Recognizing Symptoms of Myeloproliferative Neoplasms and Challenges in Clinical Trials

Symptoms of myeloproliferative neoplasms can be variable and common, which can make diagnosis difficult if you’re not looking for the right thing, said Ruben Mesa, MD, FACP, executive director of Atrium Health Wake Forest Baptist Comprehensive Cancer Center and president of the Atrium Health Levine Cancer.

It also discusses the challenges of getting patients enrolled in clinical trials, such as their limited availability and patient factors that make participation difficult.

This transcript has been lightly edited for clarity.

Transcription

What are some symptoms of myeloproliferative neoplasms that may be overlooked by patients and result in delayed diagnosis?

Symptoms of myeloproliferative neoplasms are variable, they can be problematic, but they are also common, so they can certainly be nonspecific and not necessarily point people in the right direction to begin with. In particular, patients with myelofibrosis are the most symptomatic and then those with polycythemia vera and essential thrombocythemia. Fatigue is the most prevalent symptom, but later come symptoms related to high blood counts, such as headaches, difficulty concentrating and migraines with aura. In those with myelofibrosis, the spleen may enlarge, sometimes dramatically, and cause early satiety or abdominal discomfort. There may also be a number of additional constitutional symptoms, such as inadvertent weight loss, night sweats, and even fever.

There can certainly be a delay in diagnosing myeloproliferative neoplasms, and a pre-test suspicion is really helpful. Patients typically present with abnormal blood counts done at the time of a routine physical examination or for evaluation of any of these symptoms or complications that have arisen, such as blood clots or a bleeding event, or individuals present with splenomegaly at the time of a routine examination or incidental finding,

What special obstacles do you encounter when recruiting patients with MPNs into clinical trials and what can be leveraged to overcome them?

Barriers to enrolling in clinical trials are, as it is a less common disease, the availability of these clinical trials at local centers tends to be more limited. They tend to be more concentrated in larger academic centers where there is a more subspecialized hematology practice. I think the more clinical trials evolve to allow telemedicine assessments for interim visits and allow more flexibility for people to enroll with fewer and fewer intensive visits to a registration center, that helps make them more accessible.

Additionally, as we work to further expand access to clinical trials in a way that is less burdensome to individuals, more of these trials will be able to be leveraged.

Is there a population that does better or might be more willing to participate in an online versus in-person format?

Without a doubt, based on the social determinants of health, resources are clearly needed in terms of time, ability to travel, time off work, as well as having a job that allows you to take time off for healthcare needs. All (these factors) favor participation in clinical trials by those who have good insurance, those who have more significant resources and those who actually have greater access.

The clinical trial process has historically exacerbated health equity issues because it actually limits the ability of those who have hourly jobs, transportation difficulties, and other challenges to participate.