Hunter very helpfully posted the recording of this webinar
healthunlocked.com/mpnvoice...
Much is familiar to us. Here are some high lights. It's long so seems worth a new post. My notes are sort of fragmented. Most comments here are from Dr. Rampal. See actionable item on IDH2 mutation.
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Studies have been focused on post transformation (after progression). There is need to focus on not getting to these points. Compare thinking on solid tumors, we don't wait on those. (we've discussed this in other posts)
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He compares to CML that is in MPN category wherein progression is rare with new meds.
Why aren't we like CML? Because there has been not great meds. But that is changing right now. The agents under study are focused on MF but we will bring them to earlier MPNs if/when they get approved.
Dr. Rampal: 20% MF, 7%PV, and 2% ET will progress to AML, but this cannot yet be individualized.
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Signs of progression:
-Changes in blood counts is top item, symptoms
-Night sweats, losing weight, just don't feel well, more fatigue than usual (defining fatigue is hard, can't get out of bed, being limited in activities are just part of disease. Can't climb stairs more likely anemia related.
-Keep up the regular Hem visits even if all feels well.
-He suggests may be good to start checking genetics every year or at least at some point on everyone.
-Lots of young get MPN and are more likely to die from it vs older just bec the old person (ie70 )will likely die from something else.
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-Italian study: Ropeg had some degree of less progression in young vs PLB, striking data. Only 100pts, stopped this study early bec of good results. (Stopping a study for this reason is a big deal. I am not aware of this study, if a member has it we'd love to see)
-Cornell study: Retro study Dr Rampal says be careful bec it's retro, but worth attention. I posted this in detail last year
healthunlocked.com/mpnvoice...
-Several pts of one participant had remission on INF, one INF patient had a bad recurrence after having a child.
-We need larger studies
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-Dr Rampal said mice studies show that the mutations cause MPN.
-Inflammation plays a role, but is it cause or effect (my take has been the marrow is the cause since SCT can be fully curative)
-Allele Burden (AB) used to be ignored, but that thought has changed and now Dr says it correlates to severity.
-AB alone does not mean progression, even if it relates to it. Progression requires more blood etc info.
-CALR has two types, type 1 is better, less prog risk.
-Since 2013 we know other (I think non-driver) mutations matter, and help Dx.
-One non-driver,"IDH2" is higher luek risk, and can cause leukemia by itself, but is the 1st MPN related mutation that is targetable by FDA approved luek med. (If your genetic profile has this, it seems actionable here)
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10% of population has an MPN mutation, usually Jak2, but not have blood cancer. (We've discussed in another post) Suggests something beyond just Jak2 is needed. Can we disengage the Jak2 from this other collaborating item?
-Crispr might be useful. (I wrote about this here in a reply) :
healthunlocked.com/mpnvoice...
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SCT: not too early, not too late. 20% mortality risk, hence the waiting.
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-Recent info shows Jak2 can occur decades before MPN.
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Genetic profiles should be covered by insurance as standard care.
Real-World Study Shows Promising Efficacy, Safety of Ropeg in MPN
Question for MPN patients in Canada
