Dr. Ruben Mesa provides an overview of available treatments and reviews important factors to consider when choosing a therapy for essential thrombocythemia (ET), polycythemia vera (PV) or myelofibrosis (MF).
Dr. Ruben Mesa is an international expert in the research and care of patients with myeloproliferative neoplasms (MPNs). He serves as director of UT Health San Antonio MD Anderson Cancer Center in San Antonio, Texas. More about this expert here.
Dr. Ruben Mesa:
The treatment landscape for myeloproliferative neoplasms is changing very rapidly. And in a good way, it’s increasingly having many more options for patients with Myeloproliferative Neoplasms. But I would separate it, really, into two groups. First, there are those individuals with essential thrombocythemia and polycythemia vera.
These individuals, we have newer therapies, such as interferons, we have, potentially, use of JAK inhibitors, we have some experimental therapies, as well as prior therapies we’ve used and become accustomed to, including hydroxyurea, phlebotomy, and aspirin.
But we’re learning much more about how to use these therapies; how to combine them; what constitutes success with these therapies; what should constitute a change in terms of therapy.
And there are new therapies being developed in the future that will impact this group of individuals with earlier MPNs: ET and PV.
For patients with myelofibrosis, the treatment is evolving. Patients with Myelofibrosis are affected in different ways. It is, in some ways, a more problematic disease.
There is evolution of our most impactful therapy, of stem cell transplantation. We have a better sense of in which patients we should consider that treatment, and how that can be applied in the safest way. We also have more medical treatments. We just saw in 2019 the approval of Fedratinib as the second specific JAK inhibitor approved for patients with myelofibrosis.
We, additionally, now have, truly dozens of clinical trials of new therapies in development that are in clinical trials right now that might be helpful for patients with myelofibrosis who have either had Ruxolitinib, or have a suboptimal response to Ruxolitinib, or sometimes even newly diagnosed patients. But I would say the future is very bright.
So, it is key with a treatment to first understand what is the treatment, what is the dose, and what is the goal? Each of the treatments have different goals. Some of the goals are to decrease the likelihood of blood clots or bleeding.
And frequently, we assess whether we’re protecting against the blood clots or bleeding by bringing down elevated counts. Is the plate account high, and we’re trying to bring it into the normal range? Is the hematocrit high, and we’re trying to bring that to under 45%? Is the white blood cell count high? Have we lowered each of those? First, it’s around controlling blood counts if that is the goal, as well as trying to decrease at risk of blood clots or bleeding.
Second, if patients have symptoms associated with their MPN, sometimes itching, sometimes symptoms associated with high courts, sometimes enlargement of the spleen, or symptoms associated with the spleen, have we reduced or nullified those symptoms? Have we shrunk the spleen if the spleen was enlarged?
And then, finally, we assess our goal by trying to be sure that patients are not progressing or getting worse on the disease. So, depending upon the treatment, we first asses what is our goal? Is it to improve counts? Is it to improve symptoms? Is it to shrink the spleen? And have we accomplished one, two, or all three of those goals? Or was only one those our goals to begin with?