Patients With MPNs Must Be Honest About Their Symptoms, Says Expert

Because patients with essential thrombocythemia (ET) or polycythemia vera (PV) — rare types of blood cancers known as myeloproliferative neoplasms (MPNs) — experience a range of symptoms that can significantly impact their quality of life, it’s important that patients communicate what they’re experiencing with their health care team.

Symptoms may include fatigue, symptoms from blood clots, abdominal discomfort (sometimes from spleen enlargement), fatigue, weight loss, fever and itching. Dr. Ruben Mesa, executive director of The Mays Cancer Center at UT Health San Antonio MD Anderson Cancer Center, explained how symptoms of ET and PV impact patients and affect quality of life.

“Elevated (blood) counts can give you a risk of blood clots or bleeding, but you don’t necessarily feel them. So it’s really the PV- or ET-related symptoms that are the bigger driver in terms of quality of life,” Mesa said in an interview with CURE®. “We also know that these are chronic diseases. Patients can have them for many years, sometimes decades. So the control of these symptoms (becomes) incredibly important.”

Measuring symptoms of myeloproliferative neoplasms (MPNs) helps doctors quantify something that is inherently subjective. It has been shown that questionnaires result in more complete and honest assessments of how a patient is feeling compared with an in-person physician checkup.

“(It’s) critical to understand (symptoms and) to track them. They clearly have an impact on therapy, sometimes the choice of drug, sometimes the dose (and) sometimes the need to move to a different therapy,” he explained.

To further demonstrate how symptom burden assessment may help identify treatment efficacy, Mesa and researchers examined symptom burden prior to and after treatment. The aim was to see how treatment impacted symptom burden of patients using data from two clinical trials published in Lancet Hematology:

• 114 patients (64% with ET; 44% with PV) from the MPN-RC 111 study, which evaluated the drug Pegasys (pegylated interferon alfa-2a) in patients with ET or PV resistant to Hydrea (hydroxyurea).
• 166 patients (48% with ET; 52% with PV) from the MPN-RC 112 trial, which evaluated alfa-2a versus Hydrea in therapy-naive patients with high-risk ET or PV.

Specifically, treatment with Pegasys or Hydrea was associated with an improvement in symptom burden between three and 12 months in patients who entered each trial with a high symptom burden. These medications, according to Mesa, helped decrease blood clots and inflammation, two main symptoms experienced by patients with MPNs.

However, symptom burden worsened between three and 12 months in patients who had a low symptom burden at the start of the MPN-RC 111 study, and similar results were observed in the patients from the MPN-RC 112 study.

Mesa explained that some therapies don’t reach both goals — control of blood counts (and hence decrease risk of blood clots) and the secondary goal of either improving prior treatment symptoms or not creating new symptoms as toxicity from the treatment.

“You can’t make a patient who feels well feel any better,” he said. “And (for) individuals with low baseline symptoms, there is a trade-off of some slight increase in symptoms or side effects for the protection that these medicines (give) to decrease the risk of blood clots or bleeding.”

He emphasized the importance of patients honestly discussing the symptoms they are experiencing with their health care team. He explained that many of these symptoms may not be apparent externally but that patients still feel them — so their health care team needs to know about them.

“I think this is a prime example of where ... close cooperation between patients, patients’ groups and researchers in the field has been very helpful,” he said. “Really, all of this work started because patients ... were coming up to me saying, ‘My symptoms are worse than people really recognize. And although I may (look fine on the outside), if you see me in the mall, I really don’t feel well.’ So how do we better understand that?”

Mesa also noted that some skeptics thought these symptoms were caused by the stress of having the disease; however, research has shown they can also be caused by the treatments and the biology of the disease itself.

“For many, the symptoms are caused by the biology of their MPN and a critical sign of the disease” he said. “And then when we treat the disease, things are better. They’re more stable. And hopefully then we’re able to have them in a less intensive mode where they’re on therapy, (and) they’re getting blood counts every two to three months. But overall, they’re able to really put (the) PV or ET in the rearview mirror — it’s there, but not a significant part of their day-to-day lives.”

For more news on cancer updates, research and education, don’t forget to subscribe to CURE®’s newsletters here.