Transcript:James M. Foran, MD, FRCPC: How do you make a decision regarding a treatment path in a newly diagnosed patient? Is it on the IPSS or is it on those parameters? Is that how you do it?

Azra Raza, MD: I have to say that IPSS and the WHO classification systems are all very useful, but the most useful thing is that man must remain the measure of all things. And in the final analysis, it’s the patient that counts. How is the patient looking? What is the performance status? What are their other comorbidities? What are the goals of treatment for this patient? Is it to improve survival? Is it to improve the quality of life for the patient? What does the family want us to do? Because it is a disease of the elderly, as you know.

James M. Foran, MD, FRCPC: That’s a lost point in modern medicine, which is you still have to be a doctor, and I think that’s a really critical point. You still have to lay hands upon the patient and make a decision and work with them one-by-one. Well, I’m glad you said that. And do you decide early on about allogeneic transplantation or treatments for cure? How do you approach that?

Azra Raza, MD: This is a very, very critical question, because we all know that the only potentially curative measure for this very frustrating disease is in an allogeneic stem cell transplant. And every single patient must be considered for a transplant. The question I always ask myself and pose to the patient is, ‘Look, the only cure is an allogeneic transplant, but the question we have to ask is, what is more dangerous for you? Is the disease going to kill you or the treatment going to kill you?’ Well, for low-risk myelodysplastic syndrome, the disease can go on sometimes for 20 years; it’s not going to kill the patient. But if we try to cure the patient today with a bone marrow transplant or a stem cell transplant, we are more likely to kill the patient.

So it’s like bringing a tank to kill a mosquito. I always address this question immediately with a patient at diagnosis, but then really pursue it for those high-risk patients whose disease seems to be progressing fast. And then, of course, the age is the first thing, and then comorbidities. What is the patient’s performance status? Do they have a match or not? Do they have a donor available or not? This is all considered for those patients who really are at high risk for whom we know that treatment may not produce a substantial and a durable remission of the disease.

James M. Foran, MD, FRCPC: By the way, I very much like that point. It sounds like in your practice, this is an evolving discussion and an evolving intervention, which makes a lot of sense to me and certainly mirrors what we do. But how do you decide when somebody first comes in? Let’s say they come in with a symptomatic anemia or other cytopenias associated with that. How do you go through and decide whether you’re going to start them on an erythropoietin stimulating agent or on systemic therapy? How do you do that?

Azra Raza, MD: So, if I think that their anemia is profound enough to be symptomatic and we need to help them, then the first thing we do is measure the serum erythropoietin level. And if the serum EPO level is below 500 and they haven’t received any transfusions—but the hemoglobin is trending down, and it’s 9 grams and now it’s falling even below 9 grams occasionally—then I think, given that the serum EPO is low and they haven’t received transfusions, their chance of response to an erythroid stimulating agent is about 70%. So it’s very reasonable to choose such a patient for therapy with these agents.

James M. Foran, MD, FRCPC: Do you add Neupogen (filgrastim)? Is that something you do?

Azra Raza, MD: Generally, patients with RARS, refractory anemia with ringed sideroblasts, are the ones in the lower-risk category who don’t respond so well to erythropoietin alone. And so for those patients, I will add filgrastim. But first, I try the single agent ESA.

James M. Foran, MD, FRCPC: That makes sense. That’s also a common practice that we’ve pursued, and I’ve seen others pursue also. But I’m hearing you;. I’m reading between the lines. I’m hearing you also say that we have to read the pathology report. We can’t just look at the blast count. We can’t just look at the cytogenetics. We really need to look at the pathology report and see if it’s RARS or one of the ring sideroblast variants. And that sometimes gets lost in those pathology reports. I’m glad you brought that out.

Transcript Edited for Clarity