Hello, I am very grateful for this forum. By reading the many posts I have gained much better insight into the disease. I was diagnosed with PV 2 years ago. I am 52 years old and have no history of thrombosis. As a result I belong to the low risk group and I only started with aspirin and phlebotomy. Since February of this year I have been participating in the clinical trial phase 3 of Rusfertide. I do not have official confirmation yet but I suspect that I have been on the drug (and not placebo) since the beginning since I have not needed phlebotomy since the start. My hematocrit remains below 45% with a dose increase from 20 to 30 and now 45 mg. On the other hand my platelets did go up (1.020), as did my white blood cells (17.65). Apart from fatigue and hair loss, I have few symptoms. My AB was tested 2 years ago via bone marrow puncture and was 43.8%. A year later, this was found to be 78.5% in a blood test from the study. This may not be a 1-on-1 comparison due to a different measurement method.
In any case, I would like to discuss the option of Besremi with my hematologist. Since September, this is reimbursed in my country (Belgium). Pegasys has never really been an option here because it was not eligible for reimbursement as an off-label drug. As a result, hematologists have little experience with the use of IFN. My hematologist has doubts because she judges that I, as a low-risk patient, do not actually need medication. In theory, phlebotomy would suffice. In addition, I would also like to point out that I have had periods of mild depression in the past. I have Vitiligo and therefore have a greater risk of an autoimmune disease in the thyroid. I had 3 periods of severe hives and my liver values are also disturbed (unknown cause). In order to prepare my case to my doctor, I would like to ask you the following questions:
- what were your personal arguments for (or against) starting IFN as a treatment option?
- have the guidelines in your country been adjusted in the meantime and is IFN now also a first-line therapy for low-risk patients?
- do you see any red flags in my situation for or against starting IFN therapy?
Thank you for reading this post and for your possible answers. Have a great weekend in the meantime!
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AnPV
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interesting what you say re Rusterfide, not many people are on it, do you think it increased hairloss and fatigue?
Your platelets are quite high as are whites, that itself is no reason for change necessarily but not ideal, your AB appeared to jump quite fast. AB from the marrow tends to be more accurate but it’s still quite a big jump. Was the AA in blood done as a separate test or as part of Next Generation Sequencing ? Your plan to consider a new treatment is a good one , Rusterfide will not help platelets or whites or AB as far as I know.
Your doc says your low risk and not necessarily need meds, maybe maybe not, but you may benefit from meds. Many experts like to see whites under 15 and platelets at 1000 are getting up there and AB rising , if it were me I would try different meds.
You mentioned Interferons, you are wise to consider the mood and autoimmune possible risks, hopefully EP guy will say more about the autoimmune issues he has experienced, the mood one is a possible issue, I had that on Peg.
I think some countries use Peg/Bes as first line.
Hydroxy will control your counts and less chance of autoimmune or mood issues, it has its uses and risks and tends not to lower AB apart from initially.
Then there is Ruxolitinib which I am on, usually side effect free, no mood or autoimmune issues, probably the best for controlling counts and can for some lower AB, mine is allegedly down to 1.08%. It can for some make it harder to control weight, but not impossible.
You are youngish and just starting the PV journey. My 2 cents is that it is wise to have a chat with a MPN expert to set direction and see him/her yearly or so, they can advise you and also write or call your local Haem.
I don’t know of any in Belgium, there is Kiladjian in I think Paris, he is good and a nice man, I’ve spoken with him at conferences, he is a Interferon expert but has many patients on Ruxolitinib also.
Hi, Thank you so much for your reply. It is helping me a lot.
I will look into rux with my doctor. This seems to be a valuable treatment option as well but I am not sure if this is a first line option in Belgium.
I had some doubts in making an appointment with dr. Kiladjinan but I will do now! He is the one with lots of experience and there is a lot of side effects to consider in my case. So that appointment seems a wise thing to do in order to make up my minde regarding Besremi.
Rusfertide did increase my platelets, they went from 700 to 1000. It is a known adverse effect. But I tolerate the drug very well and I also feel better with it. The reason for the loss of hair is probably due to too many phlebotomies (12) in a short period of time (4 months).
You are asking the question about using Besremi for a low-risk PV in a very logical and thoughtful fashion. These would be my answers.
- what were your personal arguments for (or against) starting IFN as a treatment option?
Besremi is an effective cytoreductive agent for treating PV. It has worked better for me and been much easier to tolerate than either hydroxyurea or venesections. HU and venesections both had side effects that were worse than the PV symptoms. In addition, I have a non-driver mutation that increases my risk of progression. There is evidence that Besremi may reduce risk of progression. My positive response is reflected in a reduction of JAK2 allele burden from 38% to 10%.
- have the guidelines in your country been adjusted in the meantime and is IFN now also a first-line therapy for low-risk patients?
The NCCN guidelines have been adjusted, recommending Besremi as a preferred option for low-risk PV. These USA-based guidelines are widely recognized.
- do you see any red flags in my situation for or against starting IFN therapy?
There are some cautions that you would need to be aware of regarding Besremi in your case. The history of depression, presence of an autoimmune disease, any thyroid issues, and the disturbance in liver function would be concerns. These are all potential adverse effects with Besremi. Close monitoring would be needed if you opted for Besremi.
In making this decision, it will be important to be very clear about your treatment goals and risk tolerance. There is more to treating PV than reducing risk of thrombosis. Managing the constitutional symptoms, reducing risk pf progression, and avoiding the adverse effects associated with the different treatment options are also important. Once you are clear about what you want to achieve through treatment and what you ae willing to risk to achieve your goals, you will be able to have an informed discussion with your doctor.
Hi, thank you so much for your reply. It is helping me a lot.
Besremi would be my first option now that it is availalbe in my country. But reading posts on MPN VOICE made we wonder whether it would be suitable for me. The A-I risk is likely to be present in my case. I will consult an experienced MPN specialist to get advice on the 2 remaining options regarding Besremi: (1) only start IFN with close monitoring as you say or (2) don't start at all.
Thanks for sharing the US guidelines, these are helpful!
As our friend ainslie suggests, you may also wish to consider ruxolitinib (Jakavi) if it is available in Belgium. Besremi and Jakavi are both effective medications for treating PV. Reviewing all of your options before making a decision is always a good idea.
Your Dr's opinion you don't need any other treatment is at odds with your high WBC and esp PLT levels. Even if you continue the Rusfertide that is not designed to address PLT or WBC.
IFN is a good option to reduce your AB (VAF) and other blood counts. For ongoing AB tests, it's usually via blood. I also got my 1st one via BMB.
With your high value it may be desirable to reduce AB as you have read here and the related publications; reducing it is now known to be preferable than not to. Some reports discussed in prior posts point to IFN being more effective in reducing the higher starting AB values than Rux but both have this ability for many.
However your autoimmune (A-I) risk is a contra indication. In this report: "14-55% of patients with vitiligo were found to have comorbid autoimmune conditions. Associated autoimmune conditions include inflammatory bowel disease, pernicious anemia, systemic lupus erythematosus, thyroid disease, and less commonly, Sjogren’s syndrome".
See Table 1. there for the list of common vitiligo-associated autoimmune conditions.
While thyroid conditions usually have good treatments most the others don't and you really don't want any added risk of "catching" them. It is a way different life experience category than low risk MPN. The IFN labels also suggest weighing existing A-I risk. If you do proceed you may want to get deeper testing for A-I; this is some of them I got:
Rux does not share this rare but dire risk and in fact looks favorable for treating alopecia areata that is in table 1 and other types of hair loss.
Closer to the point FDA has approved ruxolitinib cream(Opzelura) for vitiligo. This is not the oral form we use and I can't say it's appropriate for your particular vitiligo, but it's worth a discussion with your derm and rheum Drs.
With your A-I risk and mild mental health risk, and the potential off label benefit with Rux, these are factors to consider.
A side note, among members it's not unusual to have inadequate HCT control with IFN, at least initially or longer. So if you go with IFN and encounter this, the Rusfertide could be helpful if you still have access.
Hi, Thank you for taking the time to respond to my message. It is really helpful.
I'm sorry to hear about your AI development due to IFN. This is indeed a risk that I have to think about carefully. My life is fine as it is. I am almost without symptoms. It would be terrible to make my situation worse because of medications I would take to minimize the possible risk of progression (because of those rising AB?). But it remains a difficult decision for me. You have to weigh one risk against another without knowing if the risks will ever materialize and when that will be.
Thank you for sharing the info about the ruxolitinib cream. I'll discuss this with my dermatologist. It might be a nice benefit if using Rux would be my best treatment option.
And indeed, I discussed Besremi as an additional treatment option with my hematologist for the reason you mention. While using Rusfertide, I would be able to start IFN slowly (or very slowly) because my HCT is under control.
I'm not a doctor, but if I had your high WBC/Platelets/Jak2 AB I would be concerned about disease progression cutting my life short. onclive.com/view/disease-du...
So I would be inclined to try low dose Besremi (a 50-150 mcg shot every 2 weeks) to try and slow or largely halt progression. I agree with Hunter: "Close monitoring would [initially] be needed if you opted for Besremi." If, after several months of treatment, it turned out the side effects were tolerable, then maybe the close monitoring could be relaxed.
Thank you for your reply! It confirms what has been said before and that is helpful to make up my mind.
Those high WBC/platelets/JAK2 should not be ignored. Thanks for sharing this article! It is clear to me that I have to take action and discuss this with my doctor.
The option of Besremi comes with additional risks. I don't know (yet) if I am willing to take them but the advice of close monitoring and consulting an MPN specialist might do the tric. Also taking a deeper look into the option and the benefit of Ruxolitinib is another step to take.
AnPV, Here's a copy and paste of my "bio" for this forum, to give you another case history: For many many decades I had platelets between 500 and 750, but doctors told me that it seemed to be my "normaI" and that my visual migraines were not necessarily related to platelets. After some serious malaise in the summer of 2022, I learned my red blood cell count was off too. First I was diagnosed with ET, then quickly it was PV. My BMB allele burden was something like 38%. I was on Besremi at the start of 2023, going up by 50 mcg every two weeks. I "had to" stop the drug in spring '23 because of a liver enzyme rise when I got to 250/300mcg. Although the drug was already doing its job, my doctor at that time was unhappy with all the risks of Besremi; he had wanted me to take HU. I did nothing all summer as I let my liver mend (no symptoms that I could recognize though I felt low) and worked out how to afford Besremi. I got "financial aide" from the PharmaEssentia company for the last months of the year. After a few months they dropped my coverage but I got it again through the Pan Foundation in 2024. I was on a dose of 100 mcg twice a month, then 50 mcg as the HCT and platelet numbers improved further. In late summer 2024 I shifted to 50 mcg once a month hoping for a lessening of medication induced side effects. Some improvement but no relief yet (Nov 2024) from insomnia and fatigue.
My body reacts quickly to the drug and in February 2024 my platelets went down into the high end of normal for the first time in thirty-some years, from over a million to 361,000 in 10 injections of 100 mcg, with the HCT happily in range, though other red blood counts are off. (I do wonder whether inadequate water intake was causing the HCT rise, but doctor disagrees.)
I do not want to take a higher Besremi dose, whatever happens in the future. My energy level and erratic insomnia are quite enough for me to handle at this dose. I have different symptoms all the time, which makes me feel like a hypochondriac or whiner and makes it hard to keep track of what is caused by my PV and what by the treatment. I am ok for a few days after the injection, and then have little problems coming in waves-- for instance, an irritating burn/freeze sensation around my torso and arms or lethargy. Nothing I can't handle or really make more than a quick mention of at appointments, but yes, affecting my vigor more than I like. One recent problem is endless post nasal drip, which the oncologist says has nothing to do with PV or Besremi. Ah well, do they really know it all? Even specialists are "works in progress," I believe. Hence the function of this wonderful forum, to garner some second hand experience "on the streets" of the disease, from listening to other patients and their triumphs and pitfalls. May good health flow your family's way!
I agree with the value of our experiences. It's considered just anecdotal by most Drs, but I increasingly feel it's of true clinical value if we interpret it right for our Dr discussions.
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