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.

 

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.
• 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®) and Pipobroman (Vercyte®).

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.

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There are clinical trials for newly diagnosed patients, for patients with advanced disease, and for patients who are intolerant of or resistant to their current medications. There is also a need for disease-modifying drugs for younger patients to stop or slow the progress of the disease and also prevent the development of a more serious blood disease. Drugs under investigation to reach these goals are included in the following list.

• Peginterferon alfa-2a (Pegasys®). In the past, the use of interferon has been restricted because it was not well tolerated by patients. Much interest, however, lies in peginterferon’s ability to induce a molecular response in some patients. A molecular response occurs when there is a reduction in the number of abnormal blood cells. New formulations of peginterferon are now under study that may be better tolerated than older forms. 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 evolving to myelofibrosis or acute myeloid leukemia. 
• Ruxolitinib (Jakafi®). While ruxolitinib has been shown to be effective in patients with polycythemia vera and primary myelofibrosis, researchers are studying the use of ruxolitinib in patients with ET who are refractory or intolerant of hydroxyurea, to see whether ruxolitinib improves platelet counts and disease-related symptoms.

Treatment Planning

Doctors consider risk factors in treatment planning for ET. These factors are:

1. Conventional Score for Prediction of Vascular Complications (European LeukemiaNet)
   • Low risk (none of the 3 major risk factors):
     • Age younger than 60 years, and
     • No history of thrombosis or major bleeding, and
     • Platelet count <1500 x10⁹ /L 
   • High risk (at least 1 of the 3 major risk factors)
     • Age 60 years or older, and/or
     • History of thrombosis or major bleed, and/or
     • Platelet count ≥1500 x10⁹ /L
        
2. International Prognostic Score for Thrombosis for Essential Thrombocythemia (ET) (IPSET-thrombosis)
   • ​​​Low Risk = 0-1 points
   • Intermediate Risk = 2 points
   • High Risk = 3-6 points
   
    Points are determined based on:
   • Patient age
     • Younger than 60 years (0 points)
     • Older than 60 years (1 point)
   • Prior thrombotic event
     • No (0 points)
     • Yes (2 points)
   • Cardiovascular risk factors present
     • No (0 points)
     • Yes (1 point)
   • JAK2 V617 mutation detected
     • No (0 points)
     • Yes (2 points)

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.

Related Links

• Chemotherapy and Other Drug Therapies
• Managing Side Effects
• Integrative Medicine and Complementary and Alternative Therapies
• Food and Nutrition
• Download or order The Leukemia & Lymphoma Society's free booklets:
  • Myeloproliferative Neoplasms
  • Understanding Side Effects of Drug Therapy