Management and Treatment of Essential Thrombocythemia


Lucia Masarova, MD, discusses available treatment options for patients with essential thrombocythemia.

Lucia Masarova, MD, an assistant professor in the Department of Leukemia in the Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, discusses available treatment options for patients with essential thrombocythemia (ET).

Here, Masarova highlights interferons for patients with myeloproliferative neoplasms, such as ET or polycythemia vera (PV), as well as previous and ongoing research regarding peginterferon alfa-2a (Pegasys) and ropeginterferon alfa-2b (Besremi) in this space.


0:08 | ET is considered a benign disease, so people live with it for a very long time. The current treatment is for only those [with] high-risk disease, so patients that had thrombosis in the past, and then patients that are over the age of 60 years and have a JAK2 mutation. That is according to the revised IPSET score. Those patients are treated to decrease the risk of thrombosis, not to do anything else or disease modify, just to simply disease the thrombosis risk. So that's hydroxyurea. It's a standard frontline agent, which is an easy to take oral drug. However, it could lead to some resistance in about 30% of patients.

0:56 | Because ET also affects younger people, plenty of younger females, they don't want to take chemotherapy forever, which I don't blame him for. They are interested in taking something else. So then interferon, the standard interferon recombinant or the pegylated form, which is called [peginterferon alfa-2a]. We recently used [peginterferon alfa-2a] for a couple of years, and it has been around the MPNs, particularly in ET and PV, I will say for about 40 years almost. So a lot of data, a lot of implications about how this may be a disease modifying agent, which is an agent that actually could decrease the allele burden, could eradicate the malignant clone, and ultimately change the disease behavior.

1:44 | We were really excited that we had a novel interferon called ropeginterferon interferon approved in patients with PV back in 2021. That has been preceded by approval in the European Union before, in 2019. That's an excellent option in terms of interferon, subcutaneous items. Patients will have to inject themselves and the pegylated is once a week. The standard recombinant is more than weekly, so the novel alternative way, the ropeginterferon which is the novel monoregulated form, is kind of more convenient because it's used every other week. Once we reach complete hematologic control, it's used once a month. So that's a significant improvement in convenience for patients and tolerability, as well as compliance issues. We were excited to get it in PV. We have it in some studies in PV to expand the access and maybe try some different schedules, and will also move it to patients [with ET]. That's something that we're going to be looking at

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