had previously worked so well, but fingers crossed that the next bump up in terms of doseage will do the trick.

Ali

Hi ali,
I can understand Dougie's trauma with IVIg. Its side effects can be very hard to deal with...Tim ALWAYS gets very sick from it and it doesn't really do a lot of good for him. I bet promacta will be fine for Dougie...Tim is just hyper sensitive to meds. I think Nplate will work fine for him if the doctor would just give him the right dose. At 2k this week and they don't raise it?! Idiots! But hey, I don't have a med degree...just a son that I told them would drop and have great difficulty getting back up if they didn't give him the Nplate regularly...hmmm, guess I was right. But what do I know? He can get up to 10mcg/kilo, so hard to say what dose will work. They do tend to develop some resistance to the drug over time, so I hope that's not the case for him. I'm interviewing for a new job tomorrow that pays so much more and has insurance, so I'll hopefully get him some better coverage and move him to an adult hemonc that's not afraid to actually SEEEEE him (he never sees the doc) and give him the meds he needs.

I hope that Dougie gets a good response with promacta and that bma goes easily for him.

Ali -

We don't have any experience with Promacta but just wanted to wish you and Dougie luck on Friday. I hope all goes well and Promacta is the answer for him. Brady has a really tough time with i.v.s also, so the thought of taking a pill was appealing to him too. But when we talked about Promacta several years ago there wasn't a trial for kids and then Brady started recovering a bit and we didn't pursue anything different.

Keep us updated and I hope we hear of Dougie's daredevil excursions again soon!

Wow, to go from no treatment at all to actually treating is a big change! I know you have done a few treatments recently due to his infection. It is so much better too when the willingness comes from the patient because you know there will be full cooperation. Caitlin demanded treatment (even a splenectomy) after 9 months of no treatment because she could not stand the restrictions!

Donna-I just found out today that they can do an antibody test. Apparently they do this at least once a year, sometimes more if they feel like the patient is not getting a good response to see if they've developed antibodies to Nplate. Caitlin is getting her test done next month because it has been a year since her last test. They also said a bmb is not warranted unless they see changes with the cells under the microscope, which they do at every single visit. (I got that answer when I asked if she would need another one soon due to the reticulin two years ago.)

Hi

You are right, its a big change to go from no treatment to something like this. I'm still not at all convinced that its the right thing to do.

However, the last time we were at the doctor, we got carpeted over Dougie's activity level and this was followed up by a particularly stern letter! The doctor obviously feels that we are being highly irresponsible about safety. Since then, I've tried to limit his activity to what the doctor will allow, but its not working - I know for example that today he played football at lunchtime. The doctor is absolutely insistant that if Dougie can't be trusted to obey the rules then treatment is the right route. I do feel that there is little point in seeing the doctor if we don't listen to him.

If Dougie has to be treated, me and Dougie's dad would prefer to try rituximab, but as I said, Dougie is really really not happy about getting anything else in the form of a drip because he associates it with the evil IVIG. I am not convinced that he would take Elthrombopag for long either if it had any significant side effects though. He would be very intolerant of anything that made him feel ill because ITP itself doesn't usually give him any significant problems, so I don't think he would put up with any solution that he perceived to be worse than the illness itself. Oddly, the bone marrow thing doesn't seem to bother him at all - possibly because the effects only lasted for a couple of hours max.

Not easy!!!! Perhaps his count will miraculously creep over the 30 mark before Friday?

Ali

Dougie's count today was 15 - not bad, not good either. I thought it might be better because he has zero symptoms.

We think we will go ahead and try the ethrombopag. The trial opens in about 4-5 weeks. 2/3 of the patients get the real drug for 10 weeks and 1/3 get a placebo. Then after the 10 week period I think everyone gets the real thing. I think the whole thing is 26 weeks, after which the drug company provide the drug on a named patient basis if it is of benefit (as it is not yet approved by the NHS for children). We met the research nurse today and she explained all the commitments required - for example, you have to go weekly to the hospital until they get a stable response. She reckons that we will get some travel expenses reimbursed though which is good given the price of diesel or train tickets. I am going to talk to the school about this next week because it sounds as if Dougie will miss a fair bit of school.

All in all quite positive, although Dougie would like to start yesterday rather than in a month. :laugh:

Ali

Hello

Hiya, hows Tim doing this week?

Hi Ali,
His platets are 6k. They gave him 4 mcs again, and I was steaming. I asked to speak with someone and they at first said that it wouldn't do any good. So, finally they went to the nurse practitioner who called the homeonc. He came in to talk to us. He decided to do a BMA and BMB this Friday (Tim's first). He also said he'd send for another mcg of nplate to make it 5mcg (finally). The antibody test apparently is not available commercially, so we can't do that. He mentioned splenectomy again, and I reminded him that there are two failed splenectomies in the family already. Well, that's what happens when the doc never sees him...they don't remember the case. I'm gonna post this on the discussion board too.

Poor Tim! Its a good job that you are still there for him - my 18 year old wouldn't know where to start talking to a doctor, but equally, he wouldn't let me go with him either! I don't think its reasonable to leave a kid of Tim's age sitting on that kind of count - even if he doesn't have any bleeding, they don't seem to be able/willing to take account of activity restrictions. I would guess that Dougie is also sitting around 5-10 too, and I came home from work this evening to find him playing takedown tag in the park with friends (if you don't know what it is, it involves roughly the same level of physicality as American football but without the ball or any rules!)

The Bone Marrow thing wasn't bad for Dougie - he was out riding his bike three or four hours later, He did complain that the puncture site hurt for a few days, but it clearly wasn't much.

I hope your stropping with the doctor produces something more in the way of both involvement and treatment

Ali

Alison, Good luck to Dougie with the trial. I can understand the difficulties of moving to active treatment Niamh my 18yr old went from the wait and watch approach to various treatments in March 2011 after 14 yrs of ITP.

Thanks Mary - I am still not 100% comfortable with treating, but Dougie is, and I guess thats what counts! Can't imagine dealing with ITP for 14 years! I am still very hopeful that it will just go away by itself (this might be wishful thinking on my part, but I am holding onto that hope!)