Myeloproliferative Neoplasms

Myeloproliferative neoplasms (MPNs) are a group of diseases that affect the way the body creates blood cells. They cause your body to make too many red blood cells, white blood cells, or platelets. These chronic blood cancers can be severe, but most people can live with them for many years. There are several types of myeloproliferative neoplasms:

  • Chronic myelogenous leukemia
  • Primary myelofibrosis
  • Polycythemia vera
  • Essential thrombocythemia 


Some patients seek medical care because of a specific symptom such as fatigue, itching, abdominal discomfort on the left side, frequent headaches, and difficulties concentrating. Many patients with myeloproliferative neoplasms have no symptoms. But you may have an abnormal blood count, which would enable your doctors to make a diagnosis.

Specific symptoms of chronic myelogenous leukemia include:

  • Anemia
  • Bone or joint pain
  • Fatigue, general malaise
  • Fever and night sweats
  • Gastrointestinal bleeding
  • Infection
  • Trouble breathing
  • Weight loss or loss of appetite

If you have primary myelofibrosis, you may experience:

  • Abdominal discomfort or pain
  • Abnormal bleeding
  • Anemia
  • Bone pain
  • Fatigue, general malaise
  • Feeling full quickly with meals
  • Fever and night sweats
  • Trouble breathing
  • Weight loss

Patients with polycythemia vera often experience:

  • Blockage of blood vessels. This may cause heart disease, stroke, or gangrene (tissue death) of the arms and legs.
  • Fatigue, general malaise
  • Headache and problems with vision
  • High blood pressure
  • Intense itching after bathing in warm water
  • Purple spots or patches on the skin
  • Stomach aches
  • Throbbing and burning pain in the skin, often with darkened, blotchy areas
  • Trouble breathing

If you have essential thrombocytosis, you might have:

  • Blood in the urine
  • Bruising
  • Burning or throbbing pain, redness, and swelling of the hands and feet
  • Headache
  • Heart attack or stroke


We don’t know exactly what causes myeloproliferative neoplasms. As with all cancers, a change occurs in a single cell that makes the cell escape normal control mechanisms and grow and divide inappropriately. With MPNs, the change occurs in a stem cell, which gives rise to all the blood cells. In the case of CML, this change is well characterized and is known as the Philadelphia chromosome.

Studies suggest that exposure to radiation or certain chemicals may increase the risk of developing an MPN. In rare cases, families could have a genetic predisposition to develop a myeloproliferative neoplasm.


 Typically, primary care doctors perform blood tests as part of an annual physical. If an abnormality in your blood count is detected, your doctor may refer you to a blood specialist (hematologist) for further testing.

Hematologists use a variety of tests to help make a diagnosis:

  • Blood tests: These tests can reveal abnormal types or numbers of red blood cells, white blood cells, or platelets.
  • Bone marrow biopsy: Your doctor may take a small sample of bone marrow to make or confirm a diagnosis of MPN. Your doctor will explain this procedure to you in detail.
  • Cytogenetic analysis: This test examines the genetic material (chromosomes) in your blood or bone marrow cells. Specific changes in the chromosomes, such as the Philadelphia chromosome, can help your doctor diagnose your condition and predict the course and severity of the disease.
  • Mutational analysis: This is a test to see if there are any genetic mutations that are associated with MPNs.

Treatments We Offer

Most of the time, we cannot cure myeloproliferative neoplasms. However, there are several treatments that help improve symptoms and prevent complications. For advanced forms of MPN, we might perform a stem cell transplant. The treatment for each type of myeloproliferative disorder is slightly different.

Chronic Myelogenous Leukemia (CML)

Treatment options for CML have expanded greatly in the last 20 years. Targeted therapy can provide complete control of the disease and can even cure some patients. We use medications such as:

  • Targeted drugs: These drugs affect a specific protein that is produced as a result of the Philadelphia chromosome. This protein enables cancer cells to multiply. Targeted drugs include Dasatinib (Sprycel), Imatinib (Gleevec), Nilotinib (Tasigna), Bosutinib (Bosulif), and Ponatinib (Iclusig).
  • Interferon: This medication helps the immune system combat cancer cells.

Primary Myelofibrosis

Treatment includes medications and blood transfusion when necessary. Medications may include:

  • Ruxolitinib (Jakafi) or Fedratinib (Inrebic): These oral medications, known as JAK inhibitors, help control the symptoms of MF such as large spleen, weight loss, poor appetite, itching, night sweats, fatigue, and bone pain.
  • Hydroxyurea: This drug may control complications, such as enlargement of the liver and spleen. It may reduce the number of white cells and platelets in the blood.
  • Thalidomide and lenalidomide: These oral medications reduce symptoms and treat anemia.

Polycythemia Vera

A goal of treatment is to lower the red blood cell count. To do this, we remove some of your blood. Known as therapeutic phlebotomy, this treatment is similar to donating blood. Medications may include:

  • Hydroxyurea (Hydrea): This oral medication reduces the number of blood cells.
  • Interferon: This is a biotherapy that is used to treat some blood cancers. It is given as an injection. Interferon uses the body’s immune system to control the disease. It controls blood counts and some symptoms.
  • Ruxolitinib (Jakafi): This oral medication, known as a JAK inhibitor, controls blood counts and many symptoms of PV, including itching.
  • Low-dose aspirin: This reduces the risk of blood clots. It also reduces the skin redness and burning that some patients experience.
  • Allopurinol: This medication lessens symptoms of gout, which is a potential complication of PV.

Essential Thrombocytosis

We treat essential thrombocytosis with medications, which may include:

  • Low-dose aspirin: This reduces the risk of blood clots. It also reduces skin redness and burning that may occur in some patients.
  • Hydroxyurea (Hydrea): This drug reduces the number of blood cells.
  • Anagrelide (Agrylin): This is an oral medication that reduces the number of platelets.
  • Interferon: This biotherapy is used to treat some blood cancers. It is given as an injection. Interferon uses the body’s immune system to control the disease. It controls blood counts and some symptoms of this condition.

Surgery and Stem Cell Transplants

Depending on your condition, you may also need a surgical or medical procedure.

With chronic myelogenous leukemia, primary myelofibrosis, and late stage polycythemia vera, the body produces blood cells in sites other than the bone marrow, such as the liver and spleen. This causes the organs to get bigger. When enlargement of the spleen becomes painful and causes complications, and when it stops responding to medical therapy, surgery to remove the spleen may be necessary. This is very rare, though, because there are so many therapeutic options available.

If you have advanced or high-risk primary myelofibrosis, you may need a stem cell transplant. In this procedure, we replace abnormal stem cells in the bone marrow with healthy stem cells. After a stem cell transplant, the healthy bone marrow cells begin to grow and produce healthy blood cells.

A stem cell transplant may also be an option for a small number of people with chronic myelogenous leukemia who do not respond to other therapies. 

A stem cell transplant can have life-threatening risks and complications. For this reason, we use it only when medical therapy is unlikely to provide long-term improvement in survival.

Nutrition and Dietary Supplements

Myeloproliferative disorders require conventional medical treatment. But you can include complementary and alternative therapies in your treatment plan as well. Ask your team of health care providers about the best ways to incorporate these therapies into your overall treatment plan. Always tell your doctor about the herbs and supplements you are using or considering using. This is important because some supplements may interfere with conventional treatments.

Following a healthy diet and getting regular exercise can help to keep your body strong while coping with a myeloproliferative disorder. Try these tips:

  • Eat antioxidant foods, including fruits (such as blueberries, cherries, and tomatoes), and vegetables (such as squash and bell peppers).
  • Avoid refined foods, such as white breads, pastas, and especially sugar.
  • Eat fewer red meats and more lean meats, cold-water fish, tofu (if you have no allergy to soy), or beans for protein.
  • Use healthy oils, such as olive oil or vegetable oil.
  • Reduce or eliminate trans-fatty acids, found in commercially baked goods such as cookies, crackers, cakes, French fries, onion rings, donuts, processed foods, and margarine.
  • Avoid caffeine, alcohol, and tobacco.
  • Drink six to eight glasses of filtered water daily.
  • Exercise at least 30 minutes daily, 5 days a week.


Pregnant women with MPNs have a higher risk of complications than others. However, many women with myeloproliferative neoplasms have had successful full-term pregnancies and healthy babies. Management of MPNs in pregnancy requires close collaboration with your hematologist and high-risk obstetrics specialist.

Possible Complications

Myeloproliferative neoplasms are chronic cancers and vary widely in how they affect patients.  The complications can be serious. Some complications include:

  • Anemia
  • Bleeding
  • Enlargement of the spleen and liver
  • Gout
  • Heart attacks or stroke
  • Infection
  • Kidney or liver failure
  • Progression from polycythemia vera or essential thrombocytosis to myelofibrosis
  • Progression to acute leukemia

Clinical Trials and Research

Clinical trials are research studies designed to assess the effectiveness of a new treatment. They may involve a new drug, immunotherapy, or vaccine therapy. Mount Sinai offers a wide range of clinical trial options. The goal is to try to improve and prolong the lives of patients with myeloproliferative disorders. These clinical trials are inspired by our research at Mount Sinai’s Myeloproliferative Disorders Research Program.