experimental drug for polycythemia

Thanks for this, I have not heard of this drug before.

I suppose as it is a drug trial there could be a number of the possibly outcomes which might render it unsuitable for you or for anyone. There may be a reduction in some of the unpleasant side effects of PRV, whilst at the same time adding some which tho' not unpleasant to you might be derogatory to your better health. We would probably have to trust that if it was not continued that the reasons were for our better health and not for, say, financial reasons.

I probably wouldn't offer myself to the trial because of the bone marrow samples.

I believe I have had 3 taken in the past and the last was taken without any general anaesthetic! It was extremely uncomfortable and the thought of going thru' even one more of those would put me off.

Nigel

 

Hi clem. If  you have Polycythemia Rubra VEra, that means you weren't positive for the jak2 gene mutation. If you were tested and found positive for jak2 gene mutation, then you have polycythemia Vera. Is that right? I was positive, and diagnosed with Polycythemia Vera. Is that what you have? Anyway, if you do the experimental drug trial, and it works well for you, then it helps you to know if you want to continue on it when it becomes available on the market. I'm sure you have your reasons for wanting to participate in the trial. I wish you the best.

harrishill

 

Ruxolitinib has been approved in the US. It is sold in some other countries as well, although I'm not sure which, under the trade names Jakavi and Jakafi by Novartis and Incyte Pharmas. It is a JAK inhibitor. It was first approved as a treatment for myelofibrosis. Most probably the reason they would do a bone marrow test is to see what stage the PV is at: the progressed stage could involve secondary myelofibrosis. I have read that it has positively helped many patients in the US. It is also possible that they want to test ruxolitinib at earlier stages of PV. Another possibility is that they want to study how long the effects last after the last dose, whether they are reversible or not, in short, yes, you will be a guinea pig. I am not aware of how strict the guidelines in the UK are, but sooner or later Ruxolitinib will be on the market unless it gives some very different/undesired results in the UK trial population. Doctors generally don't put you on Ruxolitinib unless other drugs don't help because it is still in the earlier phases and long term effects are not known. However, the drug would possibly be very expensive early on, which will again limit the number of users. I know it is very expensive in India. But it sure is the new wonder drug for PV/myelofibrosis for many if not all in the US.

Much of what you say Clem I would concur with.  I was taking Hydroxycarbamide for many years and became intolerant of it.  Had some painful issues with it and the haematologist took me off the hydroxycsrbamide, and onto Ruxolitinib which I still take daily.  I was on maximum recommended dosage of Hydroxycarbamide which seemed to cause many of my problems.  I was part of the research trial though was not on the trial drug but the placebo which could only be Hydroxycarbamide.  This was termed 'best available treatment' for nothing else was available to treat PV.  The consultant had to obtain hospital authorisation for me to receive the new drug.  It was explained to me that I would need to take it forever as the improvement it gave would be reversed if it was stopped.  The spleen could become enlarged with all that entailed.  The UK trial is monitored by 2 large London Hospitas with the assistance of local hospitals elsewhere in the country.  Your experience initially is similar to mine .  I had no problem with Bone marrow testing for i received a painkilling injection first.  I have made enquiries about US programme and it is very favourable.  I am at the hospital tomorrow for the latest check-up.  The haematology team have been splendid throughout.

Clem,

I assume you are referring to the needle bone marrow biopsy, where a needle is inserted into the bone (maybe the hip bone?) and a quantity of marrow is drawn off for testing.

I have had this done three times and the first two I was given an anesthetic which meant I had no memory of what was going. I was told that it supressed memory so I was able to cooperate at the moment, but retained no memory of it. The last time I was just given a local anesthetic, which unfortunately didn't mask the strong effect I felt all down my leg!

I would suggest you try asking your consultant that if you have to undergo a bone marrow sample that they give you the former anesthetic as I truly felt nothing adverse. I am also fearful of needles.

Nigel

 

Clem,

Believe me I sympathise as I too fear needles. Once when I had a long stay in hospital I was given the chance to take a weekend at home if I would do my own injections. Of course I couldn't and so I had to stay in.

Have you spoken with your consultant about your fear? You are not the only one and I'm sure they have dealt with others. If they can't persuade you to endure  just the first needle (which will mask any subsequent). Maybe they can offer other anesthetics such as by gas or tablet. I would have thought the effort to overcome this brief hurdle will save you a lifetime of discomfort.

Good luck

Nigel