Treatment

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 Factors 

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 


Drug 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.

Plateletpheresis

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. 

Clinical Trials

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. 

Click here to read more about clinical trials.

Receive one-on-one navigation from an LLS Clinical Trial Specialist who will personally assist you throughout the entire clinical-trial process: Click Here

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.
 

Treatment Planning

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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