From the Guidelines
The initial treatment for essential thrombocythemia typically involves low-dose aspirin (81-100 mg daily) and cytoreductive therapy for high-risk patients, with hydroxyurea being the first-line cytoreductive agent, as recommended by the most recent guidelines 1. The treatment approach for essential thrombocythemia (ET) is risk-stratified, based on factors such as age, history of thrombosis, and cardiovascular risk factors.
- For high-risk patients, cytoreductive therapy with hydroxyurea is recommended, usually started at 500-1000 mg daily and adjusted to maintain platelet counts below 400,000/μL 1.
- For low-risk patients without cardiovascular risk factors, aspirin alone may be sufficient, with a dose of 81-100 mg daily 1.
- Anagrelide (starting at 0.5 mg twice daily) or interferon-alpha are alternative options for patients who cannot tolerate or have failed hydroxyurea therapy 1. The goal of treatment is to reduce the risk of thrombotic and hemorrhagic complications by controlling platelet counts, and treatment decisions should be based on the most recent guidelines and patient-specific factors 1. Regular blood count monitoring is essential to adjust medication dosages and prevent cytopenias, and treatment is typically lifelong as essential thrombocythemia is a chronic myeloproliferative neoplasm characterized by excessive platelet production in the bone marrow 1. It is also important to manage cardiovascular risk factors and advise patients to stop smoking, as these factors can impact the risk of thrombotic complications 1. Overall, the treatment approach for essential thrombocythemia should be individualized and based on the most recent guidelines and patient-specific factors, with the goal of reducing morbidity, mortality, and improving quality of life 1.
From the Research
Essential Thrombocythemia Treatment
The initial treatment for essential thrombocythemia (ET) depends on the patient's risk category, which is determined by factors such as age, previous thrombosis, and platelet count.
- Low-risk patients (age ≤60 years, no thrombosis history, and platelet count <1,500 x 10^9/L) can be observed or treated with low-dose aspirin (100-300 mg/day) 2, 3.
- High-risk patients (age >60 years, previous thrombosis, or platelet count >1,500 x 10^9/L) should be treated with hydroxyurea, plus aspirin in the case of thrombosis, to prevent thrombotic complications 2, 3.
- Intermediate-risk patients may be treated with anagrelide, hydroxyurea, or interferon-alpha, depending on their specific risk factors and clinical presentation 3, 4.
Treatment Goals
The primary goals of treatment for ET are to:
- Prevent thrombosis and bleeding
- Treat ET-related symptoms
- Improve quality of life
- Minimize the risk of malignant transformation and/or post-ET myelofibrosis 4, 5
Risk Stratification
Patients with ET can be stratified into four risk categories:
- Very low risk (age ≤60 years, no thrombosis history, and JAK2 wild-type)
- Low risk (same as very low risk but JAK2 mutation present)
- Intermediate risk (same as low risk but age >60 years)
- High risk (thrombosis history or age >60 years with JAK2 mutation) 5
Cytoreductive Therapy
Cytoreductive therapy is advised for high-risk and optional for intermediate-risk disease. First-line cytoreductive drugs of choice are hydroxyurea and pegylated interferon-α, and second-line busulfan 5.
Aspirin Therapy
Once-daily low-dose aspirin is advised for all patients, and twice daily for low-risk disease 5.
New Investigational Drugs
New investigational drugs are being developed for the treatment of ET, including specific treatment strategies in the context of extreme thrombocytosis, pregnancy, splanchnic vein thrombosis, perioperative care, and post-essential thrombocythemia myelofibrosis 5.