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MPN Workshop of the Carolinas 2024 | Results of the RUXOPEG trial: combining ruxolitinib and interferon in patients with myelofibrosis

Jean-Jacques Kiladjian, MD, PhD, Saint-Louis Hospital & Paris Diderot University, Paris, France, outlines the findings of the RUXOPEG trial (NCT02742324), which investigated the combination of the JAK inhibitor ruxolitinib with interferon in patients with myelofibrosis (MF). This combination approach was tolerable, showed encouraging clinical efficacy, and led to previously unseen levels of molecular response in patients. This interview took place at the 1st Annual MPN Workshop of the Carolinas in Asheville, NC.

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Transcript

In the study we combine interferon to ruxolitinib, the idea being that interferon is a very active drug to target stem cells in these diseases, but is often not well tolerated, especially in patients with myelofibrosis. And on the other hand, we have ruxolitinib, a drug that is very efficient on symptoms and inflammation. And the idea was to combine both drugs to have the benefit of both...

In the study we combine interferon to ruxolitinib, the idea being that interferon is a very active drug to target stem cells in these diseases, but is often not well tolerated, especially in patients with myelofibrosis. And on the other hand, we have ruxolitinib, a drug that is very efficient on symptoms and inflammation. And the idea was to combine both drugs to have the benefit of both. And indeed, we treated a total of 37 patients in this study with various dose combinations.

And the overall result is that the combination provided a very nice clinical response in terms of reduction of the spleen size by more than 50%, that was reached in almost 70% of the patients in this study. Toxicity was mainly hematological with anemia thrombocytopenia that sometimes led to dose reduction, but overall it was very well tolerated and very efficient.

Another very important point is that we could see very nice molecular responses in terms of decrease of the JAK2 VF mutation that was never achieved before at such level in myelofibrosis patients, for example, we started with an average of 84% of mutation in these patients down to less than 55% after 12 months of treatment.

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