On average, individuals with essential thrombocythemia (ET) have a normal life expectancy if they are properly monitored and treated. It's important that your doctor is experienced in treating myeloproliferative neoplasms (MPNs) or works in consultation with a hematologist oncologist who has experience treating MPN patients.
Prevention of blood clots (thrombosis) and bleeding (hemorrhaging) is the main objective of treating patients with ET. For patients with low-risk disease and no symptoms, the risk of complications may be low the doctor may prescribe low-dose aspirin or no therapy at all. On the other hand, for patients with high-risk disease, doctors may prescribe low-dose aspirin and other medications to reduce high platelet counts.
|Risk Category||Patient Characteristics||Treatment Approach|
|Very low||Age 60 years or younger, no history of thrombosis, no JAK2 gene mutation||Observation only|
|Low||Age 60 years or younger, no history of thrombosis, JAK2 gene mutation||Low-dose aspirin|
|Intermediate||Older than 60 years, no history of thrombosis no JAK2 mutation||Low-dose aspirin with or without cytoreductive therapy|
|High||Older than 60 years, history of thrombosis, JAK2 mutation||Low-dose aspirin with cytoreductive therapy|
Treatment of ET may include any or a combination of the following treatments listed below:
- Low-Dose Aspirin. Low-dose aspirin may reduce the risk of clotting complications. Low-dose aspirin helps prevent platelets from sticking together, making it less likely for blood clots to form that can cause heart attacks or strokes. The most common side effects of aspirin include upset stomach and heartburn. Low-dose aspirin may also increase bleeding risk in patients with extremely high platelet counts. For these reasons, the use of aspirin in treating ET needs to be individualized.
- Cytoreductive therapy. This refers to a group of medications that reduce the number of blood cells in the bone marrow:
- Hydroxyurea (Hydrea®)
- Anagrelide (Agrylin®)
- Interferon alfa (Intron® A [alfa-2b] and Roferon®-A [alfa-2a]) and their associated sustained-release preparations PEG-Intron® ([peginterferon alfa-2b] and Pegasys® [peginterferon alfa-2a])
- Busulfan (Busulfex®, Myleran®)
For information about the drugs listed on this page, visit Drug Listings.
This is a process that uses a special machine to skim platelets from a patient’s blood and then return the remaining blood components to the patient. It is used only in emergency situations, such as acute clotting complications, when the platelet count is very high and needs to be reduced quickly. The platelet-reducing effect of this therapy is temporary.
Taking part in a clinical trial may be the best treatment choice for some ET patients. Patient participation in clinical trials is important in the development of new and more effective treatments for ET and may provide patients with additional treatment options.
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There are clinical trials for ET patients in a number of different situations, whether they are newly diagnosed, have advanced-stage disease, or are intolerant or resistant to their current medications. Sometimes, a clinical trial is the best treatment option for a patient. Researchers are currently studying:
- Specific gene mutations and cell signaling pathways involved in the development of ET, to help develop new targeted therapies for ET
- New treatments to reduce the risk of thrombosis and cardiovascular complications and to improve quality of life for ET patients
- New therapies to prevent ET from progressing to a more serious type of blood disease
- New treatments to help high-risk category ET patients live a normal life span
Drugs being researched for use in ET treatment include:
- Peginterferon alfa-2a (Pegasys®). In the past, the use of interferon was restricted because it was not well tolerated by patients. However, there is new of interest in peginterferon’s ability to induce a molecular response (remission) in some patients. This type of treatment response occurs when there is a decrease in the number of abnormal blood cells with genetic mutations. New formulations of peginterferon are being studied that may be better tolerated by patients than older formulations. Ongoing studies are comparing peginterferon alfa-2a with hydroxyurea in patients with ET. Researchers are also trying to determine if achieving a molecular response with interferon reduces the risk of ET progressing to myelofibrosis or acute myeloid leukemia.
- Ruxolitinib (Jakafi®). This drug has been shown to be effective in patients with polycythemia vera and primary myelofibrosis. Researchers are now studying the use of ruxolitinib in patients with ET who are refractory or intolerant to hydroxyurea, to see whether it decreases platelet counts and disease-related symptoms.
- Bomedemstat (IMG-7289). This drug inhibits lysine-specific demethylase 1 (LSD1), an enzyme essential for the production and function of megakaryocytes, so it prevents excess platelet production. The FDA has granted a “Fast Track” designation for the development of bomedemstat for the treatment of ET. This FDA process is designed to facilitate the development and expedite the review of drugs to treat serious conditions that fill an unmet medical need. Its purpose is to make important new drugs available to patients sooner.
- Aspirin. Researchers are evaluating the effects of aspirin regimens using two or three doses per day, compared to once-daily aspirin regimens, to prevent thrombosis in patients with intermediate-risk and high-risk ET.
Every patient’s medical situation is different and should be evaluated individually by a hematologist-oncologist who specializes in treating blood cancers. It is important for patients and the members of their medical team to discuss all treatment options, including treatments being studied in clinical trials.
To download lists of suggested questions to ask your healthcare providers, click here.
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