Elevating the Voice of Patients with Myeloproliferative Neoplasms - Episode 5

Understanding MPN Symptom Management

CURE spoke with Dr. Rajiit Rampal, on behalf of the MPN Research Foundation, about MPN symptoms and their management.

Kristie L. Kahl: Can you discuss the disease related symptoms in MPN?

Dr. Rajiit Rampal: Yeah, so you know, there's a variety of different symptoms that we see with patients with MPNs. And that can include things commonly like fatigue, that's one of the most prevalent symptoms that we see. And there's a host of other symptoms, some of which are enriched in the different subtypes (of MPNs), you know, for example, things like feeling full early when somebody eats. So you know, one of the questions we always ask patients are, is the amount of food that you're eating or able to eat at one sitting has that decreased over time, and that's the medical term for that is early satiety, and that's often a sign that the spleen or the liver has enlarged. That's a symptom that patients can feel abdominal discomfort, they can feel a sense bloating. People may have night sweats at night, and that when we talk about night sweats, what we're really talking about is drenching night sweats where somebody is, you know, they feel soaked to the point that they need to change their clothes, as opposed to, you know, just j sort of a light sweat from having too heavy of covers on as an example, people can have low grade fevers, they can have body aches and pains, often muscle pains. In fact, bony pain is also another thing that is a sort of a distinct symptom that patients get as well. That's kind of you know, I think of a broad landscape of the of the symptoms that we see in patients. It's important to remember though we not all, not every patient has every symptom, and patients can have symptoms that are not on the list that I've just described, it can be very individual.

Kristie L. Kahl: Why is it important for patients to know about these symptoms and be able to identify them themselves?

Dr. Rajiit Rampal: Well, for a couple of reasons, you know, one of the things is that symptoms, if they emerge over time can be a sign of disease progression. And in some cases, this can be subtle, right? You know, somebody starts to notice that they're a little bit more fatigued, they can't do as much as they were doing. They try to compensate, particularly if they're younger. And it can be subtle, but that's an important clue to both the patient and their treating physician that something's changing here. I think that's one of the main things that's important for patients to recognize. The other is that, you know, s, the symptoms that I've described are not necessarily specific. They can occur for a variety of reasons. But it's important to know what to attribute them to, particularly since some of them can cause distress, and some of them are warning signs to us that, you know, again, that the diseases change, can it be from a patient's MPN? Yes. Could there be other causes for which a patient is having those symptoms? Yes. A good example of this is fatigue, right? fatigue is often, if not always, multifactorial. And fatigue can be caused by any number of things, it could be caused by the disease itself, it can be caused by anemia that's related to the disease or maybe not related to the disease, it could be caused by things like thyroid dysfunction. So when the patient has these symptoms, it's important for them to bring that to the attention of their treating doctor so that we can make an assessment as to is this a change in the disease status? And is the symptom that we're talking about really attributable to the MPN? Or is there something else that we need to think of medically?

Kristie L. Kahl: I wanted to focus also on the symptom of enlarged spleens. What should patients know about this symptom? And what could it mean for them?

Dr. Rajiit Rampal: What happens with the spleen in terms of symptoms early on, many patients have no symptoms. What people will describe is that they will occasionally notice a discomfort not a pain per se, always but a discomfort in the left side of their abdomen. And you know, many patients will notice this most prominently when they're bending down to tie their shoes, or when they're lying in bed, and they turn on their left side notice, you know, discomfort, and sometimes that's one of the symptoms that brings a patient to the attention of their physician to be worked up, you know, for being diagnosed. Over time, you know, the spleen does tend to enlarge and when it enlarges patients feel a couple of different things. One is what we just talked about a moment ago, the the term early society where the spleen starts to compress the organs of the abdomen, and patients feel that they are getting full early on, or if they could have eaten a plate of food, now they can eat maybe half that plate, because they feel full, or they will often describe feeling that food is getting stuck. And that if nothing is, is moving when they eat. So that's I think one of the most prominent signs. And then there is pain. And patients can describe having sharp pains and the left side of their abdomen, but occasionally they'll have a really prolonged persistent sharp pain. And that is something that needs to be brought to medical attention right away. Because what can happen as the spleen expands in size is that it can out outgrow its blood supply. And so what can happen is what's called the splenic infarct, were essentially part of the spleen loses its blood supply. And actually that part of the spleen dies. That is an immensely painful thing. In many cases. We don't do anything about it from an intervention side except to treat the patient's pain, but that's often a sign that the spleen has enlarged rapidly.

Kristie L. Kahl: How do these affect patients’ day-to-day lives?

Dr. Rajiit Rampal: It can have profound effects on a patient’s life. And this has really been well studied by Ruben Mesa and his group. And the impacts that this can have, you know, patient surveys that have been done in terms of how are your symptoms impacting you in terms of your ability to go to work to carry out activities that you would normally carry out. And what the data sort of on a high level says is that the symptoms do impair things like the patient's ability or desire to go to work and to, you know, again, carry out activities that they normally would, and in essence, the symptoms can impact the patient's ability to have a normal life and to affect their quality of life.

Kristie L. Kahl: How are these symptoms treated?

Dr. Rajiit Rampal: Yeah, you know, I think that the, the fundamental principle is that the treatments that we use to treat the underlying MPN are in many cases, the things that we use in parallel, treat the symptoms. A good example of this is the jak inhibitors, rock solid Nevin the dragnet, you know, those drugs have shown statistical benefit to reducing the size of the spleen, but also to reducing the patient's symptom burden. So things like the night sweats the fatigue, the feeling of the early satiety, some in some cases, muscle pain, all of those things can get better in a patient was treated with a JAK inhibitor. And to an extent we see the same thing, and for example, patients would probably say, for me, there were some of the older drugs that we use, like hydroxyurea don't really seem to have a great deal of impact on patients’ symptoms. But you know, things like the jak inhibitors certainly do seem to have an impact. And then there are, you know, other more simpler things that sometimes can be done, you know, patients with the essential thrombocythemia, or even PV in your variable sometimes complain of what we call erythromelalgia, which means that they're having a burning and tingling in their extremities in their hands. And oftentimes, that can be mitigated by giving aspirin once or twice daily. So there's a variety of things that we can use, some of which are diseases out there, directed, I would say, the majority of which are disease directed, but some of which are purely symptom directed.

Kristie L. Kahl: What is your biggest piece of advice for patients to not only understand their disease, but its symptoms?

Dr. Rajiit Rampal: I think the biggest thing I could say to the patient, and what I often say to patients, when I'm seeing them, they worry about bothering their doctors with symptoms. And I would say the opposite. If you have a symptom, you need to let us know, right? It may not be related, it might be something completely different. Or it might be a sign that your disease is worsening. But we want to know about those things. And I think our patients should not hesitate to let their physicians know about symptoms. Because you know, sometimes what happens is that we put all of the data together, and we're seeing a patient, the patient's lab values, their examination and symptoms, and that is it part of the entire assessment that we make of how a patient is doing but if we don't hear about those symptoms and things that might even be subtle, or which somebody wants to try to de emphasize, we don't have the complete picture of what's going on with the patient. So I strongly believe our patients should you know, be vocal and tell us all of the things even if they don't necessarily think that it's a big deal. We want to know about it.