Unfortunately I did not meet parameters for the PTG-300 clinical trial for PV treatment. You have to have had a minimum of 3 phlebotomies in 28 weeks. While my past history included phlebotomies every three weeks even when on hydroxyurea, my recent history is no phlebotomy for 14 months, then two in six weeks, then none for the last 5 months. I will likely need one in March. So I ended up in a bit of a Catch 22. I am interested in PTG-300 because I am having side effects from being so iron deficient. I am so iron deficient that it is doing "too good" of a job controlling the erythrocytosis to get into the study. I would definitely qualify for the study if I was not so iron deficient. The hope for PTG-300 is that it can control the erythrocytosis without phlebotomy and let you maintain higher iron levels. That is an outcome I would like to experience.
The ironic thing is that I could opt to take iron supplements, which would then require more phlebotomies to lower my iron levels and control erythrocytosis. My hematologist actually suggested that this was an option I could pursue. I guess it would get me into the PTG-300 trial, but I am not inclined to go this route.
I remain interested in PTG-300 as it appears to be a very promising option for PV treatment. It has Fast Track designation. I may have to wait until it is FDA approved. I am also interested in Besremi, which is nearing FDA approval in the USA. I think the research on the use of Besremi in low-risk PV is very promising. Technically I am not low risk, since I am age 65 and have some cooccurring medical conditions. I am a bit concerned that the iron deficiency seems to be pushing my thrombocytosis up into the 700-900 range. Fortunately, the erythrocytosis is well controlled and I remain thrombosis and microvascular symptom free. I have not seen any signs up worsened hemorrhage with the increased platelets either.
I have an appointment in May with the MPN Specialist at Johns Hopkins. Continuing to work with my local hematologist on a regular basis, monitoring labs and considering options. I am not going to rush a decision since there is no crisis, but I am thinking I may change my treatment plan and do more than phlebotomy only. Hopefully the other options I am interested in will be available soon.
All the best to all of you.
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hunter5582
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While disappointed, I do understand the need to stock to the study parameters. It is critical to the reliability of the study. I do want to see PTG-300 gain FDA approval ASAP. I really do think it could be a significant step forward. I will also be very interested to see PTG-300 + Besremi combination for some PV patients. I am thinking it could be an effective strategy for some.
Every one of your posts is extremely informative. May I say that it has triggered my journey to go back and check both my diagnosis and treatment . I am studying that at the moment before I ask for a “ re - discussion “ with my hematologist .
I have never had
No Splenomegaly
No Thrombotic events
No symptoms except rare pruritis which has now disappeared
Jak 2 mutation heterozygous
Platelets controlled in normal zone- admittedly after HU .
Hemoglobin hovering at 16 - admittedly only after HU ( 19 before that )
Mutant allele burden and gene panel - struggling to get it tested where I live .
Yet have been put on Hydroxyurea and aspirin .
After listening to Dr Spivak and Dr Silver coupled with your superb observations as some going through PV , am beginning to wonder if I should oush to try other molecules such as Interferon alpha .
Of course my other monster GCA also lurks in the background , fairly complicating the issue , though it is fairly controlled with a 3.5 mg dose of daily steroid at the moment .
Thankyou for your studied observations .
They are observations from a learned kindred spirit is how I view it
It is great to have others to talk about these issues who understand. MPNs are complex enough without complicating cooccurring medial issues. The GCA certainly does complicate things for you. I just don't know if PEG-IFN is even an option since you already have an autoimmune disease. I hope that the combo of a MPN Specialist and and immunologist can give you a good answer to that. I also would wonder about Jakafi as an option.
It does look like you are considering the right things in making a decision. Also hope you can get the additional gene testing. It really does matter. It is discouraging when systems of care make getting the testing so difficult. I am a great believer in persistence and advocacy. And sometimes in just learning how to pull the right levers in your system of care. FYI - I learned how to look up the Coverage Policy guidelines for my insurance program. It helps when I can prompt the docs to use the right terms when getting the authorizations. Also when I need to get involved in filing an appeal.
I hope you are able to review your options with your care team soon.
Was just reading an article on inflammation and myeloid malignancies that may be of interest to you and others with autoimmune conditions. There is a section of the article that reviews the links between autoimmune conditions and MPNs/other myeloid malignancies.
Will certainly go through this article . It would be of immense value to me .
Also , just today , I did ,as you said, try to pull a few levers and finally found someone who will do both the Jak 2 allele burden as well as the gene panel.
So ... happy to take small firm steps forward .
Also I eventually plan to request fir an online consultation with Dr Spivak and Dr Silver hoping they will opine on how to approach this double whammy of PV plus GCA that I am battling .
Dr. Spivak has retired from clinical practice, but is still active in the MPN world. I used to see him. Now see Dr. Braunstein at the Johns Hopkins MPN Center.
As you likely know. Dr. Spivak has some different views regarding MPN treatment, which is a good thing. MPNs are not monolithic diseases. What we really need is highly individualized care based on the presentation we each have. Considering different views and the reasons for them makes us all better informed.
Really glad to hear you will be able to access the genetic testing. You prove the assertion about assertive patients receiving higher quality care.
Just a few comments from me. I started PTG-300 a month ago. Yesterday, was my first follow up appointment. My HCT was under 45 so no phlebotomy was needed. All of my counts were near perfect. This is the first time in 2 years that this has been the case for me. I’m also on a low dose of Pegasys. I hope my luck continues. I’ll have my labs checked again in 30 days.
Concerning PTG-300, I believe in nearly all patients is has controlled RBC count and eliminated phlebotomy. This drug not only eliminates phlebotomy but it controls the HCT and therefore should lessen the risk of a clot, etc. However, it is not believed that this drug has an effect on the malignant clone. Myself and my doc believe that the perfect combo is PTG-300 and Pegasys as Interferon has been shown to have a effect on potentially modifying the disease.
I hear quite a bit of chatter about Besremi, and while I’m excited that the injections will be less, it’s my understanding that there really is not much of a difference between Pegasys and Ropeginterferon. They are a slightly different version of interferon.
One other point to note is that my specialist has said that his institution’s research indicates that out of all treatment options, patients treated with phlebotomy only tend to have the worst outcomes vs patients treated with HU or Pegasys. Just an observation.
I wish everyone the best of luck on their journey, but thought I would share my experience with everyone. Take care.
I agree with you regarding regarding the small difference between Pegasys and Besremi. IMO there’s really no point waiting Besremi’s availability before starting interferon therapy. Besremi could be a life changer for those intolerant to Pegasys, but it won’t change much the excellent IFN efficacy.
I’m in the US and receive treatment at Weill Cornell in NYC. There are many institutions offering the trial.
My doc and I just had a long conversation about interferon yesterday. It’s certainly not for everyone, but I believe most tolerate the drug just fine, especially at a low dose. The docs at Weill Cornell are interferon experts. They have been suggesting the benefits of interferon for decades.
I can only discuss my personal experience, but for me interferon has been an extremely slow worker. Along my 2 year journey I have had some thyroid issues and slightly elevated liver enzymes. This has definitely been somewhat of a rollercoaster ride as even while I have been on interferon I have required phlebotomy.
On the flip side, currently all of my counts are perfect for the first time in 2 years to include my WBC, RBC, Platelets, RDW, and LDH. Not sure of the significance but my allele burden is also down from 23 to 14 percent since diagnosis. I’m hopeful that PTG-300 will also keep me phlebotomy independent while only on a low dose of Pegasys.
I believe the future of PV treatment will be interferon in combo with other drugs. I also believe the disease will be begin to be treated as early as possible. There has also been discussion of interferon and arsenic trioxide in combo to potentially “cure” the disease.
I know my circumstances could change at anytime, but for now I’m pretty happy with the control of my PV. Hopefully it continues. I only wish that the medication worked quicker, but it’s definitely a long ride. Good luck!!
Thank you for the information. I am very hopeful about the potential for the interferons and the combination therapies. It is all a balancing act to consider both the benefits and adverse effects of our treatment options. Our options really are improving. Please stay in touch and let us know how your approach is working.
Thank you for your post. It gives me hope for treatment options when I cross that bridge. Did you incorporate any diet modifications throughout your interferon treatment to help optimize its effects?
Hello…. Iam AllEars I am interested in your current experience with Ptg-300….I was offered this a year ago and passed ( unfortunately). My RWB is so high and the fatigue from low iron is debilitating….i am on Pegasys now for 4 months with good results on platelets…..but there is no movement in high red numbers…..
Hello, my experience with PTG-300 has been good. Of course there was an FDA hold for a while, but it has since been lifted and I’m now back on the drug and so far everything is good.
As far as Pegasys goes, it is my experience that usually platelets and WBC get controlled first and RBC last. I have read it can sometimes take 3 years or more to get RBCs controlled.
I will say that after almost 3 years on Pegasys I feel better than I ever have since diagnosis. Almost normal in fact. If the PTG-300 allows me to avoid phlebotomy it will be a win-win combo with Pegasys to combat the disease. Unfortunately, Pegasys is a slow worker. Hang in there!!
From my understanding, Pegasys is considered an off-label drug for MPN treatment, whereas Besremi (pending FDA approval) will be designated as a treatment drug for PV, and hopefully ET. That could make the difference for increased insurance approvals and decreased rx costs compared to Pegasys for MPN patients.
Correct. Jakafi is the only FDA approved drug for MPNs (PV and MF). Hydroxyurea is cheap so the insurance companies don't care that it is off-label. I am very hopeful that Besremi will make it onto the formularies once it is approved. I expect many will require the patient to fail HU tx first - which I have already done. Pending approval was mentioned several times in today's MPN Advocacy PV presentation. Really hope we hear something soon.
That’s frustrating to hear that insurance groups may require failed HU treatment before approval for Besremi, certainly not always in the best interest of the patient, though I can hear insurance companies arguing that HU has the same efficacy, despite its cytotoxicity.
My daughter's hemo-doc (she also has JAK2+ ET - likely PV) plans to start her on HU then pull the plug on HU as soon as she has adverse effects and get IFN approved. (If formulary is an issue). Given the high probability of adverse effects, that plan should work. Being a believer in genetics, I will assume she will be just as HU-intolerant as I am.
Sounds like a well thought out plan for your daughter. Thank you for sharing this, it gives me some ideas on how I can approach my treatment options in the future.
It’s awful when you get rejected for clinical trials isn’t it. What is your heamoglobin level at the moment? That’s if you don’t mind me asking.It’s something I suffer from but can’t take iron because my ferritin is very high.
From my last CBC on 01/11/21 HGB = 12.3 (12.4 on 12/04) (12.2 on 11/23)
RBC = 6.26 H (6.35 on 12/04) (6.16 on 11/23)
HCT = 42.2 (42.5 on 12/04) (41.3 on 11/23)
RDW-CV = 20.5 H
MCV = 67.4 L
MCH = 19.6 L
MCHC = 29.1 L
MPV = 9.7
PLT = 701 H (900 on 12/04) (752 on 11/23)
WBC = 7.52
NEUT = 5.06 67.3%
MONO = 0.66 8.8%
EO = 0.34 4.5%
BASO = 0.13 1.7% H (my Basophils always run a bit high)
IG = 0.05 0.7%
LYMPH 1.28 17.0% L
I have not run an iron panel for a while, but I know it would show quite low. My Ferritin level has not gone above 7 for ever for about 2 years (from when I did phlebotomies every three weeks). Everything is staying pretty stable, except for my platelets. They are now cycling between 700 - 900 rather than my historic 500 - 700. Erythrocytes are very gradually creeping back up. My last phlebotomy was in August.
I am going to be reviewing the bigger picture in my next couple of appointments. I see my regular hematologist in a couple of weeks. He is a great doc, but not a MPN expert. Will be seeing the MPN Specialist in May. Plan to review the last couple of years history and how I am responding to the phlebotomy-only regimen. As Walshman and Manouche suggest, it may not be worth waiting for Besremi. I may go ahead with Pegasys and see how my body reacts. I would, however, prefer the Besremi since it is easier to tolerate.
Will be making a decision in the next several months.
Thank you for continually keeping us informed. I have my eye on Besremi also, hoping it will subsequently be approved for ET after its FDA approval for PV.
In the current clinical trial for ET, Besremi is offered as a second line therapy. When PharmaEssentia applied for FDA approval of Besremi for PV, do you know if it was filed as a second line therapy also?
Thank you for the information. PTG-300 sounds like a game changer and I hope it is approved. I have PV and phlebotomy treatment but am very iron deficient
Interesting to hear from @walshman too - to hear about the trial and positive results so far.
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