Treatment goals for patients with PV remain varied according to the clinical scenario. Treatment-free remission is currently only considered viable with IFN treatment. However, HU and ruxolitinib remain adequate options for first-line therapy through the control of disease-associated symptoms and reducing the risk of disease progression, which is especially important in younger patients. Overall, a thorough review of current treatment rationale may be important in optimizing clinical outcomes and fully exploring the potential for treatment-free remission.
This is a concise summary of current information on PV treatment goals. A deeper understanding requires referencing the studies cited as well as understanding the underlying processes involved in PV. The notion that treatment=free remission is even possible is new to the MPN space. It is a very positive step forward. While not a cure, it is a hopeful alternative to the outcomes available in the past.
We can only hope that this outcome is viable for at least some of us with PV. While not a cure, it would be the closest thing possible until we someday find a cure. Someday we will End MPNs!
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hunter5582
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Very interesting I had a whacking great dose of interferon over an 18 month period yes it caused other auto immune issues but blood counts are rising slowly platelets are a bit high but holding off going on hydrea at the moment and only been on aspirin for a year now
Wow - thank you for posting this. When I was diagnosed with PV almost 2 years ago there was no thought of this being a goal. It is exciting. I’m still early on Besremi - 7 months and counting and this is so hopeful … a wonderful vision of treatment free remission! Yes - someday the end of MPN’s!
I experienced a taste of this with 3 months off Bes. Dr Gotlib said my continued (small) Jak2 reduction was from the 6 months ago IFN rather than the freshly started Rux.
In this part:
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Patients are considered to be in treatment-free remission when therapy is discontinued and the following occur:
Complete hematologic response
Deep molecular response
Reversion of bone marrow picture
Absence of vascular events and disease evolution
No treatment restart
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Bone marrow reversion is a criteria.
In some reports from Dr Silver's group they say allele often goes down while marrow remains impaired, and this creates uncertainty on the stop of therapy. I think they have the best marrow data over long terms. Does it matter? They don't know.
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I asked Dr Gotlib whether Rux can ever stop, he said no. I wonder if maybe so for those who get to the very low alleles on it. I don't think there is any data in this area.
Time and the data will prove the outcome one way or another. The current research underway about the long-term outcome for people who experience a deep molecular remission will take time to generate outcomes. The notion that a treatment-free remission is even possible is a very big step forward. Perhaps Jakafi will also prove to have this potential for people who achieve very low allele burdens.
“However, HU and ruxolitinib remain adequate options for first-line therapy through the control of disease-associated symptoms and reducing the risk of disease progression”
To the best of my knowledge HU doesn’t slow progression, it can lower AB for a year or so but that’s all. I am hoping Rux may slow progression in view of AB reduction etc but it’s probably a bit grey area. I note Clair Harrison says none of the drugs slow progression so the usual difference of opinions. Yes let’s hope for that cure.
I agree that it was a typo or a poorly constructed sentence. I think the intent was to specify symptom control for HU. Jakafi has excellent symptom control. The information on Jakafi potentially reducing allele burden is new. Not sure that anyone thinks that HU reduces the risk of disease progression. Some think the opposite about HU.
Also agree about the MPN experts not agreeing. Those of us on the forum do not always agree either. Perhaps the one thing we can all agree on is to seek to end MPNs.
Firstly apologies to anyone who has read what I wrote re HU, I should have expressed it better. Secondly thank you for correcting me, I didnt know that about HU and I am delighted for all those on HU that it may reduce scarring and fibrosis as reprted by MPN Voice . I have never taken HU but I know many do very well on it and long may that continue.
No one was more shocked than myself when Maz posted this information sometime back. I was delighted at this positive news, as HU tends to get a lot of negative comments. 😁
I have posted somewhere on HU and fibrosis (and I recall cellularity also ). I think the original report is in there somewhere. I concur that we have seen this benefit reported and I've been surprised by the absence of follow up studies . If I recall HU was better than IFN in this area. But as is often, the study I believe was not for the long periods needed for IFN. Still the Silver group's info here suggest no magic for IFN in marrow even in the long term.
Maybe they mean no strong correlation to allele, ie marrow reversion is seen but not necessarily connected to the large allele reductions.
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For Rux a quick search finds potential benefits too:
"Ruxolitinib treatment versus BAT was associated with greater odds of BM fibrosis improvement or stabilization and decreased odds of BM fibrosis worsening"
This was a 5+ year study. Not clear what BAT was here (best avail alternate) But it was for advanced MF. Could be against PV the benefits are even greater.
I have been there with sleep meds. Decades of chronic insomnia. Thank goodness for Belsomra. It is the only thing that ever worked without disrupting sleep architecture. the dual action orexin agonists are a real benefit to those with insomnia.
I expect efficacy is related to do with the cause of the insomnia. Insomnia is a symptom, not a distinct disease. Treating the underlying issue leads to effective intervention.
I'm not sure where you found evidence that Silver said anything negative about IFN in regard to bone marrow fibrosis especially in comparison to HU.
But a quick search of the phrase: "does interferon reduce bone marrow fibrosis development in polycythemia?"
Brought me to here:
"A few years later, Silver3 demonstrated the safety and efficacy of rIFNα treatment in patients with polycythemia vera (PV) and afterward, its value in the proliferative phase of myelofibrosis (MF) was reported,4 resulting in normalization of marrow architecture and cellularity, and reduction in degree of fibrosis to normal.5 Many subsequent studies in more than a thousand patients have confirmed that rIFNα is safe and effective for treating essential thrombocythemia (ET), PV, and early-stage MF patients: in ET, it normalizes elevated platelet counts within weeks to months in the large majority of patients; in PV, it reduces or eliminates the phlebotomy requirement and the degree of pruritus, normalizes elevated leukocyte and platelet counts and reduces spleen size; in MF patients, it reduces or normalizes elevated leukocyte- and platelet counts and—as noted above—may also induce regression of bone marrow fibrosis in some patients after long-term treatment.5 All these studies have been thoroughly described in several recent reviews.6–
From:
PERSPECTIVE
New Perspectives of Interferon-alpha2 and Inflammation in Treating Philadelphia-negative Chronic Myeloproliferative Neoplasms
"we have observed persistent marrow hypercellularity and/or progressive fibrosis in PV patients receiving rIFNα despite developing a significantly reduced %V617F in peripheral blood"
" Our results (re low marrow response, BMR) question the use of JAK2V617F allele burden as the sole criterion for discontinuation of therapy."
Persistent is a strong statement, I didn't recall it being so clear.
The inconsistency you correctly note might be that most the other reports I can find were for MF, as in the ref you have, while this one was specific to PV.
It leaves the usual question as they also pose, which if any are most important- VAF and/or BMR. We're not likely to get large n data on BMR since getting that is not easy on the patient. There may be newer data with a different conclusion, which would be nice to know.
(I'm having a decent day so I can look into these things)
This is exciting. Before I started Besremi I asked Dr V about the “clinical” remission and possibly having periods off of it. And he said absolutely was possible. Now because I am hoping it will work for MS too I may be tied to it forever but I see a lot of my patients in clinic with MS that did treatment free periods too. I would just like to see both of my diseases managed and bonus would be if one drug could do it! Here is hoping for everyone
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