American Society of Hematology 2020 Podcast Collection: MPN

Abstract

David Gray (DG): Managing Editor of Advances in Therapy Massimo Breccia (MB): Policlinico Umberto I-Department of Translational and Precision Medicine, Sapienza University of Rome DG: Hello, and welcome to the Adis Rapid? podcast series. We’re bringing you a selection of podcasts covering the American Society of Hematology 2020 Conference, discussing the highlights of the data released at the conference. Today’s podcast will be focusing on myeloproliferative neoplasms and the data presented at the ASH conference. Speaking to us today is Dr. Massimo Breccia, Department of Translational and Precision Medicine at the Sapienza University of Rome. Massimo, welcome to today’s podcast, and thank you so much for speaking with us. You’re going to talk us through some of your top highlights of the MPN [myeloproliferative neoplasm] data from ASH 2020. A lot of important data was released, so please, get us started, and tell us what your first highlight was. MB: Thank you very much, David, for your kind invitation. So we can subdivide the presentation according to the disease; in particular, the first three for myelofibrosis. And the first one is the final analysis of the EXPAND trial [1]. As you know, the current starting dose of ruxolitinib for patients with myelofibrosis but with low platelet count is 5 mg BID. But evidence from the previous study that was called 258 showed that a titrated dose of 10 mg BID could be safe and effective. So the EXPAND trial tested this dose of 10 mg BID in 69 patients that were subdivided in two different cohorts. So one, the first one, with blood counts from 75 to 100,000, and the second one, indeed, from 50 to 75,000. So at first, 50% and 83% of patients discontinued the study drug, respectively. In the first cohort, the main reason was progression of disease; indeed, in the second cohort, the appearance of adverse events. So in terms of results, 40% and 38% of patients achieved a spleen reduction higher than 35% at 24 weeks, respectively. And 31% and 40% indeed improved their symptoms, measured by the total symptom score at 24 weeks, respectively. The pharmacokinetic analysis showed that it is similar to that of patients treated with ruxolitinib that had higher platelet count. So the results strongly suggest that the dose of 10 mg Supplementary Information The online version contains supplementary material available at https:// doi.org/10.1007/s12325-021-01665-2.