Patient-Reported Outcomes Drive Effective MPN Treatment

With patients with myeloproliferative neoplasms (MPNs) having long life expectancies, it’s important that treatments optimize quality of life and patient-reported outcomes, said Jennifer Vaughn, MD, hematologist-oncologist and assistant professor in the Division of Hematology at The Ohio State University Comprehensive Cancer Center – James Cancer Hospital and Solove Research Institute.

This transcript has been lightly edited for clarity.

Transcript

How are patient-reported outcomes currently being used and incorporated into clinical practice to inform treatment for rare hematological conditions?

I think myeloproliferative neoplasms are an important area where patient-reported outcomes are so important. The drug ruxolitinib or Jakafi, which is a JAK [Janus kinase] inhibitor used in both polycythemia vera and myelofibrosis, was actually originally approved in myelofibrosis because of the improvement it led to in patient quality of life.

First of all, it was able to objectively reduce spleen volume, which led to improvement in patient symptoms, and it led to reductions in symptom scores on the MPN symptom assessment form, which is sort of the standard form we use now to assess how a patient is feeling over time. While there have been some data later on that suggests there may be an overall survival benefit in certain subsets of myelofibrosis, we really do decide to put patients on that treatment because of what they're telling us about, how they feel. This was really one of the first models of using patient-reported outcomes in looking at whether or not a drug is valuable to society and to patients themselves.

Patients with MPNs become very symptomatic, and many of them will live [long] or have very excellent prognosis in terms of expected lifespan, but their quality of life is quite impeded by the symptoms of the disease. So, it's really important for clinicians to be kind of reevaluating that on a regular, routine basis.

How can patients and providers work together to develop personalized treatment plans addressing individual needs and preferences?

As the number of treatment options increase, or as the profile of different things that we can offer patients is expanding, which is a great thing, a lot of this is going to come down to some provider and patient shared decision-making. And it'll be important for patients to really consider what they value in the course of their treatment. Whether that be conservation of their time so they can focus on work and focus on things that give them an optimal quality of life. Or whether that be reduction in their symptoms. Or whether they’re someone who wants to seek out opportunities to extend their life and overall survival for as long as they can, in which case depending on the disease they have, seeking out more aggressive therapies like stem cell transplant; we’re really waiting for the data to return on these potential disease-modifying agents. I think it's a good place to be in in the MPN world, but it’s definitely going to require more back and forth conversations between patients and providers.