Promacta (eltrobopag) Not Working - Please help

I'm sorry, I'm new to this sight and posted under different categories since I was not sure where to go.
My son is 17 and this is his 3rd diagnosis with ITP. In 2013 he responded well to IVIG and steroids. In 2015 it took a little longer but he again responded well to IVIG and steroids. His most recent diagnosis was Sept 2017. His #'s are often in the single digits. He is not responding to IVIG or steroids so we started Promacta (eltrombopag) 50mg. He went up to 46K platelets but then declined to a platelet count of 5 in 1 week. We changed him to the max does of 75mg and his platelets spiked to 96, then 87, then 46 and now 7 as of yesterday. These #'s are just over 1 week so he declines rapidly. So, it appears he's not responding to Promacta which has a 95% success rate. During the past 6 months the physicians have done tons of testing. His IG levels are extremely low so they are thinking has CVID (commom variable immunodeficiancy). We have one lab to get back but they are thinking of starting weekly home infusions of IVIG for his IG levels. Although IVIG did not work for his ITP they are confident it will work for CVID. I'm wondering if anyone out there has CVID and ITP and if so, once your IG levels increased, did ITP go away. He is 17 and is an avid soccer player and skier. Needless to say, with platelets in the single digits, he cannot do anything. He's had bouts of spontaneous bleeding this time which has never happened in past ITP episodes. It seems as if this round of ITP is much worse than it has ever been and he's just not responding. Also, did anyone else try Promacta? Did you increase and fall again? Did it eventually work? I'm stressed and sad that I have a child I cannot fix. Any and all suggestions welcome. He did have a bone marrow biopsy upon initial diagnosis in 2013 and that seemed fine. Did anyone else not respond to Promacta but have results with N Plate?
Thanks for helping

yes, in my case nplate works the best.
I also first tried promacta thinking that nplate weekly injection is going to be too time consuming.
Promacta did not work.
I have been pleasantly surprised with nplate.
If you have formulaic approach to dosage (Dose only when below 40k) then you need to go once in 3 weeks to dose.
My last dose was on Dec 16th, so a good 9 weeks of platlets above 40.

This I think is result of Flu and other low level infection which keep the platelets up.

Thank you so much, you give me hope. I'll see if we can switch to N Plate. And you think your counts go up whenyou're sick, with the flu and infections? I wondered if this happened only because my son is testing for CVID and they gave him a pneumo vaccine. Right then is when he spiked to 97. I thought it was a result of the promacta but once he started he started dropping again I had a feeling his spike in platelets was due to that vaccine or something. Thank you again.

I think it's too soon to give up on Promacta. It can take a few weeks or months to get stable on it. Maybe the vaccine caused the drop? I know it's frustrating, but you could give Promacta a few more weeks before jumping into something else. N-Plate usually causes wild swings in counts from one week to the next and it can take months to get stable on that one. Hang in there.....treatments take time.

Is your son following the dietary guidelines for Promacta ?

Thank you so much. And yes, I never thought about that but maybe vaccine caused drop and now he'll go up. Thanks for your support

Yes. No Promacta for 2 hours prior to dairy or 4 hours after dairy. Since he's 17, he drinks a lot of milk and love pizza. So, we've been doing dinner at 5pm, ending by 5:30 and he will not take promacta until at least 9:30pm, waiting the solid 4 hours.. Usually before bed. He cannot take before school due to eating cereal with milk and I cannot send med to school with him as that's forbidden, so prior to bed seems to work best. If you have any suggestions, I'm all for it....

Yes. No Promacta for 2 hours prior to dairy or 4 hours after dairy. Since he's 17, he drinks a lot of milk and love pizza. So, we've been doing dinner at 5pm, ending by 5:30 and he will not take promacta until at least 9:30pm, waiting the solid 4 hours.. Usually before bed. He cannot take before school due to eating cereal with milk and I cannot send med to school with him as that's forbidden, so prior to bed seems to work best. If you have any suggestions, I'm all for it....

jkk, you can do searches on PDSA user comments by first clicking on 'search' item on the blue strip at the top. Then enter something like 'CVID' in the keyword box. Check other options available, and click 'search' purple button near the bottom of the page. Here is an example query result.

I've read through a lot of old threads on here and taken notes along the way. I've only got a couple of entries for those with CVID. The data is pretty thin. If you find someone with a similar situation as your son, let me know so I can put it in my notes.

There is 'GhostRider' who responded to steroids, takes IVIG for CVID, and needed 25mg of Promacta. Eventually got a splenectomy, which was reported as working. Don't have any numbers in my notes on how well he responded to IVIG.

There is 'DelnStyle' who responded to steroids but don't know much else.

If you don't mind, can I ask you about his IVIG response? Did he respond to it for a day or two, or, was it more like a week before he dropped down to his baseline count? Did they give him both steroids and IVIG simultaneously and that's when his counts didn't respond? Was it just a single bottle of IVIG on one day, and that was it, or ?

His response to Promacta does seem odd. Non responders normally have no response from the start. By any chance, did he start out taking Promacta overlapping his steroid and IVIG treatments? That it takes all 3 to get counts up?

It took me a long time to have a decent effect from it so I would try give it more time.

Hal9000, your info is so helpful. And to answer your questions - he used to get IVIG along with salumedrol steroids. He'd go from like 5 to 7 as far as platelets and that we testing 12 hours after the infusion ended.. They typically only ever did 1 infusion but twice he received two within 2 days and he went from like 5 to 10, again 12 hours following infusion and then in a day or 2 he'd be back down to less than 1. No significant response so they are no longer willing to do either. His last IVIG/steroid treatment was Sept 2017 and we did not start Promacta until January 2018, so no overlap with IVIG or steroid. Out local children's hospital as well as the Boston Children's hospital (2nd opinion) seem baffled as to why Promacta is not working. We are thinking it's because his IG levels are rock bottom. We will start IVIG for CVID (low IG levels) and we are hoping that once we boost his immune system the platelets will go up. I just don't know. It's a vicious cycle and it's terrible for a very athletic child. Thank you for your support and info.

jkk2229 wrote: I'm sorry, I'm new to this sight and posted under different categories since I was not sure where to go.
My son is 17 and this is his 3rd diagnosis with ITP. In 2013 he responded well to IVIG and steroids. In 2015 it took a little longer but he again responded well to IVIG and steroids. His most recent diagnosis was Sept 2017. His #'s are often in the single digits. He is not responding to IVIG or steroids so we started Promacta (eltrombopag) 50mg. He went up to 46K platelets but then declined to a platelet count of 5 in 1 week. We changed him to the max does of 75mg and his platelets spiked to 96, then 87, then 46 and now 7 as of yesterday. These #'s are just over 1 week so he declines rapidly. So, it appears he's not responding to Promacta which has a 95% success rate. During the past 6 months the physicians have done tons of testing. His IG levels are extremely low so they are thinking has CVID (commom variable immunodeficiancy). We have one lab to get back but they are thinking of starting weekly home infusions of IVIG for his IG levels. Although IVIG did not work for his ITP they are confident it will work for CVID. I'm wondering if anyone out there has CVID and ITP and if so, once your IG levels increased, did ITP go away. He is 17 and is an avid soccer player and skier. Needless to say, with platelets in the single digits, he cannot do anything. He's had bouts of spontaneous bleeding this time which has never happened in past ITP episodes. It seems as if this round of ITP is much worse than it has ever been and he's just not responding. Also, did anyone else try Promacta? Did you increase and fall again? Did it eventually work? I'm stressed and sad that I have a child I cannot fix. Any and all suggestions welcome. He did have a bone marrow biopsy upon initial diagnosis in 2013 and that seemed fine. Did anyone else not respond to Promacta but have results with N Plate?
Thanks for helping

Welcome to the group and I hope that you find lots of helpful information as you navigate the ITP waters with your son. I grew up with ITP (was diagnosed around age 8 and am now 42), so I can relate to some of the challenges of managing the teenage years with ITP. I was also a soccer player. On and off depending on counts, and I probably did more of that than I should have, but my dad was the coach! I don't know anything about CVID and it sounds like that condition might be affecting the ITP treatments you are trying. Good luck getting a combination of treatments that works well in treating both conditions.

Regarding the Promacta, this idea that it is 95% effective sounds VERY overstated (I've never heard anything close to that). There are lots of folks here for whom Promacta doesn't work, so don't let this idea that it works for almost everyone (it definitely doesn't!) get you down. That said, I have been treating with Promacta for the last year and a half or so with good results. If I recall correctly, I originally tried Promacta for a month without any upward trend in counts. I started with a dosage of 50mg, and then increased to 75mg after a month of no significant increase. After the dosage increase, I did see some improvement in my counts over the next few weeks. It is very surprising that you had these instantaneous spikes and crashes after taking the medication. It could be something else causing the fluctuation in the numbers (maybe some sort of a virus or something related to the other condition?). I think it would make sense to try the Promacta for well over a month (and increase the dosage if there isn't any improvement with the standard 50mg dosage in the beginning) to see if you can get some kind of a response. I am glad I waited and didn't throw in the towel with that after only a few weeks because I had more of a lag time in seeing the improvement.

Regarding the NPlate question, I'm sure others have replied, but there are several people for whom that works when Promacta doesn't. Good luck in getting some improvement in his numbers. Hang in there!

jkk, thanks for the info.

I scanned through the CVID comments. Looks like all those with CVID reported good steroid and IVIG responses (row 1). Though your son started out with those good responses, he seems to have lost both. From what I've seen around here, losing responses like that is not uncommon at all. It may be rare in the CVID world but it is not in the ITP world.

A now apparent row 2 response, no steroid or IVIG response, is an odd one. It's like no drug except Promacta or Nplate will bring counts up. But once counts get out of the gutter things slowly get better - often much better. Also, a bit of steroids seems to speed the process up. I'm hoping (25-50mg) Promacta or Nplate are going to do the trick for him. Just as a lot of folks before him here have done well in this same pattern.

Hope this helps.

Well, we have been on the max dose of Promacta (75mg) since 1/24/18. Counts will not stabilize. He went from less than 5 to 147 in 4 days and then 6 days later back to less than 5. He is currently covered in patechaie and blood blisters. Our next lab is Monday. So in a matter of 2 weeks he goes from less than 5 to 97 to less than 5 to 147 back down to less than 5. He's only staying at a high count for a few days, if that. The hematologist said to give it 4 more weeks UGH. I cannot let him do anything when he's so low like this. If anyone else had severe flucuation like this can you tell me about it and let me know if you ever reached a higher point and stayed there? The hematologist said he's seeing a 2 week pattern and he's hoping we'll go up and stay up but he's been on the high dose of Promacta for 2 months now and it's not happening yet. Any help or info is greatly appreciated.
Thank You

Oh my jkk. That is an extreme count response! Stunning, really. What seems to have worked with others is a small amount of steroids. For whatever reason, this seems to help.

On a related note, 'Cyclic Thrombocytopenia', characterized by repeating periods, is rare and is normally treated with Danazol.
"Danazol therapy for cyclic thrombocytopenia"
miami.pure.elsevier.com/en/publications/danazol-therapy-for-cyclic-thrombocytopenia
I, myself, am on 200mg of Danazol but not for cyclic thrombocytopenia. I am no it for regular ITP.