Ruxolitinib trial . I'm so unsure.

[maestromaverick]

My haemotologist has asked me to consider being put forward for a trial of ruxolitinib . I'm very unsure about this and quite scared to be honest . Has anyone else been on a trial of this drug . Any help would be appreciated . I didn't think NICE had sanctioned the drug . I have PRV and currently on hydroxycarbamide and aspirin . Help please.

[fleetpete]

Hi, I'm not sure I can be of much help...I was asked if I would consider going on the trial earlier this.year & have heard no more. I was under the impression that all was ok with the drug??? Maybe other members have more up to date info? In the meantime, be strong & try not to be scared, it is hard I know...I have the same medication & have PRV like you, and consider myself fortunate to have loads of support at home from my wife & family...and online. Fleetpete x

[jane13]

Hi, sorry i don't know anything about this particular drug but i think NICE is a side issue as their remit is assessing for value for money for the NHS, not drug safety. I think I would be asking a) is it licensed for use and in what situations (eg it may be licensed for treating a different disease, or in the US not UK) b) what are the side effects c) what are the potential benefits for you? good luck

[beetle]

There is masses of information about Ruxolitinib on the net. It is a Jak2 inhibitor. Google it and read all you can - the good, the bad, and the ugly. It has already been through trials for MF and it seems that there are people on it doing very well and others that can't tolerate it - much the same as any other drug, particularly new ones I guess. It is the only real hope for MF at the moment but because of the NICE preliminary decision I am having difficulty getting it - as I expect people with PV & ET will have after the trials. I seem to have missed the trials for MF but as my ET has progressed I don't qualify for trials via that route either. Good luck with your decision. There are good sites that detail the benefits and snags of drug trials in general. It would be worth checking them out too to help you with your decision.

[JediReject]

Hi MaestroM - I was due to give this new 'wonder' drug a go for my MF and was granted funding via Cancer Drug Fund as you correctly say NICE have not sanctioned it for NHS use on cost v benefit grounds thus far as I understand it. It is well worth going on the NICE.org site and reading some of the PDF submission docs from people that are on the drug and some of the general info'. You will find that some people's symptoms worsen before they start to see the benefit as their body adjusts to the drug and may need transfusion due to it causing or worsening anaemia early on.

I was concerned about switching to it also although my HU wasn't working quite as effectively and it seemed a logical progression to try the Rux. But my counts have been reasonable since the funding was agreed so I opted to stay on the HU which I tolerate very well and leave the Rux to a later date. Though if NICE don't sanction it eventually there is a concern that the Cancer Drug Funding may not be available next year.

So I would advise you to gather your facts and if you're doing well on HU and aspirin ask why your Hem thinks you are a good candidate for the trial especially in light of the fact that extensive trialling has already taken place for MF and in spite of the evidence the drug remains unobtainable. One of it's main advantages for MF is that it shrinks the enlarged spleen by up to 50 percent and this wasnt enough for NICE. Do PRV / ET patients suffer spleen enlargement.

Its a tough call but if you decide to give it a go on behalf of fellow sufferers I wish you the very best of luck with it and please let us know how it goes. Cheers

PS You could be one of the ones given the placebo as they use some of these as a comparator or control group

[eire]

I have MF and have been on the trial since October 2012. I take 15mg of Ruxolitnib twice a day and 25mg of Panobinistat every other week wed/fri and sund. The Panobinstat is quite severe and being near a bathroom is very comforting! My spleen has shrunk more than 50% and I feel better since I've been on the trial. It takes time to get used to these drugs in your system. The main downfall for me are the weekly transfusions but I was taken off all drugs over 2 weeks ago to see if this would improve but still need the trans. I don't feel as well off the drugs as I do on them. I guess we are all different but any-one can withdraw from this medication at any time so my advice would be to give it a try but be aware you do need to give it time. I'm just waiting to hear back from USA on the next course of action for me.

They are just starting another 20 people on this trial which I think is very positive. Good luck with your decision and you're not on your own!!!

[Twinkly in reply to eire]

Can I ask are you still on the drug ruxolitinib ,I'm just new to it myself! , x

[maestromaverick]

My heartfelt thanks to all of you for your kindness , sincerety and honesty . I will take up all of your suggestions . This forum is a godsend and when you live alone as I do it makes such a difference in dealing with this illness . My blood counts are faily steady at the moment but I do not tolerate the hydroxy very well and have had 21 venesections in the last three years resulting in smaller cells . On any higher dose of hydroxy than the 1x500mg daily I become very poorly even becoming pancytaemic once resulting in a blood transfusion . I understand these are the reasons that my haemo wants me to go on the trial . I'm just an awkward patient . Once again god bless you all and stay as well as possible . Friendship and care goes both ways so I hope I can be of some help to you all too . I will let you know my decision . Xxx

[JediReject]

Good luck whatever you decide to do but like you point up maybe your Hem feels it would be worthwhile givng it a go. . From what you say of your HU tolerance you surely couldn't be any worse off. But the fear of the unknown is often what prevents us all from taking a chance at some point in our lives whether a 'biggie' like changing jobs or re-locating, or something llke even trying a vindaloo.

There's no shame in taking your time to decide and without a partner there to help and support you it's all the more difficult I guess. As long as you don't feel 'pushed' or coerced into it and you own your decision then it's a good one i'd say.

There endeth the lesson for today - sorry if I seem to be preaching.

I'm going to try and get my petrol mower fired up as it's been out all Winter and the sun is shining and the grass needs a first cut. Have a good day. Cheers

[Marie95]

Hi Maestromaverick, I am in the same position as yourself, only I have ET, I have been offered a place on the trial, for Ruxolitnib versus best available therapy,and like you my head is all over the place, as i don't tolerate HU well, Do you have an enlarged spleen? please message me if you want to disscuss further, and good luck

[maestromaverick in reply to Marie95]

Hi Marie95 , I don't have an enlarged spleen at the moment and don't really want to experience one though I know it will probably come in time . Have done so much research on the net and to be honest am no further forward . My next appointment as long as my blood behaves isn't till 18th of july and that's when my haem is going to discuss it with me . Its so difficult isn't it . How are you feeling about it . Take care and look forward to hearing from you if you would like to talk further.

[Marie95 in reply to maestromaverick]

Hi again, I have just re read your orginal post, and can quite understand your feelings, especially with regard to HU, You are not an awkward patient, you are an informed one, I believe Ruxolitinib can reduce an enlarged spleen (neither of us have , as yet) and according to the ASH summary report , quite advantageous for MF, in many areas, but nobody yet knows its response in ET/PV. Having been on INF alpha, and still having to deal with some of the issues it had caused, after stopping it, I am very cautious, and can fully un derstand why you are aswell, Have you considered Anagrelide?

[maestromaverick in reply to Marie95]

My haem believes HU is a more effective way forward and because I can only tolerate a low dose says Anagrelide would not suit me either so I'm stuck as I am . INF alpha has never been mentioned .

[Marie95 in reply to maestromaverick]

This is such a difficult decsion, and in a way we are lucky to be offered the choice, dont be put of INF alpha by my experience, people on the forum with PV have had some very good results, & especially with peg INF once the PCT approve it, anyhow best of luck for what you & you Hem decide, do let me know how you get on, I have to decide by end of Month

[maestromaverick]

Good luck and please let me know what you decide to do . Take care and most of stay well. My thoughts are with you .

[Twinkly]

I'm so curious did you go ahead and take ruxolitinib ,I have only recently started taking it ,but already I feel so much better ,more energy ,my blood counts are stable ,how are you??

[maestromaverick in reply to Twinkly]

Hi Twinkle , yes I did go ahead and now 15 weeks in . Some things feel better is concentration , rash not so bad and not feeling the cold so much but the headaches and dizziness are bad . Blood counts are good and tiredness about the same . As I still don't know what I'm taking don't know what's happening but one thing is for sure I've put on weight which for me is good as was down to 49kg . My consultant does believe I am on Ruxolitinib but obviously can't be sure . Hope things continue to go well for you .

[Twinkly in reply to maestromaverick]

It's so good for me to meet you ,only 4 were given the Majic trial at our clinic ,2 were having problems I lady was 82 and a writer ultra intelligent ,she developed a tint growth on her lymph gland so was withdrawn ,iv been amazing ,very well,no sweats even the pain is much less,eat tiny portions,every 3 hours ,healthy ,fruit is essential walk 20 minutes ,rest use my brain ,ha,ha,some headaches ,but just a paracetamol sorts that out,if we work with the drug ,I'm sure it will be good for many .once we get funding..keep in touch ,twinkly..x

[eire in reply to Twinkly]

Yes I've been on the trial now for 13 months but I take Rux and a drug called Panobinistat. They're looking to see the benefits of Rux only versus the 2 drugs together. The Panobinistat is quite severe but I'm learning to handle it very well!!! I need lots of blood transfusions but think that is more to do with the Pan -I've recently started taking Wheat Grass which is supposed to be very good for your blood and my transfusions are not so often. Can't see it being because of the trial as I was on that for a year before I took the Grass so I'll keep going with it even though it's disgusting!!!! On my last visit I suggested we should all be given stats on these trials just waiting to see if that has been acted on. I have MF and was diagnosed 4 and a half years ago. I wish all of you the very best of luck.

[Twinkly in reply to eire]

I also hate the taste but take wheat grass. Queen of greens .it helps blood counts I'm sure ,,and a PH diet too ,,good luck Eire you are doing well .