PNH Clone increase/treatments/symptom mgtm

[Junior48]

Hello, sister, 27, mild AA since approx 2010. ATG in 2011, cyclosporine since then. PNH clone increased (i think about 60%) now. Now on high dose blood thinners, no soliris treatment yet. not transfusion dependent.

Questions are, monthly female cycle and high dose blood thinners wipe out iron levels, any recommendations on management (besides iron supp)? Has anyone taken the newer drug, ultomiris (ravulizumab) from the same pharma company that produces eculizumab? is this really an indefinite treatment, has anyone successfully been able to discontinue it? this might situation dependent, but are major health insurers covering majority of the cost?

From what i gather, once a patient makes the cross over from AA to PNH they dont typically regress back and will have to manage the PNH from there on out?


any other feedback appreciated. thanks,

[GoodDay5150]

Yes sir. I am not a medical professional, but I had PNH for man years b4 being diagnosed after my symptoms became very severe. That was in April of 2011 and I was transplanted later that year. My hemoglobin and red cell counts were so low that a general practitioner quickly and wisely referred me to a hematologist. It is my understanding that AA is fairly likely to come back as PNH, but I was only diagnosed w/ classic PNH. I took oral iron supplements after being diagnosed, and I think that someone told me that iron injections can be more effective. As far as Soliris is concerned, an HMO doctor was going to administer it to me b4 my transplant, but then decided against it. In my opinion, my disease was so far advanced that it probably would not have been that effective. As you probably know, PNH symptoms can range from almost non-existent to life threatening. If your sister is not aware, there are some great resources avail. online. They are

https://rarediseases.org/rare-diseas...emoglobinuria/


https://pnhsource.com/?gclid=EAIaIQo...SAAEgJOXPD_BwE

I was part of this study

https://ashpublications.org/blood/ar...-After-Reduced

we all wish your sister success in her treatment


Mario

[Junior48]

Thank you Mario.

[Junior48]

Was wondering what the consensus is on watch and wait before deciding to treat PNH with Soliris? My sister has been noticing more fatigue and weakness, although it changes day to day. some good some bad. she doesn't need transfusions and is maintaining on blood thinners at 2.5. Her doctor said its her call on whether she wants to start treatment. she wants to hold off. If anyone has recommendations for increasing energy levels, managing the fatigue, rapid heart beat etc...it is welcomed!