A discussion on assessing symptom burden and quality of life for patients with essential thrombocythemia or polycythemia vera.
Rami Komrokji, MD: We under-recognize the symptom burden in ET [essential thrombocythemia] and PV [polycythemia vera]. How do we assess the quality of life and the impact of symptoms in patients? What are our tools to assess that? Because we pay a little more attention to the symptom burden in myelofibrosis [MF] than ET and PV. In my experience, those patients are symptomatic from the disease, not just the uncontrolled counts and the risk of thrombosis.
Ruben Mesa, MD: It’s been an area of passion for myself, Jeanne Palmer, and many others working in this space, driven by conversations with our patients. They have come to us and said, “We don’t feel well. Help us make the case for the symptoms we’re facing,” and then we weave it into our decision-making for treatment. Working with many colleagues and patients, we developed the MPN [myeloproliferative neoplasm] Symptom Assessment Form as well as the Myelofibrosis Symptom Assessment Form. They’re all somewhat interchangeable. We have data in thousands of patients, both in and outside clinical trials.
Patients with MPNs are symptomatic—not everyone, but about half of those with ET, about 60% to 70% of those with PV, and almost 90% of those with MF. It isn’t universal. There are some asymptomatic patients with MF, but there are few, particularly if you look into it. With ET, it’s much more common than you’d think. It’s an important thing to factor in. Not all symptoms are equal. In ET and PV, symptoms might be very driven from high counts: difficulties with concentration, complex migraines, erythromelalgia, fatigue, pruritis, and worsening adverse effects from drugs or iron deficiency. As things evolve into MF, it evolves. There’s worsening fatigue, inadvertent weight loss—which doesn’t happen in our society unless there’s a reason, bone pain, and night sweats. The symptoms aren’t all created equal.
It’s now the standard of care to track symptoms. It’s certainly in our NCCN [National Comprehensive Cancer Network] guidelines. These tools are in the public domain. You can use them. Patients can fill out these questionnaires in your waiting room in 30 to 45 seconds. With them, you can see where they start at baseline and whether it’s getting better or worse with whatever you’re doing. Questionnaires are better than sitting them on the couch and asking, “How do you feel?” That tends to underrepresent. It isn’t very quantifiable. You can’t track it. It’s a critical part in initiating therapy and adjusting the dose.
Rami Komrokji, MD: Absolutely. You have to be applauded on your efforts because this is one of the few diseases, at least in hematologic malignancies, in which quality of life was done in an objective way using those tools. It’s one of the primary end points that the FDA looks at to approve drugs, which translates to benefit for our patients.
To your point, we all get busy in practice. Sometimes I don’t do them formally. But studies require them, and the discrepancy between what you think the patient is experiencing and what you learn when they fill those out objectively is amazing. We don’t realize the burden of the disease when we’re just chatting with the patients.
Ruben Mesa, MD: The key is that this isn’t for you as the treating physician to take your time out and walk them through a form. For me, it’s about workflow. At our cancer center, all patients fill out a general questionnaire, the Edmonton Symptom Assessment Score, which can triage a range of things, and triage them into different supportive care mechanisms and then potentially a disease-specific form. It’s about workflow. As a physician, if you’re sitting down with a questionnaire for whatever disease, you’re doing it wrong. It should be independent. You guys are busy. You’re dealing with other things. It should be part of the workflow, but it shouldn’t be something on your shoulders. Some things are underutilized because they aren’t rolled out in the right way.
Rami Komrokji, MD: Absolutely. Jeanne, you still get consults for transplant in PV and ET. Is there any role for it?
Jeanne Palmer, MD: Not really. Although if those patients come along, it’s important to make sure their ET or PV hasn’t progressed. Unfortunately, sometimes a patient’s disease will be well controlled and they’re thinking, “This is great,” but they start to develop more symptoms and an enlarged spleen and then you do bone marrow, which shows that they have myelofibrosis. At that point, we consider transplant. For PV or ET that hasn’t progressed, it isn’t something I consider doing.
Transcript edited for clarity.