6 Months with ITP- 10 Y Son (Is Eltrombopag OK)?

Hi All,

My son was diagnosed with ITP last August and was initially responsive to Prednisolone. However, when it was tapered off in 1 month, his platelets went down from 126K to 7K. He was then advised to be on 40mg but his platelets never went up beyond 25. It will range from 7-15K. As it was low, he was on this dosage for 2-3 months and he had the side effects of the steroids (moon face, not growing, etc.). We decided to have a steroids break and have an IVIG. His platelets only increased for a few days and went back down again. With this, we felt we needed to stop the steroids and taper it off. His last dose of 5mg was Jan 6, 2016. Since stopping, he has a lot of purpura and petechae all over his body. His last count is only 3K and the doctor wants us to look into a new medicine for children (Eltrombopag). I have done papaya leaf extract, Chinese herbal (2 1/2 weeks) and leaky gut help (probiotics and digestive enzymes) but nothing seem to help/work.

So far, his purpura comes up and heals in 2 days and a new one comes up.

It's been an up and down battle on this. At one point, I feel that there is too much toxins/medicines (incuding steroids) in his body that I want to wait but on the other hand, I'm also scared of his low platelet count. Wondering if Eltrombopag is an ok drug given it is quite new to children.

Hope you can give your perspective.

Regards, Concerned Mom

Aubrey:

All treatments have risks, and it is hard to decide what the best course of action (or non-action) is. Adults have been having success with Eltrombopag and as far as I know, it has been okay for children too.

When making treatment decisions, it is all about benefit vs risk. Make a list of pros and cons for treating or not treating, and hopefully that will guide you towards an answer.

Hi Sandi,

Thanks for your note. I have searched the internet and have seen case studies on adult for Eltrombopag but not for Children thus my worry. His last count last Thursday was 3K so the doctor is also worried about not giving him anything. We are trying to be as careful as much as possible. A friend of mine recommended that we see a immunologist. Is that common?

ps. we had a bone marrow test and everything is normal. I saw that one of your post mentioned that while the bone marrow test returns normal, it may not indicate that the production is enough?

Aubrey

Aubrey:

Eltrombopag is also known as Promacta. Here are some articles about its use in children:

www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm459430.htm

www.us.promacta.com/chronic-immune-idiopathic-thrombocytopenia/

www.empr.com/news/promacta-now-approved-pediatric-itp-patients/article/420331/

www.childrenscbf.org/content/fda-approves-promacta-children-rare-blood-disorder-itp#.Vqb6m1KacSU


Immunologists are not usually helpful for ITP. We've had a handful of people see one over the years and none of them got any real answers. If it were helpful, it would probably be recommended by hematologists or pediatricians and it never is.

True, a bone marrow biopsy will not determine adequate production. It has been determined that most people with ITP have destruction as well as production problems.

Here is a case study re children and N-Plate. I'm sure there are articles about Promacta too. It's late so I'll look more tomorrow.

www.bloodjournal.org/content/118/1/28?sso-checked=true

Dear Sandi,

Thanks for all the materials here. I finally ordered Eltrombopag as it takes 3 days for it to arrive here. I'm having it on standby as the last CBC was only 2K platelets. It's weird that his platelets are lower vs. last but his petechiae and purpura are lighter. I thought it's going to be higher...anyway, while we are scared, we are just extra careful. We still let him go to school but no PE. I also send and pick him up upto to the classroom to lessen the risk.

We did a liver test for him to know the base but found out that his ALT is slightly high (3x normal). Am not sure how to react to it but the doctor says it's not extremely high. We just need to check it to make sure it's not cause to any virus???

We were also surprised to see his RBC went down a bit to 9+. Again the hema says to monitor.

Now, I'm just hoping for a slightly higher less dangerous platelet level and normal results of blood and liver... I don't even know if Eltrombopag is a good option with his slightly higher number in ALT.

Praying for God's healing soon.

ps. have looked into negative ion clothing. am not sure if that is common in your area. It promotes healing and balance overall.

I would think that the elevated liver levels might be a concern. Sometimes low platelets can be the result of a problem with the liver. Also, the drug can sometimes cause elevated liver levels, so they would have to really watch that. If the doctor isn't that concerned, you could probably give it a try but I'd make sure they stay on top of that.

Hi.

When my 15 yr old daughter was on Promacta, her AST and ALT started normal, then after about 12 months of being on 62.5 mg they spiked to 10x normal levels. She stopped using Promacta for a while, AST and ALT got into range < 3x normal so she started Promacta again and her AST and ALT eventually got into normal range.

However, shortly thereafter, Promacta stopped working and she is now on Nplate. She seems to be responding to that but too soon to say for sure.

My 18 yo daughter started on Promacta in June. When she started one of her liver enzymes was very mildly elevated ( I am sorry I don't remember which one, I will look at it later). It had also been at the same level off and on several other times when she had lab work done. The doctors monitored it with her bloodwork and it actually came down within the normal limits during her treatment.

I struggled with the concept of treatment, we did watchful waiting for a while and her platelet levels were very low. She mostly had nuisance symptoms though which she tolerated, and overwhelming fatigue. Last winter though she had some GI bleeding and a platelet count of virtually zero so she had to do rescue treatment with IVIG and high does steriods which brought her counts way up. Two months later they dropped very low again and we opted for treatment with Promacta. She responded very well with minimal side effects and she is a different person now. It is really great to see her really feeling well. And it is also somewhat of a relief to not have to worry quite as much. Last year I came home from work one day and she was sledding in ice packed snow with the dogs chasing and jumping on the sled. All I could picture was a head trauma with a count less than 5k. Yesterday when I came home and she was sledding I could think about how much fun it looked like, and what good exercise it was.

Dear TerriC/SCS2521,

Thanks for sharing your experiences. I wasn't able to be online for a few days as we had to admit my son to the hospital for gum bleeding which was due to a loose tooth that was about to fall off. The doctors prescribed him some medicines (tranexamic acid and some gargle) to help with the blood clot and since his platelets were 2K, he got some IVIG (double the dosage of what he got in December). His platelets went up to 35K and we were discharged. We will see the doctor later to check on his platelets. Interestingly, we ordered the Eltrombopag for standby last week and the pharmacist called to say that there is a SHORTAGE of this medicine in the whole of Singapore and the latest shipment will only arrive end of Feb. I guess then this postpones the potential use of the treatment which maybe it's a sign.

Have your girls gone to remission post the Promacta or N-Plate?

Sandi, my son's ALT levels went down to normal so it maybe some virus or residual effects of the steroids. Shoo! Again, thanks for your passion to help 'lost/worried' moms like us. It is indeed a roller coaster ride, some days you are quite positive and some days you are so low but this site gives me the support and idea that we are not alone in this battle.

Thanks!
Aubrey

Well that's some good news! (ALT)

Hi All, What's your thoughts on wait and see vs. eltrombopag with very low platelets (below 20K) with petechiae on legs (a few)? IVIG and Steroids don't seem to work long for him as I notice also for other kids/adults.

It is a personal choice based on symptoms, lifestyle and how your son feels.

When my daughter (17-18yo) was diagnosed and her counts really dropped we did watchful waiting. I was concerned about the treatments, side effects and the possibility that I was opening the door to long term or lifelong medication usage. Her counts dropped to less than 5,000 and stayed there for 5-6 months. She was pretty lucky because her symptoms were mostly nuisance symptoms: a lot of bruising, petecchae, very mild nose bleeding, gum bleeding, lips bleeding heavy long periods (somewhat controlled by oral contraceptives). But she also never felt well during the time frame. She had headaches constantly. It was fall and winter and she was frequently sick: headaches, sore throats, stomachaches. This from a kid who was never sick. Her biggest problem was overwhelming fatigue. She got through what she needed to do but had nothing extra to give. Finally she did have some GI bleeding with a count of virtually zero and required rescue treatment. 2 months later when her counts were back under 5 we opted for treatment with Promacta. She was started on 50mg and was on that for approx 4 weeks before she was dropped to 25mg. She did have a decreased appetite and some nausea on the 50 mg dose. After that she has had no side effects at all. And she is a totally different person. I don't know if she was just having a really rough winter picking up everything and that had an effect on her ITP or if her ITP autoimmune response left her susceptible to everything or whether there was no correlation between any of it. But I am glad to see her so much better. I am also glad that we gave both options (treatment vs watchful waiting) a chance so we saw which one worked better for her.

Thanks TerriC14 for sharing your experience. I think you know fully well the feeling of being on the edge. This morning my son had a minor nose bleed and we made him take the tranexamic acid. Thankfully it stopped but he had to skip school again. What dosage is your daughter on and did the doctor see if your child can do without it? or is in remission? Sandi mentioned that some kids go into remission post taking TPO medications.

My daughter started on Promacta 50mgs the end of June. She is currently on 12.5 mg daily. She iresponded well to Promacta and her counts are good. She is being managed (still) by a Pediatric Hematology group and they are slower to taper the dose than some of the adult hematologists from what I am reading on the boards. I can honestly say that I am not overly concerned about her having issues with blood clots at this point either. Her platelet count last week was 157.

I do not believe at this point she is in remission though. A couple of months ago her schedule changed and she got a little careless with taking the medicine. She had a couple of missed doses and a couple of times where she did not follow the dietary restrictions properly and her counts dropped in half. So she started setting the pills out in a " days of the week pillbox" so she could make sure she took it and put it with her phone alarm across the room so she had to get up and not just hit the snooze and forget. And when the dose gets tapered her counts drop. I am fairly certain that if she stopped taking the medicine her counts would drop over a short period time.

My goal for her is the lowest amount of medication which will keep her counts 50 to 75. I really like her doctors and have confidence in them. I am very glad that they really never pushed steroids and were very attentive to treating the person and not the numbers. I am a little worried about her transiton to an adult hematologist, I am afraid that they will take a more aggresive approach with treatment (which may be unfounded, I admit). So I hope we find the correct balance before she needs to move along...

Good luck with your decision. I know that this was the correct one for her, but I am also glad we did not rush into it.

Terri:

It looks like she is doing great! It's fantastic that she was able to get to such a low dose. I hope she can get off of it at one point, but it must feel great to be able to manage it this easily (I know it's not easy, I just mean in terms of dose, stability and effectiveness).

Dear TerriC,

Thanks for sharing. It really helps to hear somebody's perspective on this as there is only 1 case here in our country and so far I haven't heard any results from that yet as he also just started. Experience wise, it is very little here so this community really helps a lot!

I still hope that your daughter gets into remission as I believe that is still a possibility for everyone. Glad that she is able to get good number with low dosage and I agree that if you can get it to as low as possible, that will be best.

All the best!

Sandi - To be honest I do feel that it was pretty easy. She responded very well to Promacta with very few issues from it. I know how lucky she is that there is a treatment that has given her such good results, and the time I have spent on these boards have demonstrated how much easier she has it then so many others. In general her course through ITP has been no where near as difficult as so many others: we found good doctors who we have confidence in, even with extremely low counts she did not have major problems and she has responded well to treatment. I never take that for granted. I am hopeful that she will be off medication at some point but it is great that she is maintained at such low doses.

Wow Terri, you seem so calm about this whole thing while I'm the opposite and panicky all the time especially since my kid has counts less than 10k with petechiae...but after going through this forum (and with inputs like yours/Sandi), it really helps to know you are not alone and there are options out there which can help our kids. It reassures me a bit that our kids can have a normal life. Of course, needless to say, I'm still praying everyday for remission and I'm not giving up HOPE.

aubreyty - I am calm now because we have been handling this longer than you have ! When my daughter was diagnosed her count was 50, and she hovered between 40-50 for 11 months. Just as the doctors were going to lengthen then time between bloodwork from every 3 months to every 6 months since her counts were stable they started to drop very quickly and she went to 7k. I was a wreck but as she stayed in that low number and nothing dramatic happened you adjust. The reality of her world is bloodwork and more doctors visits and things that are not part of my sons world but we just accept that as reality. It is her normal. My heart goes out to some of the other members here who have a much tougher time! You will also as you process all of this and get used to making these decisions!

And I also have a medical background so it was not all unknown territory for me (long time nurse)!

Aubrey - I can assure you that people with ITP have normal lives. Sometimes adjustments have to be made, but they can end up being positive ones. I'm not saying it's always easy because it isn't. But many can plow on through and still have a normal life.

Your son will be a teen soon. I am on the PDSA Scholarship Committee and read the essays that are submitted. Most of the students write essays about the ways that ITP changed their lives, and most of the time, it is a positive change. Some of them say that when they were first diagnosed, they were devastated that they couldn't play a particular sport any more. Instead of feeling sorry for themselves, they discovered that they had another talent, such as music, and it led them in a totally different direction. They end up grateful that discovered a passion that they didn't know they had. Other teens say that having ITP led them in their career choice. Many of them choose the medical field because they want to help others. Very few of the essays focus on devastation or sadness.

As an adult, I was diagnosed after I had my three kids. I was working full time at a stressful job that I had just started, and all of my kids had activities in the evenings. My husband travels for his job so most of the time, it was just me. I decided from the beginning that I was going to keep life as normal as I could for them, so I did. Nothing stopped and I never missed a beat. Was it easy? No. I kept going with counts below 5 and I kept going during treatments. Most of the adults here will all say the same thing. Life goes on.

Terri/Sandi,

Thanks for the encouraging words. It breaks my heart to find the right words when my son asks me when will he get well and be normal again. I try to be positive and still hopeful that he can be normal as the % are high for remission for his age and his gender but of course, there is this small chance of it being a looong-term for him. I always remind myself and him that it could be worse and we should always look at the bright side. He can go to school like any other kid. While he can't continue with Tennis, he can take Robotics. Needless to say the food in our house became healthier as well (less to almost no fastfood), more organic, more veggies, more gluten-free, less cow's milk. My other son now has flu/fever and I'm extra careful of him coming close to my other son for fear of infection which might trigger the worse ITP reaction.

I still get jumpy whenever I get calls from school like 2x last week for his nosebleed. I try to be calm but still worried so I drop everything else. In a way, lucky that I don't have a full-time job else I don't know how to manage it. I think it can be manageable but very hard so kudos to both of you for juggling all these!

Later is our doctor's appointment. Keeping my finger's crossed!

Aubrey - your son IS normal. That's what you need to tell him. He just has low platelets for now. Things will get better. If you end up using a TPO and if he responds well (which he probably will), life will go back to the way it was before ITP. In no time you'll wonder what all the fuss was about. Promacta would probably be the least disruptive choice and it will only be a matter of reminding him to take it every day. I have seen this time and time again.

You'll get there.

Hi Sandi,

Thanks for the advice. We just got back from the doctor and his count is up! 50K!!!! highest without steroids and highest for the past 6 months! We are so happy. There is no talk of Eltrombopag as the count is good. I hope this continues!

Thanks for setting this up and for helping us go through this situation. It helps us (and am sure a LOT of other folks as well) a lot! Knowledge and Helpful experiences do HELP a lot!

Wow, that's great! When is the next count? Maybe he will keep going up - fingers srossed!

50k is a great count! Hopefully it keeps going up!

I don't think there are restrictions with 50 - I think even contact sports require a 30k count (I could be wrong though)!

Hi! Thanks! Next checkup is mid-March. Really praying it holds or it goes up! Interestingly, the Eltrombopag order came today (after 1 month of wait). I think it was a sign not to give it earlier for my son but I'll still get it since apparently, I'm the only one who needs it in the one and only children's hospital in SG... Anyway, will keep you up-to-date and thanks for being with our journey so far!!!

Hi,

Counts went down to 10K mid March and today it is 4K. Doctors say that the IVIG probably only lasted about 1 month which was when we did the 50K. He is having a few bruises and petechiae. Not as bad as when he had the 2k but the doctor is still worried given his numbers are low. He has a bit of phlegm and when he coughed out the phlegm this morning, there was a bit of blood. His doctor prescribed him tranexamic acid and told us to consider IVIG and Eltrombopag again. We will wait and see until Monday for any next steps.

Again, we are on a decision point... to go with Eltrombopag or do a wait and see. On one end, we don't want any unnecessary side effects..on the other end, we don't want another trip in the emergency for him.

What a tough decision...

It is a tough decision. Sorry to hear that his counts went down again. Whatever you choose to do will be the right thing. Keep us updated.