Long-term promacta thoughts

Hi folks - really appreciate all the insight and interesting discussions on these boards. My doc is about to start me on Promacta and I'm of course concerned about the long-term effects. I've read all the warning labels but would be interested in personal experience too. Anyone out there who's been on Promacta for a while, like more than a few years? Any problems with side-effects that worsened? Liver issues? Bone marrow issues? Cataracts? Any tips on what to look for (other than the prerequisite blood and liver tests)?

For context -- 56 years old, male, diagnosed ITP Nov. 2014 (plus AIHA; Evans Syndrome) March 2016. Failed all the usual first-line treatments. Two rounds of Rituxan 2015 and 2016. Splenectomy Aug 2016. Been on and off prednisone (up to 60mg/day) for the last year and a half. Pred mostly works, but once I taper the platelets crash.

Thanks!

Al

Hi Al,

I share your concerns. I will be taking my first promata pill tonight after a glorious 3-year remission from N-plate. My current count in under 10.

I thought I would give promacta a try for the convenience of the pill form. When I went to pick it up, the pharmacist gave me a very serious talk about the side effects. The one that alarmed me the most was the high incidence of Myelofibrosis... I am hesitant to quote the exact percentage she cited, in case it was wrong, but suffice to say it was high.

I must say I am a little scared and second-guessing my treatment decision. She told me to speak to my dr, which I will, but like you I am comforted to hear from the real-life experts on this forum.

www.ncbi.nlm.nih.gov/pmc/articles/PMC4008112/

this may be of use

Caro, it is interesting to note that the counts of both you and Jay fall very, very slowly - over a matter of weeks. Incredible really.

Are you starting on 25 or 50mg dose? When you took NPlate before, did you react strongly to the drug and raise your counts a lot with small/weak doses?

Cheers to another remission !

Hello everyone.

Al, I realized that in my panic last night I may have hijacked your post. I really meant to bump it, as I too am interested in hearing the experience of promacta users and the frequency of issues and complications that go along with what is really a relatively new drug in terms of long-term effects. (I sometimes feel like we are guinea pig pioneers!!

Hal, I will start a separate post to document my promacta journey as others have so generously done. We learn a lot from each other about this mysterious condition with no simple solutions! (I am so impressed by how versed everyone is in the latest medical literature!!)

Thank you all!!

My answer to both of you:
We've been following Promacta users here since it's inception. It is a commonly used drug at this point. I have only seen a few cases of bone marrow fibrosis which were not serious and did reverse when stopping the drug. Some people have been able to get off Promacta in time and hold stable, safe counts.

I cannot speak as to the long-term side effects since the drug has not been around long enough. No one knows what could happen 20 years down the line. All I can say is that every drug used for ITP has long-term risks. The best thing to do is to use the lowest possible dose to maintain safe counts and hope that you can get off of it in time. It has a good track record so far and seems to give people the ability to return to a normal life once they get stable.

Hi Al,
I'm not an expert but in my experience most people quit the drug if they are experiencing side effects and the issues resolve. I have quit Promacta 4 times because of side effects. But my side effects were very mild, nothing like liver/bone marrow issues. I just didn't feel overall great on it- headachey, brain fog, sort of spaced out. My thinking cleared right up within 24 hrs of quitting. And after quitting, my counts did not crash, they drifted down over a period of a couple weeks.

I have heard of a couple people who have had strokes on the TPOs so I think its very important to follow the manufacturer's recommendation and keep the counts around 50K and not try to normalize the counts. You'd be surprised how many doctors will have their patients maintain counts of over 100K. Also counts can be erratic and can accidentally shoot up very high. Thats something to watch for. Dr Drew Provan, an ITP expert in the UK likes for his patients to have their counts between 35K -50K when on the TPOs.

Also when I was beginning Promacta, I always had some side effects for the first week but those discomforts usually went away. Hope it goes well for you!

Promacta did not work for me, but nplate worked great.
As Posey said before do not go for normalization.

Currently my hema will only give me a nplate shot below 50, works great for me because it costs $377 out of pocket for every shot.

Thanks, everyone, for sharing your experience. I appreciate hearing your thoughts.

Along similar lines, has anyone had any experience with long-term, low-dose (5-10mg/day) prednisone? Is there any research on the long-term risks/side-effects of being on pred forever?

Thx.

I've been on Prednisone for 12 years using between 5 to 15 mg's daily. I don't use it for ITP but I can tell you that it's not a good idea.

These might be of interest alevitt
academic.oup.com/ndt/article/18/5/861/1833137/Steroid-induced-osteoporosis-how-can-it-be-avoided

academic.oup.com/jcem/article-lookup/doi/10.1210/jc.2015-1218

Thanks Sandi, that's what I needed to hear.

You are an angel for moderating this forum!