Many treatment options are available for myeloproliferative neoplasms (MPNs), including types like primary myelofibrosis (PMF), essential thrombocythemia (ET), and polycythemia vera (PV). Additionally, new therapies are under development in clinical trials. Hematologist-oncologists (blood disease and cancer specialists) recommend treatments tailored to an individual’s specific MPN type, previous treatments, current blood counts, and unique risk factors. Ultimately, you have the choice to decide which treatment aligns best with your needs and preferences.

In most cases, MPNs are not curable. However, advances in medication and treatment options have made MPNs manageable as a chronic condition. Because MPNs often progress slowly, many people with these conditions can enjoy a near-normal lifespan with appropriate treatment.

Assessing Your Risk Before Treatment

There are several types of MPNs, each affecting different types of blood cells. These variations in blood cell production are caused by specific genetic mutations.

Before recommending treatments for MPNs, oncologists assess an individual’s risk factors. These factors can increase a person’s likelihood of developing serious complications, such as blood clots. They may also increase the risk that their MPN will transform into a more aggressive blood cancer, like acute myeloid leukemia (AML).

Risk factors doctors consider include:

• Age
• Presence of MPN symptoms
• Blood cell count results
• Specific genetic mutations in cancer cells
• Overall health status
• Other health conditions, such as diabetes or hypertension (high blood pressure)

For people with low-risk MPNs who don’t have symptoms, doctors may recommend a watch-and-wait approach rather than beginning treatment immediately. Also referred to as watchful waiting or observation, this approach involves closely monitoring a person’s condition for any signs or symptoms of progression. If your risk level changes, your doctor may recommend beginning treatment.

Treatment options for MPNs fall into several categories. Some therapies are designed to target and destroy abnormal cells, while others slow the cells’ growth or reduce the risk of serious complications. Supportive treatments are available to help manage symptoms, minimize complications, and improve quality of life.

1. Chemotherapy for MPNs

Chemotherapy drugs, also known as anticancer agents, work by killing or damaging cells, including abnormal blood cells. Chemotherapy for MPNs may involve a single drug or a combination of drugs, depending on the treatment goals and disease progression.

In MPNs, traditional chemotherapy is typically reserved for advanced-stage myelofibrosis, especially when there is a high risk of progression to AML. Chemotherapy can also be used in cases of chronic myeloid leukemia (CML, also called chronic myelogenous leukemia) that have entered the aggressive blast phase, although kinase inhibitors have largely replaced chemotherapy. CML is a type of MPN where too many white blood cells called granulocytes are made. CML is characterized by the BCR-ABL1 gene mutation (Philadelphia chromosome), which distinguishes it from other MPNs​​.

High-dose chemotherapy may be intensive in preparation for a stem cell transplant. If a transplant is not an option, doctors may recommend a low-intensity chemotherapy regimen to manage symptoms and reduce disease progression.

Several chemotherapy drugs may be prescribed for treating different types and stages of MPNs:

• Azacitidine (Vidaza) and decitabine (Dacogen) — These may be given as part of a low-intensity regimen for advanced MPNs
• Busulfan (Myleran) — This drug may be used in specific cases of PV, ET, and CML to help control excessive blood cell production.
• Hydroxyurea (Droxia, Hydrea, Siklosi) — This chemotherapy drug is often used to treat most types of MPNs. It works by interfering with DNA synthesis, which prevents cells from dividing and helps to control high blood cell counts.
• Omacetaxine mepesuccinate (Synribo) — This chemotherapy drug may be used to treat people with CML who have not responded to other treatments.

Some of the most common side effects of chemotherapy for MPNs include:

• Nausea and vomiting
• Diarrhea
• Fatigue (extreme tiredness)
• Loss of appetite
• Changes in blood cell counts

2. Kinase Inhibitors for MPNs

In the past 20 years, several medications in a class known as kinase inhibitors have been approved by the U.S. Food and Drug Administration (FDA) to treat different types of MPNs. Kinase inhibitors are considered a type of targeted therapy because they target specific processes that cancer cells need to grow. There are two main types of kinase inhibitors used to treat MPNs: Janus kinase (JAK) inhibitors and tyrosine kinase inhibitors (TKIs).

Janus Kinase Inhibitors

JAK is a protein that helps cells grow and develop. In some people with MPNs, the JAK protein becomes too active. JAK inhibitors help to decrease how many blood cells are made. These medications can also help improve MPN symptoms and reduce the size of your spleen.

JAK inhibitors approved to treat myelofibrosis include:

• Fedratinib (Inrebic)
• Momelotinib (Ojjaara)
• Pacritinib (Vonjo)
• Ruxolitinib (Jakafi)

Tyrosine Kinase Inhibitors

TKIs can be used to treat chronic myeloid leukemia. TKIs can help block abnormal proteins in cancer cells that cause uncontrolled growth. When these proteins are blocked, the CML cells die.

Several TKIs are used to treat MPNs:

• Bosutinib (Bosulif) — This drug is used to treat Philadelphia chromosome-positive CML in the chronic phase.
• Imatinib mesylate (Gleevec) — This was the first TKI approved to treat CML and it is still often the first-line therapy for people who are newly diagnosed with the condition. It is also used in some cases of myelodysplastic and myeloproliferative diseases, acute lymphoblastic leukemia (in people with leukemia who test positive for BCR-ABL1 translocation), and chronic eosinophilic leukemia (CEL, also called hypereosinophilic syndrome).
• Nilotinib (Tasigna) and dasatinib (Phyrago, Sprycel) — These drugs are FDA-approved to treat some cases of CML, particularly for individuals who become resistant to first-line therapy.
• Ponatinib (Iclusig) and asciminib (Scemblix) — These are prescribed to treat CML that does not respond to other TKIs.

Rarely, do people using kinase inhibitors develop serious cardiac (heart) problems, such as congestive heart failure. They often have other health conditions, such as a history of heart disease, high blood pressure, or diabetes, so it’s important to monitor them closely.

3. Interferons for MPNs

Interferons are proteins the body uses to signal and trigger an immune response. They may be prescribed as part of treatment for most MPNs to help reduce the number of blood cells. Before the advent of TKIs, interferons were the mainstay of treatment for CML. However, they are now used as add-on therapy in individuals who no longer respond to TKIs or who are pregnant.

Interferon drugs used for MPNs include:

• Peginterferon alfa-2a (Pegasys)
• Ropeginterferon alfa-2b (Besremi)

Interferons are a type of immunotherapy. They are approved by the FDA to treat several types of blood cancers.

Common side effects of interferons include:

• Flu-like symptoms
• Fatigue
• Nausea and vomiting
• Difficulty concentrating
• Low blood cell counts
• Muscle and bone pain

These side effects will last as long as you’re taking the medication. However, most people find they become easier to handle over time.

4. Angiogenesis Inhibitors for MPNs

Thalidomide (Thalomid), lenalidomide (Revlimid), and pomalidomide (Pomalyst) are angiogenesis inhibitors. They work by preventing the growth of new blood vessels that deliver nutrients to cancer cells. Lenalidomide can also help treat anemia.

Danazol (Danocrine), a hormonal drug, and corticosteroids, such as prednisone, may be prescribed in combination with angiogenesis inhibitors for MPNs.

5. Stem Cell Transplant for MPNs

In rare cases, a stem cell transplant after high-dose chemotherapy can be a cure in some people with myelofibrosis and CML. The purpose of a stem cell transplant (also referred to as a bone marrow transplant) for MPNs is to replace cancerous bone marrow cells with stem cells that will form healthy bone marrow. Stem cell transplants take place after the cancerous cells of the bone marrow have been destroyed with chemotherapy, radiation, or a combination of the two.

Allogeneic stem cell transplants are used to treat MF and CML. In an allogeneic transplant, stem cells are harvested from a donor. This is usually a sibling or a close blood relative but is sometimes an unrelated person who is a good genetic match.

While allogeneic transplants can potentially cure MF, they carry a risk of severe side effects, including graft-versus-host disease (GVHD). In GVHD, the transplanted donor cells attack the host’s tissues. The risk for GVHD is lower — but still present — when the donor is a close genetic match to the recipient. The recipient may need to take antirejection medication for life to prevent GVHD.

6. Supportive Treatments for MPNs

Some treatments are not prescribed to treat the cancer itself, but to reduce symptoms of MPNs and lower the risk for serious complications and related conditions. Supportive treatments can include medications and procedures.

Removal of Excess Blood Cells

Each type of MPN involves the overproduction of blood stem cells. Excess blood cells enter the circulation and cause MPN symptoms. Procedures to remove excess blood cells can temporarily improve MPN symptoms and lower the risk for serious complications.

In polycythemia vera, excess red blood cells may be removed through the process of phlebotomy, or blood drawing. This is similar to donating blood. Phlebotomy can temporarily improve PV symptoms and lower the risk of blood clots.

Apheresis is a procedure to remove specific types of excess blood cells. Apheresis is also similar to the process of donating blood. The blood flows through a machine called a separator, which removes only the type of cell required and returns the rest of the blood to the body. Different types of apheresis may be used depending on the type of MPN and type of excess blood cell:

• Plateletpheresis is the removal of platelets. It may be used to treat essential thrombocythemia.
• Leukapheresis is the removal of white blood cells. It may be used during pregnancy in cases of CML.

Splenectomy

Splenomegaly (enlarged spleen) is a common and uncomfortable symptom in some types of MPN. Several medications, such as JAK inhibitors, are effective at reducing the size of the spleen in most people.

In cases where the splenomegaly is severe and doesn’t respond to medications, your doctor may suggest splenectomy (surgical removal of the spleen). Splenectomy is usually effective in relieving pain and discomfort and may improve blood cell counts. However, splenectomy carries significant increases in the risk of death or progression to a more aggressive form of blood cancer.

Radiation Therapy

Radiation therapy (also called radiotherapy) isn’t a common treatment for MPNs. Radiotherapy may be used to destroy abnormal bone marrow and blood stem cells before a stem cell transplant for myelofibrosis. In some cases, radiation may be used to treat symptoms of MPNs with either external beam radiation or the intravenous infusion of a radioactive drug. Radiation is also sometimes used to reduce the size of an enlarged spleen.

Platelet-Reducing Medications

For some people with essential thrombocythemia and polycythemia vera, doctors may prescribe medications to reduce the platelet count and lower the risk of blood clots. Anagrelide is a platelet-reducing medication. Aspirin can also help decrease the risk of blood clots even though it doesn’t reduce the number of platelets, it causes the platelets not to function properly so they cannot form clots. Similarly, if you are at high risk for blood clots, your doctor may prescribe a blood thinner such as warfarin.

7. Clinical Trials for MPNs

Clinical trials may be an option for some people with MPNs. Clinical trials can offer people with MPNs access to an investigative treatment while furthering scientific knowledge about better ways to treat MPNs.

MPNs With No Standard Treatment

Some MPNs are so rare that no standard treatment has been established. This is true for chronic neutrophilic leukemia, chronic eosinophilic leukemia, and myeloproliferative neoplasm, unclassifiable.

Find Your Team

On myMPNteam, the social network for people with myeloproliferative neoplasms and their loved ones, more than 4,800 members come together to ask questions, give advice, and share their stories with others who understand life with MPNs.

What treatments have you taken for MPNs? How did you and your health care team choose the best treatment for you? Share your experience in the comments below, or start a conversation by posting on your Activities page.