What Is the Current State of MDS Treatment, Care?

A roundtable discussion, moderated by Hana Safah, MD, of the Tulane University School of Medicine, focused on the latest data in myelodysplastic syndromes presented at the 65th ASH Annual Meeting & Exposition. Dr. Safah was joined by Jamile Shammo, MD; Andrew Brunner, MD; and Tiffany Tanaka, MD.

In the first segment of the roundtable series, Dr. Safah gave an overview of myelodysplastic syndromes and addressed the current state of patient treatment and care.

Watch the next segment in this series.

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Dr. Safah: To start our discussion, I’m going to go with an overview about myelodysplastic syndromes (MDS) that is now being also recognized as a myeloid neoplasm by the WHO [World Health Organization]. Myelodysplastic syndromes is an acquired disease where patient’s bone marrow usually shows evidence of dysplasia, ineffective erythropoiesis, resulting in cytopenias. When we speak about cytopenias, we’re talking about leukopenia, anemia, and thrombocytopenia. Patients usually present with different phenotypes of the disease, so it’s a heterogeneous group of diseases. Usually at presentation we make the diagnosis by bone marrow biopsies, doing the karyotyping and molecular studies, and it’s becoming very important to do the molecular studies early on to detect all these genetic mutations and alterations that can affect the classification of the disease.

Patients with MDS, when they present with cytopenias, usually need what we call supportive therapy for the cytopenias related to disease, whether the anemia or the leukopenia or thrombocytopenia. However, if they have a higher-risk disease, or what we call intermediate or high-risk disease, we do treat these patients with HMAs [hypomethylating agents]. Having said that, we know at this point that there is no curative treatment other than bone marrow transplantation. Some patients need to be referred to a stem cell transplant program to be evaluated for a stem cell transplant. When we talk about patients with MDS, we’re talking about an elderly population. The median age is around 71 years of age, and when we focus on patients with low-risk disease, we’re focusing on according to the IPSS-R [Revised International Prognostic Scoring System]. These are patients who have very low risk, low risk, and intermediate.

These patients usually have an expectation, a median overall survival of five to six years. They usually comprise the majority of the patients in our clinical practice; 77% of the patients are usually at presentation belong to the low-risk MDS. Having said all that, the median overall survival, talking about the anemia in that elderly population with a median age of 71 years of age, anemia becomes a chronic disease in these patients who usually suffer from multiple other diseases and other comorbidities. As such, they end up with decreased quality of life and complications from the transfusion if they become transfusion dependent.

The expectation is that 40% of patients who present with anemia are going to end up with the need for transfusion or transfusion dependent. These patients not only suffer from shortened overall survival, to start with, but as I said, the need for transfusion does add a burden on their health and quality of life.