Myeloproliferative Neoplasms

At Montefiore Einstein Hematology (Blood Disorders), you can access exceptional care for all types of myeloproliferative neoplasms including polycythemia vera, essential thrombocytopenia and primary myelofibrosis. Our multidisciplinary team has extensive experience caring for this rare group of blood disorders. Montefiore Einstein continues to make advances in the diagnosis and treatment of blood disorders. Our team of world-renowned hematologists is passionate about uncovering the latest diagnostic approaches and treatments that can improve outcomes.

We have expertise across the entire spectrum of blood and bone marrow disorders, including stem cell transplants, precision treatments and targeted therapies (such as Janus kinase 2 inhibitors), phlebotomy or transfusions, chemotherapy, radiation therapy and surgery.

At the forefront of treatment and research for myeloproliferative neoplasms, our clinical research team ensures you have access to a robust offering of clinical trials. These trials focus on everything from immunotherapy, which harnesses the power of your own immune system, to targeted therapy based on your specific biology and CAR T-cell therapy, a leading-edge treatment that helps your body’s own immune system better fight myeloproliferative neoplasms.

When you need myeloproliferative neoplasm care, turn to our dedicated providers who will develop a highly personalized treatment plan.

The type of myeloproliferative neoplasm is based on whether too many red blood cells, white blood cells or platelets are being made. Sometimes the body will make too many of more than one type of blood cell, but usually one type of blood cell is affected more than the others are. Myeloproliferative neoplasms include:

• Chronic myeloid leukemia
• Polycythemia vera
• Essential thrombocythemia
• Vert and prefibrotic primary myelofibrosis (also called chronic idiopathic myelofibrosis)
• Chronic neutrophilic leukemia
• Chronic eosinophilic leukemia
• Myeloproliferative neoplasms unclassifiable

These types are described below. Myeloproliferative neoplasms sometimes become acute leukemia, in which too many abnormal white blood cells are made.

Tests that examine the blood and bone marrow are used to diagnose myeloproliferative neoplasms.

In addition to asking about your personal and family health history and doing a physical exam, your doctor may perform the following tests and procedures:

• Complete blood count (CBC) with differential checks a sample of blood for:
  • the number of red blood cells and platelets
  • the number and type of white blood cells
  • the amount of hemoglobin (the protein that carries oxygen) in the red blood cells
  • the amount of hematocrit (whole blood that is made up of red blood cells)
• Peripheral blood smear checks a sample of blood for:
  • whether there are red blood cells shaped like teardrops
  • the number and kinds of white blood cells
  • the number of platelets
  • whether there are blast cells
• Blood chemistry study uses a blood sample to measure the amounts of certain substances released into the blood by organs and tissues in the body. An unusual amount of a substance can be a sign of disease.
• Bone marrow aspiration and biopsy is the removal of bone marrow, blood and a small piece of bone by inserting a hollow needle into the hipbone or breastbone. A pathologist views the bone marrow, blood and bone under a microscope to look for abnormal cells.
• Cytogenetic analysis checks the chromosomes of cells in a sample of bone marrow, blood, tumor or other tissue for broken, missing, rearranged or extra chromosomes. Changes in certain chromosomes may be a sign of cancer. Cytogenetic analysis is used to help diagnose cancer, plan treatment or find out how well treatment is working.
• Genetic testing is done on a bone marrow or blood sample to check for mutations in JAK2, MPL, or CALR genes. A JAK2 gene mutation is often found in patients with polycythemia vera, essential thrombocythemia, or primary myelofibrosis. MPL or CALR gene mutations are found in patients with essential thrombocythemia or primary myelofibrosis.

There are different types of treatment for patients with myeloproliferative neoplasms.

Different types of treatments are available for patients with myeloproliferative neoplasms. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

The following types of treatment are used:

• Watchful waiting
  • Watchful waiting is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change.
• Phlebotomy
  • Phlebotomy is a procedure in which blood is taken from a vein. A sample of blood may be taken for tests such as a CBC or blood chemistry. Sometimes phlebotomy is used as a treatment and blood is taken from the body to remove extra red blood cells. Phlebotomy is used in this way to treat some myeloproliferative neoplasms.
• Platelet apheresis
  • Platelet apheresis is a treatment that uses a special machine to remove platelets from the blood. Blood is taken from the patient and put through a blood cell separator where the platelets are removed. The rest of the blood is then returned to the patient’s bloodstream.
• Transfusion therapy
  • Blood transfusion is a method of giving red blood cells, white blood cells or platelets to replace blood cells destroyed by disease or cancer treatment.
• Chemotherapy
  • Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy).
• Radiation therapy
  • Radiation therapy is a cancer treatment that uses high-energy X-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the area of the body, such as the spleen, with cancer.
• Other drug therapy
  • Prednisone and danazol are drugs that may be used to treat anemia in patients with primary myelofibrosis.
  • Anagrelide therapy is used to reduce the risk of blood clots in patients who have too many platelets in their blood. Low-dose aspirin may also be used to reduce the risk of blood clots.
  • Thalidomide, lenalidomide and pomalidomide are drugs that prevent blood vessels from growing into areas of tumor cells.
  • Erythropoietic growth factors are used to stimulate the bone marrow to make red blood cells.
• Surgery
  • Splenectomy (surgery to remove the spleen) may be done if the spleen is enlarged.
• Immunotherapy
  • Immunotherapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct or restore the body’s natural defenses against cancer.
    • Interferon: Interferon affects the division of cancer cells and can slow tumor growth. Interferon alfa and pegylated interferon alpha are commonly used to treat certain myeloproliferative neoplasms.
• Targeted therapy
  • Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells.
    • Tyrosine kinase inhibitor (TKI) therapy: TKI therapy blocks signals that cancer cells need to grow. Ruxolitinib is used to treat polycythemia vera and certain types of myelofibrosis.
• High-dose chemotherapy with stem cell transplant
  • High doses of chemotherapy are given to kill cancer cells. Healthy cells, including blood-forming cells, are also destroyed by the cancer treatment. Stem cell transplant is a treatment to replace the blood-forming cells. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the patient completes chemotherapy, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells.