Ruxolitinib

[Ameliav]

Hi all, I have PV and AB is high , around 81% , spleen size slightly increased but if I didn’t know I have PV I would think I am the healthiest person around as I have no symptoms. I was diagnosed end of 2018 but had it al least since 2015. I am considering taking a part in clinical trial for Ruxo and would appreciate any comments , pros and cons, side effects ect. Are any of you taking this therapy , can you share your experience please . I am not taking any medications and was always again HU because of increased risk of skin cancer . I’ve just done some readings and looks like Ruxolitinib can also cause skin cancer . I would appreciate your thoughts

[Bullace]

Hi. I have MF and was on Rux for about 10 years. I was eventually taken off it because it had stopped reducing my spleen, which is large. I found it a really amazing drug in reducing the symptoms I was having, but they did have to do a bit of balancing to find the right dose. I did have skin problems with it. A few squamous cell carcinomas which had to be excised and quite a few basal cell ones which were easier for dermatology to deal with. It is a problem but it seemed worth it to me for how much the drug helped me in other ways! It's always a balancing act. I do wish you well in your decision making and hope you continue to be symptom free.

[Otterfield]

I was on Ruxolitinib for about a year and a half. It did a fantastic job of reducing my spleen. I was very careful to avoid infection but I did manage to pick up Campylobacter (probably from some dodgy pastrami) and I got Shingles. I felt really well generally.

Previously I had been on Hydroxycarbamide for a number of years and felt well, apart from a few mouth ulcers.

Skin cancer is not inevitable on these drugs. I have pale skin, am careful in the sun and had no problems.

[monarch5000]

All this was known about Pegasys interferon 9 years ago, but there are still alot of hematologists who have no prescribing experience with it and so won't mention it as a treatment option:

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[Ameliav in reply to monarch5000]

thank you , I am very aware of Pegasys and done lots of reading on it over the year . I’ve been pushing for this medication for a while , my haematologist suggested a clinical trial including pegasys and Rux but after reading more about Rux I think I made my mind not to take part as I don't suffer with any symptoms and Pegasys is in my opinion my best option .

[ainslie in reply to monarch5000]

as previously written, not that many docs agree with what Hasselbalch has written and this phrase “operational cure” is either a bad translation or nonsense. People are either cured or they are not, unfortunately they are not. For a minority of those that can tolerate Peg it may and the word is may in the opinion of some doctors slow progression however in recent vids some experts such as Clair Harrison and Tefferi have said none of the drugs have been shown to slow progression. I don’t know which docs are correct but by definition some must be wrong. For the record with current info I still think Peg/Bes would be my drug of choice if I could tolerate it but unfortunately I can’t. It’s probably the best current drug but we have to be realistic and responsible about what it can and can’t do.

[dogsandhorses in reply to ainslie]

May I ask why you couldn't tolerate Peg/Bes?

[ainslie in reply to dogsandhorses]

I don’t know why but it made me depressed and incredibly itchy, if you are considering it don’t let that put you off it only happens to a minority, especially the incredibly itchy bit

[Rachelthepotter]

I have MF and been on rux fir 6 yrs. Got nasty facial sccs- skin cancer effects not recognised in England then. Rux also makes me anemic I’d suggest give it a go , and watch any sun exposed areas carefully. H

[Orangeboykitty]

I was on Rux for over a year. It was great for reducing my spleen size. It did lower my immune system temporarily and I got shingles followed by an eye infection. I had no problems after those things went away.

[Flufferdoodle]

I was diagnosed with PV when I was pregnant with my youngest daughter back in 2011. Since then my PV as progressed to secondary Myelofibrosis. I have been on Ruxolitinib for 2 and a half years and now I’m well enough to go ahead with a transplant. Ruxolitinib changed my life dramatically. I was ill 90% of the time and the difference is amazing, I still have a few bad days but that’s to be expected. I haven’t had many side effects apart from some weight gain (a good thing in my case) and a little nausea on a morning but my spleen size has been reduced too. I highly recommend trying the Ruxolitinib. I hope everything works out well for you and you have as much success as I have ☺️

[rcan_pv]

Hi. First up, HU is chemotherapy while Rux/Jakafi is not. Yes, both are implicated with certain skin cancers but this is a prime example of where benefit can exceed risk.

As Otterfield mentioned, a careful regimen of protection, i.e., sunscreen, hat or umbrella, limited exposure to the sun itself and visits to the dermatologist can help prevent malignant cancers. If you suspect a problem don’t wait to contact your doc. Skin cancers can progress rapidly.

[Ameliav in reply to rcan_pv]

thank you for your reply . I am trying to weigh up the best course of therapy for me . I have PV with no symptoms and very slightly increased spleen and wondering if I would be better off with Pegasys . From all the replies I got here most people used Rux for symptoms and enlarged spleen management , which is not applicable in my case .

As always it is about weighing up the pros and cons i.e. the best management of the PRV/ Myelofibrosis which improves quality of life and overall survival versus 'potential' toxicities. Often the literature can seem very daunting and the honest truth is that we will never know whether or not you will get any of the potential side effects unless we try.

[Jamesxyz]

Hi That's great that you feel well.

What is your age and what are your blood counts like?

Are you jak2+ ?

[Ameliav in reply to Jamesxyz]

48 yrs old , HCT well controlled , now 39 and have maybe 2 venusections a year . High platelets and wbc , platelets 900-1050, wbc 18-21

[ainslie]

to drug or not is a tricky one and then which drug is also a tricky decision. All drugs have sides sooner or later and many have benefits, so it’s hard. Probably wise to see a MPN expert to discuss options. Personally I would be a tad wary on going on a trial just because you were offered it. What’s more important is you chose best treatment or drug for you. Drugs can have pros and cons.

You asked about Rux, I’m on it for PV since six years, for me it’s been good, I feel much better on it , some experts are saying it is lowering allele burden etc whatever that means in reality. About the skin cancer bit, in my opinion with what I have heard from experts it’s unclear if it’s the Rux or previous heavy Hydroxy long term use which many Rux patients have been exposed to because until recently Rux was mainly for those who failed on Hydroxy. That’s my understanding but if others have different experience please chip in.

I haven’t been on Hydroxy but have had ten years of UVB light therapy before and during Rux high dose and my skin is so far perfect. There is a view that Rux can maybe bring out skin cancers after a lot of previous excessive sun exposure, this is tricky to pin down as how do we know if it’s the excessive sun exposure or the drug.

[monarch5000]

The Silver MPN Center in New York City treated 470 PV patients over a 30 year period and this is how their outcomes differed depending on their treatment:

20 years after diagnosis:

95% of PV patients who had been treated with interferon were still alive, 15% of them had progressed to post PV myelofibrosis

63% of PV patients who had been treated with hydroxyurea were still alive, 41% of them had progressed to post PV myelofibrosis.

57% of PV patients who had been treated with phlebotomy-only were still alive, 49% of them had progressed to post PV myelofibrosis.

Source: tinyurl.com/544sybph

[GardNerd]

With the obvious disclaimer that I’m not a doctor and wouldn’t want to sound like one… It seems like at your age, with a relatively recent diagnosis, and given that your spleen isn’t very enlarged and your symptoms are minimal/non-existent, an interferon (Besremi or Pegasys) would be the first thing to try. Based on everything I’ve read here and elsewhere (which definitely isn’t everything), the interferons have a chance to modify disease progression and even lower the allele burden.

My doctor at MD Anderson planned to move me to Jakafi if I had progressed to MF. Once it was determined I still had PV, he said we should keep going with the interferon.

Good luck with your decision and with any path you decide to take!

[K-itty]

I've been on Ruxolitnib for only about 2 months...very low dose as sensitive to medications. So far, it's helped with slowly lowering my platelet levels and has relieved the inflammation. The only major side effect that I've had is extreme constipation when I first started on the medication. Thankfully, I discovered a natural Senna product called Swiss Kriss that immediately helped! I also drink a small glass of Aloe Vera juice with a tsp. of magnesium and probiotics before going to bed. I didn't notice where you're posting from, but Ruxo is available in the United States after going through clinical trials. I couldn't tolerate HU so grateful this is available.

Good luck!

[Ameliav in reply to K-itty]

thank you , I am in UK , and also this is a clinical trial that has been offered , so 50% chance of going on Pegasus and 50% on Rux .

[monarch5000 in reply to Ameliav]

California MPN specialist that started prescribing Pegasys about 5 years ago was surprised by how much better rather than worse most of her patients felt: youtu.be/OsdoYoA1kLQ(Not working as expected?)

[ritaandscooter1]

Many other variables play into your diagnosis and whether or not to go on a drug. Are you considered low risk? Are there are mutations or drivers that could make you progress? If you have no symptoms, are healthy, have been deemed low risk and have no other noted bad drivers why go on a drug at this point?

I've had PV jak2 now for over 20 years (age 62 now) and have practically no symptoms but fatigue/anemia, considered low risk, very healthy, no identified other drivers, no progression (BMB recently) and take no drugs except 2 aspirin a day with 3-4 phlebotomies a year.

My recent BMB showed my AB at 91% with no signs of progression. My MPN specialist is Dr. Tefferi out of Mayo Rochester. He doesn't put much significance on AB as nothing has been factually proven thus far. Have briefly tried Pegasy's but did horrible with side effects on a dose of 22.5mg. Every one is different, so don't feel like you have to be on a drug. My regimen has worked for me with all the stated variables. Feel great on most days and deal with lower iron stores by a low dose iron pill every Sunday. Daily exercise is largely the key for me. Best to you.....and find a individual plan that works for you! Kerry

[Ameliav in reply to ritaandscooter1]

I have Jak2 and no mother mutation , I have mayloid gen panel test and didn’t show any mutation . I was also against any medications but because my wbc and platelets are slowly increasing and not going down that’s why they are considering medication.

[monarch5000]

In 2021 four MPN specialists were claiming interferon could be considered an "operational cure" for a substantial subset of PV patients:

.

[ainslie in reply to monarch5000]

the only problem with this there is no such thing as a operational cure, it’s either a cure or it’s not and unfortunately it’s not, it’s a good treatment for most

[Manouche in reply to ainslie]

The term **operational cure** is used to describe a situation where a patient with a chronic disease achieves a long-term remission and symptom-free state, even though there may be some residual traces of the disease in their body. This term is often applied to diseases that are usually considered incurable, such as some types of cancer or viral infections.

For example, in the context of multiple myeloma, a type of blood cancer, operational cure is defined as achieving minimal residual disease (MRD) negativity, meaning that no cancer cells can be detected by sensitive tests, and maintaining this status for at least five years. Similarly, in the context of polycythemia vera, another type of blood disorder, operational cure is defined as having a very low level of the JAK2 mutation that causes the disease, and maintaining a complete hematologic response (CHR) for at least two years without disease progression or complications³.

Therefore, it is not correct to say that there is no such thing as an operational cure. Operational cure is a valid concept that reflects the possibility of achieving long-term disease control and quality of life for patients with chronic diseases that are otherwise difficult to cure. However, operational cure does not necessarily mean that the disease is completely eradicated from the body, and there may still be a risk of relapse or recurrence in some cases. Therefore, patients who achieve operational cure may still need to undergo regular monitoring and follow-up care.

(1) Requirements for operational cure in multiple myeloma. ashpublications.org/blood/a....

(2) TOWARDS A POTENTIAL OPERATIONAL CURE IN PATIENTS WITH POLYCYTHAEMIA .... library.ehaweb.org/eha/2021....