Recognizing Symptoms of Myeloproliferative Neoplasms and Clinical Trial Challenges

The symptoms of myeloproliferative neoplasms can be variable and common, which can make it difficult to diagnose if you aren’t looking for the right thing, said Ruben Mesa, MD, FACP, executive director of Atrium Health Wake Forest Baptist Comprehensive Cancer Center and president of Atrium Health Levine Cancer.

He also discusses the challenges with getting patients enrolled in clinical trials, such as the limited availability of them and patient factors that make it difficult to participate.

This transcript was lightly edited for clarity.

Transcript

What are some symptoms of myeloproliferative neoplasms that may be ignored by patients that may result in delayed diagnoses?

The symptoms of myeloproliferative neoplasms are variable, can be problematic, but also are common, so it can be, certainly, 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 then would come symptoms related to high blood counts, such as headaches, difficulties with concentration, and migraines with aura. In those with myelofibrosis, the spleen can be enlarged, sometimes dramatically, and lead to early satiety or abdominal discomfort. As well there can be a range of additional constitutional symptoms, such as inadvertent weight loss, night sweats, and even fevers.

There can be certainly a delay in the diagnosis of myeloproliferative neoplasms, and really a pretest suspicion is certainly helpful. Patients typically will present either through abnormal blood counts being done at the time of a routine physical or for evaluation for any of these symptoms or complications which have arisen, like blood clots or a bleeding event. Individuals are found to have splenomegaly at the time of a routine exam or incidental x-ray finding.

What special hurdles do you encounter when recruiting patients with MPNs for clinical trials, and wht can be leveraged to overcome them?

Barriers to enrollment clinical trials is, as a less common disease, the availability in local centers of these clinical trials tends to be more limited. They tend to be more concentrated in larger academic centers, where there's more of a subspecialized hematology practice. I think the more that clinical trials evolve to allow telemedicine assessments for interim visits and allow greater flexibility for people to enroll with fewer and less intensive visits to a registering center help to make those more accessible.

Additionally, as we work to further expand the access to clinical trials in a way that it is less burdensome for there to be access to clinical trials closer to home for individuals, the more these trials are able to be leveraged.

Are there any populations that do better or might be more willing to participate with an online vs in-person format?

Undoubtedly, based on social determinants of health, it clearly takes resources in terms of time, ability to travel, time off of work, as well as having employment that allows you to be absent for health care needs. All [of these factors] favor clinical trial participation by those that have good insurance, those that have more significant resources, and those that truly have greater access.

The clinical trial process has historically exacerbated issues in health equity, because it really limits the ability to participate for those that have jobs that pay them by the hour, difficulties with transportation, and other challenges.