Trial Study of the Promacta for my 5 year old

So we have been on the study for 7 weeks now. NO results yet but we are limited to a low dose and can only increase every two weeks. Her age group is not a pill but a liquid that she takes daily. We go weekly and have had to treat her twice with the traditional Prednisone and IVIG to get us through since her numbers dropped to 2K. The last two weeks she has been at 24K. She rarely holds still so I am hoping, just maybe this medicine might start kicking in. 18 months of this is wearing on me and my concern is what is next??? If this doesn't work, I am not sure what we will do.

Jennifer
Mother to Charlotte
Diagnosed March 11, 2010

Hello Jennifer. Glad you found these boards. Though I'm sorry you had to! There is a whole lot of support here. I don't have children with ITP so I'm not going to pretend like I know what you're going through. I do know that with younger children there is a chance that they will grow out of it. How you got into a study like that? Thank you for letting your child do it for all the other children out there who need help.

Hi-

My 3 year old son is on the Promacta trial as well. We are in week nine and are on our fourth dose increase. He is currently taking 17 ml (34 mg). What dose is your five year old on? I am nervous about what it might do to his body given that we are approaching the adult dosage amount. I mean he is 36 pounds and most adults are well over 100. We have not see the expected results. His platelets were up one week to 53,000, but now are back to 21,000. We started the trial at 7,000. He has tried most other treatments with no improvement, steroids, IVIG, rituxan. He is RH negative so he can't do WinRho. In any case I know there are only 5 of us right now on the trial in this age group. Where are you from? We are in Chicago.

She is now on 23.5 (47mg), she weighs 56 lbs. We saw a bump last week to 25K, first time so we are hopeful. We are in Charlotte NC. I am nervous as well about the long term effects but I reassure myself that I am doing what is best for her now and trying to give her a "normal" life. We have done the normal drugs, IVIG, Steriods and WinRho. We did not do Rituxan. We have the best results from WinRho. We have had to treat during the study because she got down to 2K. I believe there are only 3 kids in the US in the trial. I was interested to find out how the others were doing. They told me they think a delayed response is possible.
Please keep me posted, it is so nice to hear from someone else going through this. How is your son dealing with it all. My daughter was diagnosed at 3.5 years old.

Hi-

Wow, I didn't know there were only three kids. My doctor said there was supposed to be five for this first part. My son Sawyer seems to be tolerating the medication okay. Sawyer was diagnosed in March of 2010 as well. He turned 3 last month. We have reached our maximum dosing, so we can't go any higher than the 34 mg. I am glad your daughter's numbers went up. Sawyer's counts are in the 20's as well and I feel like if that's as good as it gets that's good enough for us. How do you feel? When your daughter's counts are low, does she have significant bleeding?

I totally have reached my breaking point as well after 18 months of this. I'm glad you posted on these boards. I don't feel so alone anymore.

Laura

I was wondering what Promacta is? And how you found out about it? Our daughter is 7 and has had IPT5 yrs and nothing is working. In fact it seemas as if everything we have tried is making it worse. Her platelets are around 3, oo all of the time and she has alot of active bleeding. I have been looking for other things to try.

Brookechris,

Have you considered looking at more natural methods of treatment? My boy was at zero for 3 months and we finally started using homeopathy. His last count was 136K and his entire body is healing of a lot of medical issues he's had over the past 10yrs. You can look in the natural section and you'll find stories, information, etc.

patti

Patti, No we haven't tried anything like that yet, but we are open to it. I will look into it because I would rather try something that is natural and more healthy than to keep trying experimental drugs. I worry so much about her health in the future and the side effects of everything we are already using. Thank you so much.

Brooke and Chris

Laura,

We went today and they were back down to 16K. Still holding on for hope. There are 5 kids, but only 3 in the US. Two in Europe. They bumped her to 30ml(60mg) because of her weight. This will be it. It is nice to meet someone else going through it. I feel like when I explain it to people they don't get it. I get tired of saying it could be worse, so now I say it could be better.

Today C told me she doesn't mind the pokes since it will make her healthy. Comments like this are sometimes hard to swallow and just don't seem fair but I know she can handle it will be strong from all of this.

If this doesn't work we might move to a homeopathic route. We haven't tried Rituxan and of course a spenectomy is an option but not sure we will do either of those.

My husband and I agree to at least give it 4 more weeks to see what the max dose does or if there is a delayed response. What is your plan? HOw is your dr feeling about the drug?

Jennifer

PS, I think I would take consistant 20's. I am okay in the 20's and feel like she could be normal in that range.....

Good news, her numbers went up to 30K. So maybe the meds are working. I am hopeful. I ready for her to learn to ride a bike, not say no to most birthday parties and just be normal. 30K is exciting and gives us a week to relax!!

Amen! Glad to hear they are up.

30K-that is great! Sawyer's platelets went up from 21,000 to 101,000 last week, but then back down to 60,000 this week. I am just glad they did not go below 50,000, because now we don't have to go back for blood tests for a month. We need a break. I think their bodies just have to get used to the drug. I also have been hyper-vigilent about not letting him have remotely anything to east that is vitamin enriched or even contains the slightest bit of calcium 2 hours before and 4 hours after he takes the drug, so I wonder if that has helped. It is hard to know. Sawyer wants to learn how to roller skate now that his platelets are up.

We fell back to 22K but that is good. Wow 101 that is exciting. Even 60 is exciting. I knwo I was thinking of throwing her a platelet party to celebrate. Take her Ice Skating. The little things that are exciting. We are bumping up again to 75mg. Crazy!! I am excited for you. I am sure Sawyer is ready to be normal!! I am going to start keepinga food diary of her food. Maybe that will help!

Well we are riding around the in the 30's. This past week was 38. I will take it. The meds seem to be working, just not sure why not a little more. She actually had a lot of bruises on her legs so I thought for sure we in the teens.

Charlotte is thrilled she can do most of the things she was limited to.

Thanks for following!!

Jen

That is great. Yes, it must be so nice for Charlotte be able to be a "normal" kid again. How is she doing taking her meds every day? Do you have to mix yours in water from a powder? I am kind of tired of having to make Sawyer's meds everyday and save the packets, but it is working and that makes it all worth it. Sawyer was at 70,000 last Thursday and we go back in a month. Right now, we are at week 15 I believe. We took the kids (i have an older son who turns 5 today) to Disney World a couple of weeks ago and it was great to watch Sawyer have so much fun and ride on rides and what not. I am just so excited that we can potentially go sledding this Winter. We missed out on that last year. Sledding is probably not the best idea when you have 5,000 platelets!

Laura

Laura,

We jumped to 65K this week! So we now got approved to go every 2 weeks and I think one more good number we will be monthly. It is so exciting to not worry. Charlotte requested to learn to ride her bike, we held off since there are bound to be lots of falls, ride her scooter and also go to bouncy houses. I bet Disney was so fun. Funny how something so simple as Sledding can be dangerous to our kids. I am tired of mixing, I kind of wish I could make a batch for the week and just pour it each night. For now I won't complain since it is a lot better than getting hooked up all day to an IV.

Keep me posted on Sawyers progress. I like knowing someone else is out there!

Jen

Well it has been more of a rollercoaster. The Promacta is working. we bounced up to 68, dropped to 3K (she had a cold) then to 75K and then 455K. They now have lowered her dose back to 54mg and she fell this week to 86. Our concern is the dose is too low so another CBC on friday will give us a better idea.

I get excited and then let down but I am now happy to know this drug is working and giving us some relief. She is so excited she can do all the activies we have worried about.

That's crazy. We have had quite a rollercoaster ourselves. So Sawyer's count last month after we got back from Disney was 73,000. He was sick for about a week straight with a highish fever for 4/5 day. When we went back for our monthly visit on the 17th of November his count was 910,000. We stopped medicine at that point and then went back in for another count the wednesday before Thanksgiving. It was 1,042,000. We continued ot to give him meds and then went back this past Monday and his counts were down to 400,000. We go back in next Monday. The doctors have no explanation. It is sort of ridiculous. I don't know what is going on with his body and since he is not on the meds I am just waiting for something bad to happen. Two nights this week I woke up panicked expecting him to start bleeding from his nose and gums like in the past. It is kind of a nightmare.

Are you guys doing okay otherwise? Did you have a nice Thanksgiving at least? Take care and keep me posted.

Laura

Well we reduced the meds and she went back down to 14K. UGH!! So now they are calling the study chair to bump her back up.

That is crazy about Sawyer's numbers. 1mil???? I know it is hard to believe that we beg for high numbers and then all of a sudden we want them low. ITP can be so frustrating on so many levels.

Keep me posted!!! Thanksgiving was fabulous, we traveled to Ohio to see family and with her 400K numbers it felt good for me to let her be and just play normal with her cousins. Back to reality and more pokes.

We go back this wednesday for more blood work.

jen

So we went back in on Monday and Sawyer's counts were down to 29,000. We are back on meds, but one dose amount lower than the originally dose amount that was working. This dosage amount did nothing for Sawyer until we increased it to 34 mg, so I am not expecting much to happen. We go back in Monday. I don't understand. How am I supposed to regylate this? If he gets really sick, his platelets go up and the doctors can't explain why.

Laura

Laura,

We are in a similar situation, we boosted to 455, the lowered it and we fell to 3 then climbed back up. She was sick when it dropped. They then moved it to 65mg and we fell again last week to 25, we went today and we were at 58. Again she was sick last week with a cold. Seems like we can't get it to level out.

how is sawyer? I hope he had a good holiday. Charlotte got a new scooter and it was alittle nerve racking watching her learn and fall and bruise.

Hang in there keep me posted!!

jennifer

Laura - unfortunately, you can't regulate the counts. The goal is to just keep a safe count (over 30,000) for as long as it takes.

Well we are off the study and released back to our Doctor in SC. So no more weekly 3 hour round trips except for the last few eye exams. The numbers fell again but we can't pinpoint why. I am thinking food....

At least we have enjoyed 30's. I will take it. She loves not goign for her weekly poke and I love not seeing the dr every week waiting on the number.

Jen