Experiences with Promacta?

We went for yet another opinion with a new hematologist today and decided to try Promacta. I was hoping to get some feedback of experiences and opinions. I'm concerned about side effects and the fact that there isn't any long term data in children.

I know there are no kids at the hospital that we go to that are currently on Promacta. I think we've had one or two kids on here who have tried Promacta and I'm not sure if it was successful for them or not. I hope they will post.

Promacta and Nplate are both TPO drugs, which mean they stimulate the bone marrow to produce more platelets. Promacta is the pill form and Nplate is the injectable form. Promacta must be taking daily while Nplate is given once a week. My daughter has been on the trial and I think there are currently pediatric trials for Promacta. You will have to keep us updated on your daughter's progress as well as her side effects since this is a very new drug for children.

I'm not sure, but I think that Promacta maybe still at trial stage for children, here in the UK anyway. There are some trials at children's hospitals going on for about the last 18 months - the one I know about is at Manchester Royal Children's Hospital. There must be some published data by now you would think???

Lucidawn (donna) who posts about her teenage son Tim might be able to help you, because I seem to remember that Tim took it for a while (but wasn't reliable enough about taking it for it to work properly!)

We're participating in the study of Promacta at the Children's Hospital.

We decided to try Promacta before N-plate because I think a daily pill will be easier on DD than a weekly injection. There are very specific ways you have to take it though (2 hours after or 1 hour before any food. 4 hours before or after any dairy or fortified juice). It seems complicated, but we're just gonna give it to her at bed time and no milk with dinner.

Tonight will be her first dose and the new hematologist said she should show a response with the week, if she is going to respond at all. I'll update as to what happens.

We're all pretty upset that the Rituximab was a complete failure. I would definitely say this past year has been the roughest on her in the 4 years since she's been diagnosed. DH and I had recently decided to go ahead with the splenectomy, which one of her docs has been really pressuring us to do. My poor little girl has been really depressed lately, saying things like, "I want to be a normal kid...I don't want to be sick anymore." We thought that surgery was the only thing we hadn't tried.

I'm really glad I asked for another opinion! The new doc talked about these TPO meds but also about several other options like 6MP and Rapamycin. Anyone have any experience with those? She also talked about Vincristine but I'm more willing to try an immunosuppressant than a chemo at this point.

6MP IS a chemo drug, as well as vincristine. 6MP is given to children with leukemia. I am glad to see you contacted April. She helped us tremendously, and I know she can help your daughter! I'm sorry she's going through this so young. I'm so very glad you did not decide on a spleenectomy at this age. Hang in there.

patti

I wasn't really sure what the difference was between immunosuppressants and chemo drugs, and this new doc talks about them as if they're in different categories. The immunosuppressants don't actually kill cells the way that chemo drugs do. Chemo can suppress the immune system, but that doesn't make all immunosuppressants chemos. That's how it was explained to me...I think

She said that the 6MP is an immunosuppressant that's given in really low doses AND it's been used for more than 30 years. I guess that's why I would feel more comfortable with it. There's some data about long term side-effects.

But I'm honestly not THAT comfortable with any of it, all of the meds are scary. We just want to fix our little girl.

How is your daughter doing on the Promacta so far? Is she having any side effects? Caitlin's main side effect (and I think they are the same with both Nplate/Promacta) when she first started was the headaches and she got them often. Her stomach hurt every now and then too, but it was still better than what she experienced with IVIG.

Hopefully your daughter's count will start to rise in a few weeks and then you will see a pattern of stabilization with her counts. It took Caitlin a few months to get there. I am curious how they will work the doses with children in the pill form since they come in certain doses. With Nplate, I give her the exact amount she needs based on her weight.

Read up on those other drugs on here. Be wary of the side effects for some of the other drugs. The best thing about the TPO drugs (and this isn't for everyone), is the minimal amount of side effects that are experienced, but the downside is that we have no idea what this is doing long-term. (Caitlin has had the increased reticulin in her bone marrow from Nplate, but they decided after a few months that she should be fine to go back on because the reticulin goes away when they stop the drug. She's been on Nplate 8 months this time around and they haven't done another bone marrow biopsy. I think they also haven't seen any changes with her CBC's to warrant another BMB.)

So far, she hasn't had any side effects. Nothing that I've seen and she hasn't mentioned anything yet. I take her to get her levels checked on Friday so I still don't know if it is helping or not. The dr told me for sure by 2 weeks we'd know, but should show an improvement by this week. The rules about how she has to take it upset her some. Last night at her Girl Scout meeting, she wasn't allowed to eat the snack and she got pretty upset. I want her to be as compliant as possible, so that way we know for sure we tried our best with this treatment.

The increased reticulin thing horrifies me and I'm not sure I want her on this medicine long-term, although I do fully understand how it works. I flip-flop daily and am really unsure how to cope these days. She got another long bloody nose today and has more bright blue bruises. The petechiae on the tops of her feet seems to be fading a little but it's sticking around everywhere else.

*sigh* I dunno.

Anyhow, I'll make sure to keep you updated on how it's working and on any side effects.

Oh, almost forgot: right now they've started her on 25mg, so I am not sure how it will be adjusted. The dr briefly talked about it along with using a combo of meds with it, but I guess we'll cross that bridge when we get there.

I understand your fears about the reticulin. I was very scared when I first found out Caitlin had it and I still have that fear in the back of my mind because I don't know if it is still there or if it will get worse or stay the same. And what's worse, is that the drs don't even know what could happen if the reticulin gets to the point where it can turn into fibrosis or collegen(sp) because they haven't really come across enough adults who have had this. I researched this over and over again and really found out that many people have reticulin already in the bone marrow so that was why I decided to allow her to go back on when we felt like there was no other option. Thankfully, she is doing great and there have been no weird changes with her CBC's that make me think other things are going on.

Oh the decisions we have to make as a parent! I just hope they never come back to bite us in the butt! Caitlin is old enough though that she participates in the decision-making process. When she was being withheld from some competitions last year, she kept begging to try more Prednisone and other meds so that she could compete. She wanted to do whatever it took to get her counts up.

I am keeping my fingers and toes crossed that you hear some good news tomorrow!

Well, here's our update:

The Promacta has done nothing to improve DDs platelet count and she's been complaining about joint pains and headaches. I was supposed to hear from the new hematologist yesterday to suggest the next direction, but I don't think we're going to continue down this road anymore. The risks don't outweigh the benefits (of which there are none thus far).

I'm a little disappointed but at the same time relieved because I, personally, was never comfortable with this treatment anyhow. We're making decisions about her healthcare as a team (her opinions, the docs and our opinions, as parents, are all taken into consideration.) and Promacta was what "the team" decided to try.

So, in the meantime, she's covered in bright blue bruises, her nose is bleeding like a faucet, but her petechiae seems to be fading slightly. I've kept her home from school most of the week, but since we've been pummeled with snow, they've had a couple snow days anyway. She's technically only been absent one day so far, so I suppose the timing is lucky. Last count was 14 and I'm a little surprised she's so symptomatic at that level. It's almost as if her blood seems "thinner."

I wish I had better news to report, but this is where we are. Something we hadn't considered before is a homeopathic remedy, so that's our next step. Anyone have experiences to share about that?

Jupiter,

Our son has been doing homeopathic treatment since Aug. of last year and this week his count was 188K. He's had ZERO side effects from the treatments and as a bonus, 39 of his 40 food allergies are now gone. We are down to dairy being the only allergy he has left. Since the entire body heals with h-pathy, we are seeing his whole life change. His platelets have bobbed around as his body has healed but never less then 35K (for someone who'd been at zero for months, we were fine with 35K!). I highly recommend the h-pathy. Just make sure you get a good practioner because it matters! If you don't have someone who knows what they're doing, it won't work. It has to be prescribed correctly.

With the drugs, it can take a bit longer for h-pathy to work as the body has to clear them out. We didn't get much response until we cleared out the benadryl from his body - then he went up. Then we cleared out the prednisone and he went up yet again. He has bobbed between 35K and 240K since then. Two weeks ago our son went through a MAJOR healing crisis and as best we can tell (h-path, ND, and us) his body was clearing out the IVIG out of his cells that he had last May. We were told IVIG only lasts in the system for 4-6 weeks most of the time. But it was clearly inhibiting my son because as soon as it cleared his system he completely changed. I expect we won't be seeing him jump around so much now, in terms of counts. So you will need to be patient with the h-pathy as her body clears out her system of all the drugs she's had. But it will and does work!

patti

I still think not enough time is being given to allow the Promacta to work. BUT, at the same time, you need to be comfortable with it and obviously you are not, so you are doing the right thing by taking her off.

I wish you the best of luck following the natural remedies method, but please take her to a dr if she is having any major bleeding. Homepathy isn't going to save your daughter in the event of an emergency. Make sure her hematologist is aware of the approach you will take with her and hopefully they are on board with you. I know ours is fine with whatever we want to try as long as we make sure we bring her in at the first sign of major problems.

The first two weeks will be the hardest because she will have alot worse symptoms than before. We kept our daughter on Prednisone for the two weeks afterwards to try to minimize some of the symptoms and it did help. Her platelet count did not get better though until 6 months later when we put her back on the Nplate. For the most part, her symptoms were not too bad either, but we did have a few visits to the ER during those 6 months. I wish you and your daughter lots of platelets and minimal symptoms during your new journey!