Had my once-every-four months appointment with my MPN specialist. He is quite happy with the seamless switch to Bes fromm Peg (as am I). All bloods came back WNL (still amazes me after a decade of high PLT readings to see PLT at 346)...no Liver or thyroid issues and also importantly the kidney readings came back in line (so thinking that the focus on hydration likely helped). We had been doing bloods every 2 weeks (a week after the Bes shot) since the switch, but he now feels fine going back to doing them once every four months in line with our appointments.
For an overall boring appointment (how I like them) we did discuss two interesting points perhaps of wider interest. One, he was surprised when I told him that the health fund had approved me for Bes through June 2027 (he had expected until end of year). His feeling is that given the initial positive results on the Bes for ET trial (SURPASS-ET) and the expected results later this year for the EXCEED-ET trial that PharmaEssentia will be aggressive in pushing the FDA for approval of Bes as asecond line med for ET. If this happens, he fully expects Pharma&, makers of Peg, to perhaps not ramp Peg production back up as Bes would then be approved for ET and PV and likely have easier market access. Again, this is only his speculation but I thought it was an interesting point.
The other point we discussed was the best level of Bes. I am a big believer in trying to find the minimal amount of med necessary to be effective. I am currently on 70mcg every two weeks (which corresponded to my bi-weekly Peg dosage of 90 mcg). It seems to be doing the job as far as blood results go (and with no side effects). I questioned as to whether it would also be enough to hopefully achieve a molecular response and induce a reduction in Jak-2 allele burden. He thought it was a good question...he said the hard evidence for a direct correlation between blood results and Jak-2 he couldn't say 100%, but that they should "move together". The only way to make sure is to re-test for Jak-2. While it would be possible to do via a blood test, I would prefer re-testing via BMB as that is how I first tested. So he agreed that we could do a BMB this summer, which would be 3 years after starting interferons, to see if there have been changes in the bone marrow and with the AB (we will also test AB via blood at the same time to have a base line going forward and to be able to compare the levels via BMB and blood). Hoping that the BMB will show some improvement overall (especially as I was likely on cusp of pre-fibrotic PMF vs ET at initial diagnosis). If not, then it will be interesting to think about perhaps an increase in the interferon to see if that would move the needle..but we will cross that bridge when and if we need to.
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Solyesh
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it will be interesting to see how AB from bloods compare to AB from BMB, also 3 years isn’t that long for interferons to achieve best results (some take 2 years just to control Hct), so if you don’t see what your hoping for in the BMB it might just need more time.
Yes - slow and steady is my mantra. Hoping that given how relatively high my AB was for ET that we might see some improvement...but also curious as to perhaps there is a threshold level of interferon needed (by individual) for hematological response vs molecular response....
we also do not have Bes for ET here either but it is not approved yet for ET anywhere in the world to my knowledge (hence the two trials I mentioned in the US)..as my MPN specialist reminded the review panel when we went for approval to temporarily use Bes - Peg is also not approved for ET - it is an off label use for secondary treatment for those who might be intolerant to Hydroxyurea - so if Peg is an accepted (but not yet fully approved off label secondary treatment - why could not Bes be as well given the Peg shortage)...
Thanks for sharing your pos news Solyesh, I am curious where you are in the world, just with regard to Bes - as like you I am doing so well 4 mnths in with my Bes journey (its the first meds I have taken for PV despite being diagnosed 7 years ago) and hoping that even when Peg is more freely avail, I can stick with this meds that is suiting me. Like you I was diag aged 49, and also take the small amount of 70 mcg per 2 wks.
I like also that your specialist feels that there is chance for the blood counts to be also mirrored in the AB red- it will be this Oct/Nov time before I find out - ie a year after starting the meds.
Great to see your plt doing so well, I am about 590 atm (on my last appt) but its coming down from around 1600 as my max so a very encouraging and steady decline, similarly for whites, and also HCT well controlled now at around .41 which is good for a female of slim build.
I wish you well on the journey and thank you this news has lifted me (ie that the counts dec- may also be an indicator that the AB is also getting more into reasonable levels too, fingers crossed) Best wishes Sarah
Sarah - thanks for your note. I am in Israel where medicines are approved by the national health committee. It took some time but we got Bes approved (at least until June 2027) for use for ET. So good that you have responded so well to the Bes. I had been on interferons for a little over two years when I made the switch to Bes. Given the younger side of things at diagnosis, was really interested in the potential disease modifying potential of the interferons...my plt had been at 1,400 before I started treatment (in fact it was a spike from 900 to 1,400 and some bleeding at the gums which led to being advised to use meds (I had been on watch and wait)...
Sounds like you are on the right path with a decline in plt and white bloods as well and HCT well controlled! Wishing that the good trend continues and good to see at the same level of Bes I am on!!
I know we are BES buddies - even across the miles! I am waving at you and as you say, it's essential we get to control our quirky condition as best we can......... as we still have lots of miles left in us
All best wishes on your journey and it's lovely to be in touch with you. Have a great weekend. Sarah
Glad you've experienced a smooth transition from Peg to Bes, as medication changes can be unsettling. Also a relief to see Bes approval through '27, although the often changing medical and pharm landscape can surprise us. I've twice had to re-gain approval for Bes in the middle of an already approved time period, although the particulars of my situation differ as I'm in the USA and have PV.
I agree with your belief that the minimal dose of medication needed to be effective would be best, and should run the least risk of negative side effects and adverse events. With Bes, it's easy to quantify what is effective to control blood counts by following lab result trends. As your MPN specialist touched on, it's more difficult to assess the minimal Bes dose needed to induce a durable molecular response for JAK2.
I'm eager to see hard evidence that supports that the two results "move together", at which point I could immediately begin lowering my own Bes dose to the lowest level that maintained CHR. The significant symptoms I endure could be a direct result of my current Bes dose of 400mcg 1X monthly, after 2.5 years total use at the recommended titration schedule. My dose was only lowered from 500mcg to 400mcg because of adverse events and side effects possible linked to Bes. My MPN specialist has been reluctant to reduce below this dose, and believes that the maximum dose tolerable may promote molecular response.
I recently had a lengthy discussion with an RX nurse at my dispensing pharmacy discussing the possibility of Bes inducing dizziness, cognitive impairment, fatigue, arthralgia, and dyspnea. I was informed that although Bes typically improves many PV symptoms when blood counts are controlled, each of the symptoms I've listed may be associated with Bes itself. Fatigue was reported by 47% of patients in the PEGINVERA study. Severe fatigue itself has been associated with the potential to cause dyspnea, dizziness, cognitive impairment, and malaise.
Reviewing the experiences of other Bes users on this forum, it seems many of us experience these symptoms and others even with CHR. If proof is obtained that molecular response correlates with hematologic response, Bes guidelines could be modified to ensure a minimal dose is used that will control blood counts, without degrading long term molecular response. This would improve the quality of life for a significant population of MPN patients without reducing length of life.
This battle of Quality of Life vs Quantity of Life may factor in if you decide to "move the needle" in search of molecular response. My sincere hope is that your summer BMB shows improvement in AB and other indicators, and your future appointments remain boring.
Could not agree more. I sat with my MPN doctor a little after my initial diagnosis and laid out my long term treatment goals. I am all for being as aggressive as we need to be until it starts to severely impact quality of life. Quality is my ultimate goal (although of course I hope it comes with Quantity as well). I know there are multi-year studies out there that indicate at the recommended trial doses (the higher Bes doses) that it has been quite effective in inducing both CHR and molecular response. To your point and my question to my MPN specialist, it would be great to see studies (longer term) that showed that connection at lower doses.
I've posted on various reports finding the correlation of AB to CHR. The Bes trials found and stated it as I recall, and conversely no dose correlation. I've posted long ago on this. A recent one I posted on (I don't have it handy) stated this relationship as established thinking. But truly definitive evidence might never happen since there are always confounding variables. One example is non drivers which may not decrease even as Jak2 does.
My opinion is your Drs idea is best, set the min dose to your response on CHR and look for the AB response.
On Marrow vs blood, some reports discuss "hot spots" where the mutation is higher in some marrow locations and a BMB can miss these. So it's possible blood will give a more homogenized reading (this 2nd thought is not in any reports I know of). Maybe the hot spot concept is why BMB can sometimes give quite different results, mine were.
Taking the above idea further, getting a ""not detected" result in blood might still allow that some marrow hot spots remain. So a period of non-detected might clear out the marrow hot spots. This a similar to some viruses. There are no studies of this I know of since reducing AB is still a new ability in MPNs. I'm interested since my AB was down to 3% last Dec. But I may have to quit Rux before I find out.
Thank you for the reminders on past posts/studies - I will look them up and discuss with my MPN specialist. Agreed that we are on the right track of setting dose to min to effect CHR and then testing AB to see if it has also moved the needle. Interesting point also on the non-driver mutations as well. Hopefully as more data is collected overall it will help us make even more informed decisions.
It is hopeful to see that the Rux continued the reduction in your AB that you started with the interferons.
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