Education on MPN Symptoms, Treatments Leads to Greater Involvement in Care

Learning more about the different symptoms and treatment goals of myeloproliferative neoplasms (MPNs) can help patients be more involved in management decisions throughout the disease trajectory, an expert said.

“It's not just the doctor and the nurses; it's the person who has the disease [that] is the main person, so their involvement is very important,” said Dr. Swati Goel at the recent CURE® Educated Patient® Updates in MPNs at Montefiore Medical Center in the Bronx, New York.

Goel is the Leader of the Myeloproliferative Disorder Clinic, assistant director of the hematology-oncology fellowship program and associate professor in the department of oncology and medicine at Montefiore Einstein in New York, New York.

Throughout the event, Goel discussed that there are three types of MPNs: polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis.

Polycythemia Vera

According to the National Cancer Institute, PV is when there are too many red blood cells in the blood and bone marrow, resulting in thick blood. The number of platelets and white blood cells may also increase with this condition. This may also lead to an enlarged spleen from the extra blood cells that collect in the organ, in addition to bleeding issues and clots in blood vessels.

Essential Thrombocythemia

With ET, patients have an increased number of platelets called thrombocytes in the blood without a known cause.

“The bone marrow produces too many platelets, and it leads to increased platelets in [the] bloodstream,” Goel said during the event. “The main thing we worry about [is] that this increased platelet [count] can lead to increased blood clots. There could be bleeding problems.”

She also noted the enlarged spleen in patients with ET, especially as it regulates the amount of blood cells in the body. Patients with ET may also have an increased risk for stroke, heart attack and circulation issues in extremities. Other symptoms that may be present include dizziness, headaches or vision changes.

Several factors may increase the risk for complications from ET such as genetic mutations, advanced age and, very rarely, exposure to certain chemicals like benzene.

Myelofibrosis

Myelofibrosis refers to a disorder when bone marrow is replaced by fibrous tissue. Goel noted that myelofibrosis is a little different than PV and ET, as it may also result in the production of too few cells in addition to too many cells in the bone marrow.

“The word ‘fibrosis’ means stiffness,” Goel explained. “So instead of being like spongy, nice marrow inside our bones, the myelofibrosis causes the stiffness. And when the bone marrow is stiff, it’s not able to produce the blood cells as it’s supposed to, and this, in turn, can lead to complications. So this causes scarring or the fibrosis in the bone marrow, and it disrupts the normal blood cell production.”

Primary myelofibrosis can be a result of the same genetic mutations as ET — JAK2, CALR and MPL — whereas secondary myelofibrosis can occur from progression from other blood disorders. Of note, Goel added that the mutations associated with myelofibrosis may happen over time.

“People are not born with these mutations,” Goel said. “They happen at some point in life, and this is not caused by smoking or other things.”

Common symptoms of myelofibrosis include fatigue, shortness of breath, abdominal pain and fullness from spleen enlargement, night sweats, fever, bruising or bleeding, weight loss and bone pains, among others.

“Bone pains are not [like] arthritis pain, like knee pain or joint pain,” Goel said. “It is pain inside the bones, like a different type of bone pain.”

Progression From One Disorder to Another

During the event, one participant asked whether ET or PV can progress to myelofibrosis, for which Goel answered that it can.

“There’s a small proportion of patients with ET or PV who can progress to myelofibrosis, as it’s the same mutation which causes all three diseases,” she said.

Progression from ET or PV to myelofibrosis would be noted upon the onset of symptoms or via monitoring from your care team with blood counts.

“We don't offer bone marrow biopsies if someone is doing well with PV or ET,” Goel said. “We don't offer them routinely, or let's check if there's fibrosis there or not. Usually, if there are symptoms or change in blood counts or something else, then we offer them. Even in the context of clinical trial, we might do it, but it's mainly symptoms and blood counts.”

Treatment Goals

Depending on which disorder a patient has, there are different goals of treatment. For example, for patients with PV, the main goal of treatment is to reduce the risk for blood clots, manage symptoms, control red blood cell counts and prevent complications such as strokes or heart attacks, Goel said.

Although somewhat similar, the goal of ET treatment is to control platelet counts, lower the risk for blood clots, manage symptoms and potentially prevent progression to “more serious conditions,” Goel said.

For patients with myelofibrosis, the goal of treatment is to improve blood cells, decrease symptoms, slow disease progression, improve survival, improve quality of life and reduce the spleen size.

“Many times, the spleen is big because the bone marrow is not functioning,” Goel explained. “The spleen starts producing all these abnormal blood cells.”

Patients with serious conditions may undergo a bone marrow transplant, which Goel said is a “cure in a way.”

“[This means] you get the transplant from someone else, not your bone marrow, which is very invasive and can be a lengthy procedure, but we do it in serious conditions,” she added. “But many times, we don’t need the bone marrow transplant because we can effectively manage the disease with treatment, with the regular monitoring, with trying to maintain the blood counts and have a very good quality of life for years or even decades.”

As MPNs can present in many ways in patients, treatment plans are typically personalized, Goel said. She noted that the most commonly used medication is hydroxyurea to reduce blood cell production. Interferon alfa, which is an injection given every week or every other week, may also be administered to patients. Goel also mentioned a new class of medications called JAK inhibitors to treat MPNs. Low-dose aspirin may be used to reduce the risk for blood clots in patients with ET or PV.

“Even though it might seem like, ‘It’s just baby aspirin,’ it can change the life of someone who had a devastating stroke versus preventing it.”

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