Disease Characteristics in MDS

Transcript:Thomas Prebet, MD, PhD: If I’m to draw the portrait of the classical patient that we have to see in consultation for myelodysplastic syndromes, it’s mostly someone between 70 and 80 years of age. And most of the patients are coming in because of anemia-related symptoms. Anemia is a diagnosis symptom in 60% to 70% of the cases. So, it’s really what’s driving the patient to go to clinic and to have some more tests done. Anemia is a common feature in elderly patients. Myelodysplastic syndromes is less frequent than other causes of anemia, but we see more and more patients with a diagnosis of myelodysplastic syndromes.

When we talk about the population of myelodysplastic syndromes, basically there are some common features between low-risk and high-risk disease, but also some big differences. The main characteristic that we need to keep in mind when we talk about myelodysplastic syndromes, is that it’s mostly a situation that we have to face for elderly patients, meaning age 75 years. There’s a group of patients which are therapy-related myeloid neoplasms. It’s mostly younger patients that have been exposed to genetic insults such as chemotherapy, radiation therapy. But it’s undersized compared to the most common and general population of de novo myelodysplastic syndromes, where we have this elderly population.

As I mentioned, we basically have two big groups of patients, the low-risk population, which represents something like 70% to 75% of the new diagnoses of myelodysplastic syndromes, and 20% to 25% are the high-risk patients. In low-risk patients, in this case, we need to consider myelodysplastic syndromes as a chronic disease like we consider diabetes or hypertension. It will be a chronically ill patient where a main issue will be to deal with cytopenias. And, in that case, patients sometimes usually have increased needs for transfusion, mostly anemia and red blood cell transfusion. And that’s what drives the prognosis. Dealing with cytopenias and the consequences, including iron overload, for example, are the main drivers of prognosis for this low-risk patient.

Transformation and evolution to leukemia in that case is less frequent. That’s what we’d call lower risk, in fact, and it’s not driving the prognosis. Most of the patients in that case will have some issues and potentially at the end die because of the consequences of transfusions and the cytopenias.

When we talk after that about the high-risk population, the situation is completely different. These patients more or less behave like acute myeloid leukemia patients. They have cytopenias, but the evolution of the disease is really driven by the proliferation of cells, in that case, and by the potential evolution to acute myeloid leukemia. So, the type of approach that we have to deal with is completely different as compared to the lower-risk patient, where we can expect a median overall survival of 8, 9, 10 years. While with myelodysplastic syndromes with high-risk features, we can only expect, without treatment, median survival of 1 or 2 years in some cases.

Transcript Edited for Clarity