Trial result of Peginterferon combined with Ruxo. Worked especially well for early stage MF patients although side effects/20% discontinuation rate a concern. Remember Ropeg is around the corner which should reduce Interferon related side effects.
“Follow‐up was 12 months. Partial remission (PR) and sustained CHR were achieved in 9% and 44% of PV patients, respectively. In MF patients, complete or partial remission was achieved in 39%, and sustained CHR in 58%. The median JAK2V617F allele burden declined significantly in both groups. Hematologic toxicity was the most common adverse event and was managed by dose reduction. Thirty‐seven serious adverse events were recorded in 23 patients; the discontinuation rate was 20%.”
While it some instances this might appear on the surface to be positive news, it also seemed to imply that those of us with the CALR mutation might not fare so well as some of the others.
One other matter that might also bear our notice is that a large percentage of the authors of this article are or were funded by Novartis, who are of course the manufacturers of Ruxolitinib, and therefore one might deduce ulterior persuasiveness might be at work here – Conflict of Interest?
The article makes the following declaration:
CONFLICT OF INTEREST DISCLOSURE
–
Stine Ulrik Mikkelsen and Niels Pallisgaard have received a
travel grant from Novartis Oncology. Hans Carl Hasselbalch,
Claus Henrik Nielsen, Niels Pallisgaard, and Mads Emil Bjørn
have received research funding from Novartis Oncology.
Niels Pallisgaard is in the Novartis Speakers Bureau. Ole
Weis Bjerrum and Daniel El Fassi have conducted educational
activities for Novartis. All other authors have no relationships
to disclose.
Novartis:
"Novartis International AG is a Swiss multinational pharmaceutical company based in Basel, Switzerland. It is one of the largest pharmaceutical companies by both market capitalization and sales." Wikipedia
Of course this does not necessarily indicate that all of their findings are false, however, we should still be informed...
Thanks Steve, you make a very good point and I think this applies to many trials and is unavoidable. I’d like to think that Hems as reputable as Dr Hasslebalch would ensure that results are as clinically accurate as possible.
Worth studying in detail. Most of the 50 had been on Interferon before this trial and had problems. Hence it appears the Ruxo/Peg combo, overall, is better tolerated than sole use of Interferon?
I assume doses will be adjusted for Phase 3 and Ropeg appears superior to Peg (fingers crossed) and could be available to us next year.
Re CALR, it seems to me that there is no ‘one size fits all drug’. We are have slightly different mutations/genetics and possibly different drivers. It’s a question of finding which drug works best for you.
On the CALR front... we may be waiting a tad longer it seems. Here in Oz we are also still waiting for Peg' to become available via the PBS. Most reports concur that it is a better product than Interferon Alpha... we shall see I guess.
Yes, or something very similar not so long ago. It implies that the relationship between CALR mutations and MPL might lead to discoveries in genetic manipulation, so still a ways off as yet.
And in answer to a previous question about SCT, I am still awaiting a MUD (matched unrelated donor). My specialist still prefers that I involve my siblings and I personally am not convinced that there is a real need to do so...
How is your possible SCT progressing? My new local Hem a SCT expert, he said survival rates now 90% for related donor, 80 - 85% for unrelated. He’s just successfully SCT’d a 75 year old.
Evidently there have been no quantum treatment breakthroughs, just slowly getting better all the time. GvHD looks to be where the most short term progress being made?
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INCA033989 phase 1 clinical trial is recruiting patients 
Prefibrotic MF
ET & MF 
