What Are Treatments For Essential Thrombocythemia?

Essential thrombocythemia (ET) is a rare chronic blood disorder characterized by the overproduction of platelets in the bone marrow. While many people live with ET for years without significant complications, others may be at risk for serious events like blood clots or bleeding. The goal of treatment is to lower that risk, especially in people with higher-risk features, and to manage symptoms that affect quality of life.

Treatment is individualized and depends on factors like age, medical history, genetic mutations, platelet levels, and whether complications such as thrombosis have occurred.

What medications are used to manage ET?

The most common way to manage ET is with medications that lower the platelet count or reduce the risk of clotting. These include:

Hydroxyurea

Often the first choice for people at higher risk of complications, hydroxyurea helps slow down the production of platelets. It is widely used due to its ability to lower both platelet and white blood cell counts, which contributes to reducing the risk of clotting and disease progression.

Anagrelide

This medication works directly on the bone marrow to reduce platelet production. It’s often used when hydroxyurea isn't a suitable option. However, when used alongside aspirin, it may slightly raise the risk of bleeding, so it's typically prescribed with care.

Interferon Alfa (especially pegylated forms)

This treatment can reduce platelet counts and inflammation without affecting DNA, making it a safer option for younger or pregnant patients. Pegylated interferon is effective and is associated with fewer side effects than earlier versions. 

Low-Dose Aspirin

Aspirin helps reduce clotting risk in many people with ET, especially those at low or intermediate risk. However, in people with extremely high platelet counts (over 1,000,000), aspirin use may be limited due to a higher risk of bleeding and acquired von Willebrand disease. In these cases, your care team may monitor clotting factors before recommending aspirin.

When Medication Isn’t Enough: Platelet Apheresis

Platelet apheresis is a procedure used to rapidly decrease platelet counts in emergency situations, such as a stroke or heart attack. It involves removing blood from the body, separating out the platelets, and then returning the rest of the blood to the body. This procedure is usually reserved for patients with severe symptoms or extremely high platelet counts. It provides temporary relief and is typically reserved for critical cases.

3. Lifestyle Changes

Supportive Care and Lifestyle Changes

While medications are key, lifestyle adjustments can also support your health and reduce the risk of complications:

• Exercise regularly to improve blood circulation and prevent clot formation
• Eat a heart-healthy diet rich in vegetables, whole grains, and lean proteins. A diet low in saturated fats can help improve overall health and reduce the risk of blood clots.
• Avoid smoking, which increases the risk of blood clots
• Limit alcohol, especially if you're on medications that may affect bleeding

What If a Treatment Isn’t Working?

If a treatment isn’t reducing your platelet count effectively or is causing side effects, your care team may consider switching to a different option. For example, people who don’t benefit from hydroxyurea may be transitioned to interferon or anagrelide. Your doctor may also evaluate emerging treatments or clinical trials if conventional therapies aren’t achieving their goals.

Keep reading How Is Essential Thrombocythemia Staged And Classified?

Sources: 

• Essential thrombocythemia: Biology, clinical features, thrombotic risk, therapeutic options and outcome
• Essential thrombocythemia: 2024 update on diagnosis, risk stratification, and management
• Essential Thrombocytosis