Iron deficiency and PV(phlebotomy)

[Bobadog]

Hi all,Has anyone had any experience of hepcidin therapy instead of repeated venesections to control the HCTor instead of cytoreductive therapy etc.

This could prevent the iron deficiency?

Thankyou for any feedback on this

[hunter5582]

Rusfertide (PTG-300) is the hepcidin mimetic that is currently in phase 2 clinical trials in the USA. There ae not a whole low of people with experience with it yet, but the results a very promising. This is one of the most promising treatment options for PV in development. It helps to reverse the negative impact of phlebotomy-induced iron deficiency and is well tolerated.

Here is some of what is out there about Rusfertide.ashclinicalnews.org/on-loca...

onclive.com/view/rusfertide...

ncbi.nlm.nih.gov/pmc/articl...

[Bobadog in reply to hunter5582]

Hi Hunter,Thankyou so much,this is really interesting and I would not have found this information.

I’m starting Peg on October 25th,due to the negative effects of continual venesections

I was delaying,for a more ‘accurate ‘ treatment,but Rusfertide may not be available in the UK for some time?

Thankyou so much,

Take care

[hunter5582 in reply to Bobadog]

It will be a while before it is commonly available anywhere. You might check into whether there are any clinical trials that will be done in the UK.

[Bobadog in reply to hunter5582]

Many thanks.I will enquire about this,

Thanks again,

[gvibes]

Hi Bobadog,

I never enrolled but was in discussions with the PTG-300 trial at one of the sites in NYC. It seemed that I would need to go to the trial site initially and monthly during the course of the study for bloodwork and exams. You self administer (i.e., inject) the supplied drug weekly at home. They adjust dose. At some point, they randomly separate participants and give placebos to half the people for a bit. at the conclusion of the study, I believe they allow you to continue for a while if you are getting good results. It is a multi location study with several of the major MPN hospitals participating across the US.

After looking at it, it didnt seem that I qualified yet so I ended up seeing one of their MPN specialists who recommended pegasys. I think its possible I end up on both pegasys and PTG-300 someday depending on if the pegasys dose that I tolerate can prevent phlebotomies.

good luck, respond if you have questions.

[Bobadog in reply to gvibes]

Thankyou very much.I live in the UK,but attend the Haematology clinic at Guy’s Hospital,London,(Professor Harrison)where they are sure to know about US trials.

I will enquire about this,

I hope the Pegasys reduces the need venesections .

I think my start dose will be 45mg fortnightly,so presumably I will have to continue with venesections until the drug kicks in,

Thankyou and good luck with your treatment.

[gvibes]

I could be wrong, but my sense was that they were having trouble finding enough p-vera patients for the trials. Its a rare disease and it is only under pretty rare circumstances that someone would want to go into this trial. I assume that is why it is at multicenters and I am pretty sure the hospital I was at was only trying to get approx 5 people. so maybe it isnt a stretch for professor Harrison to contact the drug manufacturer and get some people going in the UK. Pegasys is taking a while to get going - 5 months now and it seems recently to be improving my overall blood and bringing down hematocrit. Two phlebotomies since starting so far but frequency appears to be diminishing....we are all finding our way, its good to get information from others with these diseases

[MPNBlog]

Hi Bobadog, I enquired with my haem about the possibility of use when PTG-300 becomes available, and they were reluctant to add another drug to treatment unless there was some other factor to warrant it - such as veins failing for venesections, especially in older patients. I guess significant iron deficiency could be another reason. But as a preventative I gathered there was reluctance to prescribe extra drugs. Something to consider as I too just wanted done with venesections, but I do tolerate them well. But good to know there may be an option if/ when it's needed. Best wishes.

[Bobadog]

Many thanks for the sharing.Unfortunately during the last 5 years,I am having venesections more regularly (3-4weekly) ,and the PV is now not being controlled successfully .

How regularly are you receiving venesections ,and when did this commence,if I may enquire?

Thanks

[gvibes in reply to Bobadog]

wow, 3-4 weekly phlebotomies is crazy - that is tough. I had 8 over a three week period when I first diagnosed but the iron deficiency slowed my red cell production way down - probably monthly phlebotomies would work for me with no cytoreductive. Get that pegasys going.

[Bobadog in reply to gvibes]

I meant ‘every 3-4weeks’ ,sorry to be so unclear.I’m still hoping the pegasys will ease things,when I commence.

Thanks very much.

[gvibes]

Still alot but not insane - that makes more sense...take care.