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Are New MPN Medicines Improving Survival?

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Published on May 25, 2013

Relieving symptoms is significant when it comes to debilitating illnesses like myelofibrosis. But can pain relief lead to longer life? Besides relieving symptoms, it may be possible that new medicines may have a positive effect on the bone marrow where the disease starts. Dr. Claire Harrison gives her perspective on the many ways these new medications are helping patients with myeloproliferative disorders. Watch now.

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Transcript | Are New MPN Medicines Improving Survival?

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Andrew Schorr:

Hello.  I'm Andrew Schorr for Patient Power. 

All of us living with a myeloproliferative condition want to live better of course, but we also want to live longer.  Where are we now with understanding whether a new JAK2 inhibitor, ruxolitinib (Jakafi and Jakavi), is actually extending our survival?  We want to get the perspective of an expert. 

Joining us now is Dr. Claire Harrison.  She is from Guys' and St. Thomas' Hospital in London.  She's attending a medical conference, European Focus on Myeloproliferative Neoplasms.  Dr. Harrison, what's the evidence now about survival? 

Dr. Harrison:

It's my belief that the strength of evidence supporting survival advantage for patients who respond to JAK inhibitors and who receive them is growing.  We had the initial message from COMFORT 1, which was the randomized study with ruxolitinib (Jakafi and Jakavi) compared to placebo, that patients, early in that study, had a survival advantage compared to those who were receiving placebo, even though the placebo patients crossed over. 

In the COMFORT 2 study the situation was a little bit different.  Here we were comparing ruxolitinib, Jakafi, to best available therapy, which for most patients was active therapy.  We were randomizing patients, two to receive ruxolitinib (Jakafi and Jakavi), one to receive standard therapy, we also recruited a smaller number of patients.  What is the status of the survival message from COMFORT 2, has been a question that's been asked a lot, and I'm happy to be able to tell you that we have shown a survival benefit for patients treated with Jack inhibitor, Jakafi, Jakavi, in this study. 

Why did we show it later?  So, I think that the likelihood here is a complicated story.  First of all, we had many patients in the control arm of the study who didn't continue in the study and who we didn't follow for survival, initially.  We're doing some work to go back to those patients, or their families, to say, can we enter this data.  We have some patients who look like they live still or might look for in perpetuity that they're alive in the study, and we're working hard to fix that. 

Secondly, we can't be sure that standard therapies might not add a little to survival benefit. 

And, thirdly, we were recruiting far more patients to the active arm of that study, to the Jakafi arm.  That means that, statistically, it's likely that a later benefit would be seen.  However, the survival benefit now from COMFORT 2, from COMFORT 1, from the MD Anderson cohort of patients treated in the Phase 1/2 study is very consistent, and that's very reassuring.  The message, in particular, from COMFORT 2 is becoming very important for reimbursement, because they want to know the benefit over standard therapy, so we do see this with this drug.  We do not yet see cure. 

We do have some very exciting patients who are having very good resolution of abnormal bone marrow findings.  I suspect we'll be showing that data soon.  Might we see this with other agents?  I suspect we probably will, but we can't be certain until we actually have the data from properly conducted clinical studies.  The reason for that is that it's very important to try to ensure balance, to not look back at historical cohorts of patients, because we know that, actually, studying patients over time, that survival is improving but too slowly for many of us for patients with this disease. 

Andrew Schorr:

Dr. Claire Harrison, thank you so much for being with us on Patient Power. 

Dr. Harrison:

Thank you. 

Andrew Schorr:

We'll have more with Dr. Harrison as she takes us through the unanswered scientific questions about the understanding and treatment of MPNs. 

I'm Andrew Schorr.  Remember, knowledge can be the best medicine of all.

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

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