While JAK2 inhibitors are approved for use in intermediate-2 or high-risk myelofibrosis (MF), there are inadequate treatments for patients in early disease stages. Ruben Mesa, MD, UT Health San Antonio MD Anderson Cancer Center, San Antonio, TX, discusses the results of a pilot study of ropeginterferon, an inferon alpha cytokine therapy, in MF patients. Ropeginterferon was trialed in cohorts of early disease and advanced disease patients, based on a reduced clonal burden seen in chronic myelogenous leukemia and polycythemia vera with the use of cytokine therapy. The results show that the treatment was well-tolerated and may be of clinical benefit to MF patients. These initial findings support the ongoing investigation into ropeginterferon in myelofibrosis. This interview took place during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 2020.
Transcript (edited for clarity)
This was a study done as a pilot study with my colleagues at the Mayo Clinic in Arizona, and that, after I transitioned to San Antonio, was taken over by my good friend and colleague Dr. Jeanne Palmer. And we were looking at the impact of ropegylated interferon, for patients with early myelofibrosis. Indeed, I’m very excited about ropegylated interferon, it’s clearly had very favorable data in PV and may become approved on that basis...
This was a study done as a pilot study with my colleagues at the Mayo Clinic in Arizona, and that, after I transitioned to San Antonio, was taken over by my good friend and colleague Dr. Jeanne Palmer. And we were looking at the impact of ropegylated interferon, for patients with early myelofibrosis. Indeed, I’m very excited about ropegylated interferon, it’s clearly had very favorable data in PV and may become approved on that basis. Dr. Srdan Verstovsek and I are co-leading the global Phase III study of ropegylated interferon as second line in ET.
So it’s natural to ask the question whether this therapy might be beneficial for patients with early myelofibrosis. It’s previously been shown both by the French and Dr. Dick Silver, that there is activity of interferon in myelofibrosis, particularly if used in more early disease. Individuals with very advanced splenomegaly, very fibrotic bone marrows, marked cytopenias, probably not the ideal group, so much earlier myelofibrosis.
So in this pilot study of 10 patients, where we were really looking both for tolerability and activity, we saw one, the drug was well tolerated with a mean follow-up now of almost three years for these individuals. We had, even though many of these were low- or intermediate-risk individuals in the low-risk cohort, there were three individuals that had benefit out of the three. In the intermediate- and high-risk cohort, we saw a clinical improvement in 30%, a stable disease in the vast majority, 57%, one patient progressed.
Recording progression-free survival was part of our goal in that therapy and in that aim overall very successful. Patients received up to 47 cycles on this particular pilot study. And now overall we view it as a very favorable pilot study, showing safety and efficacy for this population. Clearly would need larger studies, randomized, potentially against other standard approach or observation. The goal with the eligibility for this study was not individuals who normally would have been treated with a JAK inhibitor, but these are individuals who otherwise would have normally been observed. But we were, again, looking for activity and progression-free survival.
But excited to see that and feel that a ropegylated interferon likely will play a role in early MF. It may also be a natural fit, of course, with people in ET or PV who have evidence of moving toward fibrosis.