Are There Treatment Options for Transfusion-Dependent MPN Patients?

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Topics include: Treatment

What therapies are available to the subset of myeloproliferative neoplasm (MPN) patients with low hemoglobin levels? Can the need for transfusions be reduced? A panel of MPN experts including Dr. Naval Daver, from The University of Texas MD Anderson Cancer Center, and Lindsey Lyle, from the University of Colorado Anschutz Medical Campus, discuss treatment options that may help stimulate red blood cell production and clinical trial research underway for patients with MPN-related anemia. 

Sponsored by Incyte Corporation. Produced in Partnership with the University of Colorado Anschutz Medical Campus.

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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:

A subset of patients with myelofibrosis has low hemoglobin levels and are transfusion dependent, so they can’t take ruxolitinib (Jakafi)—is what Chris said—are there other drugs currently available or in the pipeline for these patients? Do you want to talk about that Lindsey?

Lindsey Lyle:              

Sure. So, a few of the medications that we mentioned earlier such as erythropoietin, which can sometimes stimulate the production of red blood cells. That’s an injection. That may be effective if your erythropoietin level is low, and this can be tested in the blood. It can possible help with increased red blood cell production. 

Another therapy that we talked about briefly is called danazol (Danocrine), which is a very old medication, but it’s a testosterone-like medication that—it doesn’t work all of a sudden, but maybe over three months, can start to increase red blood cell production. Which may reduce the number of transfusions that they need. A therapy in clinical trial at MD Anderson right now is looking at anemia related to myelofibrosis, and every three weeks infusion…? 

So, that’s underway right now. That’s called AC-11 in the clinical trial world. So, that is currently being studied itself. But needing transfusions is not necessarily a contraindication to taking ruxolitinib, they can be done at the same time. And perhaps after some time on ruxolitinib, the hemoglobin levels, actually, we saw in clinical trial data, hemoglobin levels can rise, and sometimes reduce or eliminate the need for transfusions, even though at the on-set of starting Ruxolitinib it may increase the transfusion requirements for a while. 

Andrew Schorr:          

Okay, now Dr. Daver. This gentleman, Chris, also asked about whether there’s anything in the pipeline where it could alleviate this need for transfusions. 

Dr. Daver:                   

Yeah, so the standard one that we’ve been using for about the last 10 years has been erythropoietin stimulating agents and the common names are Procrit and Aranesp. They work in about 40 percent of patients with myelofibrosis or MDS associated anemia. So, we usually try those first. 

While we’re doing transfusions. The new drug that Lindsey mentioned, there is an agent called sotatercept, which is in clinical trials. And actually, in MDS may actually be close to approval, because they moved ahead a little bit earlier, but now myelofibrosis large Phase III are starting. So, if they darbpoetin alfa (Aranesp), epoetin alfa (Procrit) don’t work, I would try to get on one of those trials with one of the sotatercept-like drugs. And the third is, the Jakafi, I think, should be evaluated independently. 

So, if you have a large spleen, large symptoms, and large liver, et cetera, then you would benefit Jakafi. We often do add the Jakafi, a low dose, with growth factors. So, the determination for Jakafi is not based on anemia per se, it’s really based on whether the person would benefit from Jakafi. But those are probably the three. Then there are others like danazol, and lenalidomide (Revlimid), et cetera. But those are the three most commonly used ones. 

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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Page last updated on September 11, 2018