Although myeloproliferative neoplasms usually cannot be cured, there are treatments for all patients with the condition. Treatment of MPNs depends on the type and on the presence of symptoms. In general, treatment aims to correct the abnormal blood counts.
Some people don't experience any symptoms and therefore don't require treatment.
Low dose chemotherapy pills, such as hydrea, can be given to patients with essential thrombocythemia and polycythemia vera to lower their platelet and red blood cell counts. Phlebotomy (the removal of blood intravenously) is also an effective strategy for lowering red blood cell counts and controlling symptoms.
The treatment of choice for chronic myelogenous leukemia is a new anti-leukemia medication, called Gleevec. Testoterone therapy can sometimes improve anemia in patients with myelofibrosis. Other potential therapies for myelofibrosis include chemotherapy, radiation therapy, thalidomide and alpha interferon. In addition, patients with myelofibrosis frequently require red blood cell transfusions.
Chemotherapy uses drugs to kill extra blood cells in the body. Chemotherapy may be taken orally in a pill format or may be administered intravenously (through an IV). Chemotherapy is called a systemic treatment because the drug enters the bloodstream, travels through the body and can kill cells throughout the body.
Radiation therapy uses high-energy X-rays to kill cells and is sometimes used to relieve symptoms of MPN. External beam radiation therapy, which uses a machine outside the body to administer the radiation, is usually the type of radiation therapy used to treat MPN. In addition, a radioactive drug called P32 can be given intravenously to lower the number of red blood cells made by the bone marrow.
Surgery to remove the spleen, called a splenectomy, may be performed if a patient's spleen is swollen.
Biological therapy is being tested as a possible treatment of MPN. This type of therapy uses materials made either by the body or in a laboratory to boost, direct or restore the body's immune system. Biological therapy sometimes is called biological response modifier (BRM) therapy or immunotherapy.
Hormones may be used in certain instances to treat side effects of MPN. In patients with agnogenic myeloid metaplasia, hormones called glucocorticoids may be given to increase the life span of red blood cells. In addition, hormones called androgens sometimes are used to stimulate the bone marrow to produce more blood cells.
A person's recovery or response to therapy depends on their type of MPN, age and general health. Most people with essential thrombocythemia and polycythemia vera live more than 10 to 15 years with few complications. People with myelofibrosis live approximately five years and in some cases, the disease may develop into acute leukemia.
Patients with advanced MPN may qualify for a number of experimental therapies including stem cell transplantation. The only curative therapy for MPN (excluding chronic myelogenous leukemia) is allogeneic stem cell transplantation, which is the transfer of stem cells from one person to another.
There are several experimental studies open at UCSF Medical Center utilizing stem cell transplantation in patients with MPN. Whether stem cell transplantation is an appropriate therapy for an individual patient depends on a number of factors including age, MPN subtype, current symptoms or the extent of disease and the availability of a donor. People are encouraged to consult with a stem cell transplantation doctor to learn more about MPN, possible treatments and stem cell transplantation.
Reviewed by health care specialists at UCSF Medical Center.