ASCO: You’re listening to a podcast from Cancer.Net. This cancer information website is produced by the American Society of Clinical Oncology, known as ASCO, the world’s leading professional organization for doctors who care for people with cancer. The purpose of this podcast is to educate and to inform. This is not a substitute for professional medical care and is not intended for use in the diagnosis or treatment of individual conditions. Guests on this podcast express their own opinions, experience, and conclusions. The mention of any product, service, organization, activity, or therapy should not be construed as an ASCO endorsement. Cancer research discussed in this podcast is ongoing, so the data described here may change as research progresses. In this podcast, Cancer.Net Associate Editor Dr. Guillermo Garcia-Manero will discuss some of the new research in leukemia and myelodysplastic syndromes, or MDS, that was presented at the 2018 American Society of Hematology Annual Meeting, held December first through fourth in San Diego, California. Dr. Garcia-Manero is the Deputy Chair of Translational Research and Professor, Department of Leukemia, at The University of Texas MD Anderson Cancer Center in Houston, Texas. He is also the Chief of the Section of Myelodysplastic Syndromes at MD Anderson Cancer Center. ASCO would like to thank Dr. Garcia-Manero for discussing this topic. Dr. Garcia-Manero: Hello, I'm Guillermo Garcia-Manero. I am 1 of the leukemia physicians at the University of Texas MD Anderson Cancer Center, where I also serve as the chief of the section of myelodysplastic syndromes. I'm happy to do this podcast today, trying to highlight a few of the studies that were presented at ASH this past December 2018. I have to start saying that this is basically mission impossible because the good news is there were so many important presentations, with active meaning for our patients, with new drugs, and that makes the selection of these 4 presentations extremely difficult. I'm going to mainly focus on presentations on myeloid malignancies, mainly MDS and AML. And I'm going to start with the first presentation. It's a very important phase III trial that was conducted all over the world, known as the MEDALIST trial. This study was, again, a phase III trial, was presented by Dr. List from the Moffitt Cancer Center, but it was a large effort, basically, countries all over the world. And the idea here was to test the activity of a compound known as luspatercept. This is a brand-new drug. This is an antibody that basically has the capacity to affect signaling of TGF-beta pathway in MDS. And the hypothesis is that by doing so, it may promote the capacity of the bone marrow to produce red cells and therefore improve or ameliorate anemia. As all of you know, anemia is a major complication of multiple disorders, in particular, myelodysplastic syndrome. So the study was a randomized study. The patient population was for a specific subset of patients with myelodysplastic syndrome that are known as patients with refractory anemia with ring sideroblasts. And these patients had received already prior therapy, mainly with a growth factor, drugs like erythropoietin. The study had what they call a 2:1 randomization, meaning that for each 3 patients, 2 got into the investigational compound, luspatercept, and 1 into placebo. This drug has already been tested on prior phase I, phase II studies and is extremely safe, and we know that we can administer this compound every 21 days. The major endpoints of the study were basically to see what was the rate of transfusion independency, and that actually is measured every 8 weeks. This is criteria that investigators in the field pick. And there were other endpoints, like 12 weeks, and then also measuring increases in hemoglobin and, importantly, of course, duration of response. This data was quite significant, and it's probably one of the first phase III trials for patients with myelodysplastic synd