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MPN Workshop of the Carolinas 2025 | An update on BET inhibition for the treatment of myelofibrosis
In this video, Lucia Masarova, MD, The University of Texas MD Anderson Cancer Center, Houston, TX, provides an update on the development of bromodomain and extra-terminal motif (BET) inhibitors for the treatment of myelofibrosis (MF), highlighting the need to redefine endpoints and identify patient subtypes that benefit from combination therapy. This interview took place at the 2nd Annual MPN Workshop of the Carolinas, held in Charlotte, NC.
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Transcript
The bromodomain inhibitors are kind of a hot potato in the myelofibrosis field, especially after we’ve seen the kind of disappointing data from the regulatory perspective, of the phase three randomized studies that were using ruxolitinib-pelabresib versus ruxolitinib placebo, where we had a tremendous improvement in the spleen size, but not so in the symptoms...
The bromodomain inhibitors are kind of a hot potato in the myelofibrosis field, especially after we’ve seen the kind of disappointing data from the regulatory perspective, of the phase three randomized studies that were using ruxolitinib-pelabresib versus ruxolitinib placebo, where we had a tremendous improvement in the spleen size, but not so in the symptoms. And now, ever since then, there has been a little bit of changes on how do we assess the symptoms? Do we use the standard 50% improvement? Do we use the total symptom score changes? Do we look only at subtypes of patients with different degrees of symptoms? Do we look at symptoms in correlation with other needs of these patients? All of these things, I think, are going to be dissected and looked at.
The new data were coming with the mature 72 weeks follow-up of that frontline study that kind of reinforced the superior efficacy for the spleen and durability of the combination versus just a single agent. There were no more concerning AML or progression to acute leukemia signals that kind of add up with the single agent, so that turned out to be more heavy.
We’ll see what the next development brings us in terms of do we need to do a new study. I think that’s been an ongoing effort to, okay, how this is going to go, how it’s going to look, so there is some movement behind the clock that we don’t see, but I think the most important thing would be to answer who are these patients that could benefit from the combination and how do we best assess their benefit, so that is the most important question that I think came from this development that we all learn that we actually have to realize not everybody needs a frontline therapy as a combination, at least not with the current and established endpoints that we have.
I would say maybe, and probably everybody would need some kind of combination to really go faster on the disease clone and stop the progression. But we have to figure out what it is, how to assess it, and when to use it. I think that’s the most important thing from the development. Whether the next study takes us to having the drugs approved or not is really very important for patients and clinicians. But along that, I think we have to learn who would be the best fit and how to use these agents to really direct the best benefit and plus how to continue developing new drugs in this field which is rare but very needed.
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