Educating Patients on MPN Disease Progression


CURE spoke with Dr. Rajiit Rampal, on behalf of the MPN Research Foundation, about disease progression in myeloproliferative neoplasms.

Kristie L. Kahl: Why is it important for patients to be aware of disease progression, especially in MPN?

Dr. Rajiit Rampal: Well, you know, I think one of the things we worry about is disease progression and let's just take a step back and define what we're what we mean by that, you know, patients with essential thrombocythemia or polycythemia vera, we worry about disease progressing into myelofibrosis, or potentially but much less likely acute myeloid leukemia. with patients in myelofibrosis, we worry about disease progression, meaning a couple of different things, that can mean that the symptoms are getting worse, that can mean that their spleen or liver is getting more large, that can mean that they are having to now need transfusions of things like red blood cells. And that can mean that the disease is progressing to leukemia. So progression encompasses a number of different things, I should take a step back and say that symptom progression can also occur both in ET and PV. So when we talk about progression, it encompasses all of those things. Now, why is that important? Right? I think that, you know, fundamentally, if we're talking about symptom progression, those are things we want to try to intervene on as best we can to improve our patient's quality of life. If it is a disease progression in terms of, as an example myelofibrosis, coming to a point where now a patient needs a blood transfusion, or their spleen is come to a point where it's very symptomatic and troublesome, those are indications for us that it's time to intervene with treatment, if we've had a patient, for example, who is under observation, if these events start to occur, that's often an indicator that it's time to start treatment. And in many cases, that may also be the trigger for us to say it's time to think about a stem cell transplant if that if the patient's a candidate for that.

Kristie L. Kahl: How often are patients with MPNs monitored?

Dr. Rajiit Rampal: Well, I think it depends, I think, you know, what I can say, from our perspective and our practice here, we tend to see our MPN patients roughly every three months. And now you know, of course, it depends on the patient's disease and how they're doing. And patients who have maybe more advanced myelofibrosis, it may be every week or every month that we're seeing them. And in some of our essential thrombocythemia patients who are low risk younger patients who are doing clinically Well, sometimes it's every six months. So it's a range, depending on the individual patient.

Kristie L. Kahl: And are there signs of disease progression that patients should be aware of?

Dr. Rajiit Rampal: Yeah, I think, you know, the, the symptoms that I've talked about, are really the main indicator from the patient's perspective, but you know, also their bloodwork, I know, our patients are highly educated and their disease well versed in their disease, and so they look at their blood counts. And you know, what, what I would say to patients is that we look for trends, we don't look for values that maybe anomalous one time, right? A patient can have a blood count, check. And, you know, today, their, their white count is a little bit higher. And the next reading, it's, it's back to normal, what we look for really are trends. And, you know, in somebody with metal fibrosis, we're looking to see, is the hemoglobin going down? Is it trending down over time? Are the platelets trending down over time? Or in somebody with PV? Are we keeping their inadequate, consistent and controlled? Or is it you know, spiking to very high levels? So trends and blood counts are also things that I think our patients should know about.

Kristie L. Kahl: what is the importance of risk stratification in identifying patients with progressive disease?

Dr. Rajiit Rampal: The stratification tools help us in a couple of ways. So you know, the tools that we use for polycythemia vera and essential thrombocythemia are similar. And they're the risk stratification essentially tells us is the patientat a relatively higher or relatively lower risk of having a clot. And that's clotting is really what we were about an 18 PV, those risk scoring tools really don't tell us too much about the chance of the disease progressing, but it tells us relatively what's the risk of clotting, and that informs our decision right now into what we do. Then people who fall into the higher risk category, we tend to put them on medication to control their accounts. And in the lower risk category, we tend to just treat them with either aspirin or phlebotomy, whatever it is, I think we are coming to a point where we're trying to develop models of what's the risk of progression and at MPV and, you know, there's a number of molecular studies that are addressing that that point and have given us at least some guidance into how to think about the likelihood of progression. In myelofibrosis. It's a little bit different because the risk scoring tools there are really telling us what is the likelihood of the patient's disease progressing not you know, it's not talking about a singular event, but what's the likelihood of the disease getting worse progressing, becoming leukemia, and what's the patient's chance of survival and that has different implications in In myelofibrosis, those tools don't tell us how to treat the patient they serve as is essentially a guard rails. And what they tell us is that if a patient with myelofibrosis comes in and they have a low risk by one of our scoring tools, we say, okay, you know, we can watch and monitor, we don't need to think about a stem cell transplant. But if somebody comes in and they're at the high-risk category, again, that doesn't say to us, we need to treat them with X, Y, or Z. But it does say to us, if we think this patient may be a candidate for stem cell transplant, they should be referred and have that consultation now rather than waiting.

Kristie L. Kahl: What is the prognosis in patients with vague symptoms compared to those with significant symptoms?

Dr. Rajiit Rampal: There is some degree of correlation between, you know, symptom burden and how the patient is doing clinically, does it necessarily change prognosis? The answer is to some degree, yes. If you think about this, in terms of our risk scoring tools, our risk scoring tools take account of a number of variables. You know, for example, a patient's age, whether they know they have circulating blasts, what their hemoglobin is, what their platelets are, but also if they have what we call constitutional symptoms. Constitutional symptoms are things like night sweats, and body aches and fevers and feeling fall early. So those are those fall under the heading of constitutional symptoms. And when we do the risk scoring system, patients will get a point on the scoring system if they have symptoms versus if they don't, and each point increases the risk of progression in the scoring system. So really, the answer is that it does play a role into how we think about the risk of progression.

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