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Myeloproliferative Neoplasm

Myeloproliferative neoplasms (MPNs) are a group of blood cancers in which the bone marrow produces too many blood cells. When this happens, patients are at risk for blood clotting or bleeding, bone marrow scarring and enlargement of the organs. Most patients affected are in their 60s or older.

There are no known cures for most MPNs, although stem cell transplants can help people with myelofibrosis. Still, many patients can experience few or no symptoms for long periods of time, and people with these cancers can live for a long time with proper monitoring and treatment.

Types of MPNs

Essential Thrombocytosis (ET) 

Essential thrombocytosis is a rare disorder in which the body produces too many platelets for unknown reasons. This can cause abnormal blood clotting or bleeding. The condition usually affects people in middle age, although it can be seen in younger patients. A small number of patients may develop acute leukemia or a bone marrow disorder called myelofibrosis.

Treatments

Treatments for ET can vary depending on a patient’s symptoms. Some patients may not need active treatment but should still be monitored by a physician knowledgeable about ET. Treatments include:

  • Low-dose aspirin – This may be given to reduce the risk of blood clots.
  • Platelet-lowering medications – Drugs including hydroxyurea, anagrelide, and interferon may be prescribed to reduce the risk of bleeding or clotting complications.
  • Blood transfusions – This increases the number of normal cells in circulation.
  • Plateletpheresis – Following an emergency such as a stroke or severe clotting issue, this procedure can quickly lower the number of platelets. Blood is removed through a tube, stripped of platelets, and returned to the body through another tube.
  • Bone marrow transplant/stem cell transplant – A bone marrow transplant gives a patient healthy stem cells—immature cells that grow into different parts of the blood. The cells are taken from the marrow (the soft, fatty tissue inside bones), replacing the diseased bone marrow. Most patients receive high doses of chemotherapy, radiation, or both before the transplant to kill any remaining cancer cells and make room for new stem cells to grow. Stem cells are then delivered through a tube injected into the bloodstream. They find their way into the bone marrow and begin reproducing to make healthy new blood cells.
  • Novel therapies – Investigators are developing and testing potential new treatments for ET. One is pegylated interferon, another version of interferon that has fewer side effects.
  • Chemotherapy – Because MPNs can progress to acute leukemia, patients often are treated with regimens used in that disease. Chemotherapy reduces the amount of abnormal stem cells and allows healthy ones to grow back. 

Polycythemia Vera (PV)

Polycythemia vera is a rare blood disorder in which there is an increase in all blood cells, particularly red blood cells. This makes the blood thicker and can lead to strokes or tissue and organ damage. It is caused by a genetic change, or mutation. In most cases, it is not known why this happens. A small number of patients may develop acute leukemia or myelofibrosis.

Treatments

Treatments for PV can vary depending on a patient’s symptoms. Some patients may not need active treatment but should still be monitored by a physician knowledgeable about PV. Treatments include:

  • Low-dose aspirin – This may be given to reduce the risk of blood clots.
  • Phlebotomy – A procedure that involves removing blood from the body. It can thin the blood to let it flow more easily.
  • Radiation – to help suppress overactive bone marrow cells. This therapy helps lower the red blood cell count and keeps blood flow and blood thickness closer to normal.
  • Medications – Drugs including hydroxyurea and ruxolitinib may be prescribed to reduce the risk of bleeding or clotting complications, and decrease the occurrence of an enlarged spleen.
  • Novel therapies – Investigators are developing and testing potential new treatments for PV. One is pegylated interferon, another version of interferon that has fewer side effects.
  • Blood transfusions – This increases the number of normal cells in circulation.
  • Bone marrow transplant/stem cell transplant – A bone marrow transplant gives a patient healthy stem cells—immature cells that grow into different parts of the blood. The cells are taken from the marrow (the soft, fatty tissue inside bones), replacing the diseased bone marrow. Most patients receive high doses of chemotherapy, radiation, or both before the transplant to kill any remaining cancer cells and make room for new stem cells to grow. Stem cells are then delivered through a tube injected into the bloodstream. They find their way into the bone marrow and begin reproducing to make healthy new blood cells.
  • Chemotherapy – Because MPNs can progress to acute leukemia, patients often are treated with regimens used in that disease. Chemotherapy reduces the amount of abnormal stem cells and allows healthy ones to grow back.

Search for ongoing clinical trials

Myelofibrosis (MF)

Myelofibrosis is a chronic blood cancer in which the bone marrow function is affected by scarring. MR can occur either as a progression of ET or PV, or in patients with no prior history of an MPN.

Treatments

Treatments for MF can vary depending on a patient’s symptoms. Some patients may not need active treatment but should still be monitored by a physician knowledgeable about MF. Treatments include:

  • Low-dose aspirin – This may be given to reduce the risk of blood clots.
  • Medications – Drugs including hydroxyurea, ruxolitinib and fedratinib may be prescribed to reduce the risk of bleeding or clotting complications, and decrease the occurrence of an enlarged spleen.
  • Novel therapies – Investigators are developing and testing potential new treatments for MF. This includes medications that inhibit the JAK2 cell signaling pathway, drugs to treat anemia and medicines that change the ways genes are organized to make them more or less accessible for use.
  • Blood transfusions – This increases the number of normal cells in circulation.
  • Bone marrow transplant/stem cell transplant – A bone marrow transplant is the only curative treatment for MF. It gives a patient healthy stem cells—immature cells that grow into different parts of the blood. The cells are taken from the marrow (the soft, fatty tissue inside bones), replacing the diseased bone marrow. Most patients receive high doses of chemotherapy, radiation, or both before the transplant to kill any remaining cancer cells and make room for new stem cells to grow. Stem cells are then delivered through a tube injected into the bloodstream. They find their way into the bone marrow and begin reproducing to make healthy new blood cells.
  • Chemotherapy – Because MPNs can progress to acute leukemia, patients often are treated with regimens used in that disease. Chemotherapy reduces the amount of abnormal stem cells and allows healthy ones to grow back.
  • Other therapies may be used to treat specific symptoms of MF. This includes radiation therapy for enlargement of the spleen.

MPN Experts

Patients are seen in a multidisciplinary clinic within the Center for Bone Marrow Failures, where patients with difficult cases are discussed by a group of experts who work together to develop short- and long-term goals for the overall best treatment plan. MDS patients are frequently enrolled in ongoing clinical trials. Patients with aggressive forms of MDS that do not respond to traditional chemotherapy have access to innovative leukemia regimens being tested in clinical trials. And some patients may be eligible for potentially curative stem cell transplants, even if they would not normally be on a transplant list because of their age or lack of a donor. Our experts include:

 
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