ASH 2024 | The evolving treatment landscape for patients with polycythemia vera

Polycythemia vera is a disease that we have been using a lot of the same treatments for many years and the goal of the treatment is really to, right now the goal of the treatment is to decrease the risk of thrombotic events that these patients are at high risk for. And historically what we’ve been doing is really trying to control the hematocrit which is done by phlebotomies as well as cytoreductive therapies for those patients that are considered at high risk. And we’ve been using in a way the same drugs for many years. So hydroxyurea is a drug that we commonly use. It’s been used for decades. It’s very well known. It works well but it is a chemotherapeutic agent and it has its own issues. In addition for many years we’ve been using a drug called interferon and over the years we’ve had many different or not many but a few different formulations. And so the one that is currently FDA approved and is probably more used most often currently is something called ropeginterferon. And it has been shown to do a good job of controlling hematocrit as well as achieving complete hematologic responses meaning controlling all blood counts as well as many of the symptoms of PV. And importantly, it can bring down the amount of abnormal JAK2, so the JAK2 variant allele frequency. Also has its own potential side effects, of course, as many drugs do. And then the other, really the only other drug that we use in polycythemia vera is a JAK inhibitor ruxolitinib, currently approved as a second-line therapy after hydroxyurea, but commonly used, is very effective in controlling patient symptoms, especially symptoms like itching and fatigue, but also of course controls the hematocrit in many patients and allows patients to achieve complete hematologic remissions as well. And with this drug also has been shown most recently in the MAJIC-PV trial is that, especially as patients remain on the drug for many years, they do achieve molecular responses where we see that JAK2 allele burden goes down. It has been correlated with event-free survival in these patients. So these are sort of the landscape that we currently have. What we’re really trying to get to for polycythemia vera is we need drugs that are clearly more disease-modifying, meaning that in addition to controlling the blood counts and helping with the symptoms and decreasing the risk of thrombosis, we also want drugs that will clearly decrease progression of disease to later stages of disease, such as myelofibrosis or acute leukemia. And then in many patients, the symptoms of PV and the treatments of PV that we offer them can be very difficult to tolerate. And so we need medications that allow us to help with those issues. So these drugs that are affecting the hepcidin pathway potentially could really improve patients’ ability to improve their quality of life because of this decreased need for therapeutic phlebotomies.

This transcript is AI-generated. While we strive for accuracy, please verify this copy with the video.