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Is the Use of a JAK inhibitor Prior to Stem Cell Transplant Recommended?

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Published on April 26, 2016

Is there a positive correlation between the use of a JAK Inhibitor and stem cell transplant (SCT)? UK viewer, Peter, asks about the recommended use of a JAK inhibitor prior to having a SCT. MPN expert Dr. Naveen Pemmaraju from MD Anderson Cancer Center responds with a discussion of two studies on this subject and the role of SCT for MPN patients.

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Transcript | Is the Use of a JAK inhibitor Prior to Stem Cell Transplant Recommended?

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: 

Peter writes in from England, from the UK.  He says, “I understand that there is a view that the preferred treatment for myelofibrosis is ruxolitinib or Jakafi before stem cell transplant or rather than going straight to transplant.  Is that correct?” 

Dr. Pemmaraju: 

Wow—another outstanding question.  Unfortunately, there’s no randomized large data trial or otherwise to answer that question.  It is one of the fundamental questions. So I have to say that we don’t yet know, but there are two studies I can point to.  One is a retrospective study that was done that was just published recently.  That study did 100 consecutive patients.  It was a multi-center study.  My colleague, Dr. Verstovsek is one of the co-authors.  

 

In this study it was shown—it was retrospective, which means that the investigators went back in time to look. But in patients who have intermediate to high-risk myelofibrosis, it was found that stem cell transplant benefited this group of patients, meaning that their outcomes were thought to be better than they would have been with traditional therapy alone. 

Importantly, in that study, the investigators specifically looked at patients who’d had JAK inhibitor therapy, so it’s really one of the only large studies of its kind.  So that is reassuring, and that is in line with exactly what our questioner is asking. 

A second study is a pretty interesting one.  It was done by my colleague, Dr. Uday Popat, who is a researcher here at MD Anderson.  He’s a transplant doctor.  And this is not yet published, but it was presented at ASCO last year, one of our big conferences.  In that study, it was approximately 50 patients.  The median age was under the age of 60, so these were younger, fairly healthy myelofibrosis patients. But in that study with a very specific conditioning regimen, our group showed that again the same thing, that stem cell transplant, allogeneic transplant did benefit patients.  Now, this is follow-up at three years, so that means looking at overall survival disease response.  And again, this is a high-risk group of patients.

It’s important because of two reasons: one, there are no curative medical or medication therapies for myelofibrosis, so that’s important; and two, allogeneic stem cell transplant is still in 2016 the only curative option.  So coupling that you would think, hey, there should be a lot of data but there isn’t yet.  So we do believe that there is a role for allogeneic stem cell transplant, possibly in the younger, fitter patient, also possibly in someone who has intermediate to high-risk for disease but also in the older patient with myelofibrosis, possibly with reduced intensity conditioning and all that.  So I think this is an important question. 

As far as the timing of the JAK inhibitor, not only do we not know the optimal timing—although these studies have shown that hey, yes you can do well with a JAK inhibitor up until the time of the transplant.  I would raise two other questions.  One would be what about the role of the JAK inhibitor after transplant?  We don’t know, but studies are being launched for that.  And two is, what about the timing of these things?  How closely to the time of the transplant and how quickly after?  So let those be the two big research questions over the next five years, but yes there is a role for allogeneic transplant still in myelofibrosis.

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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