Many Myelodysplastic Syndrome (MDS) patients struggle with low blood platelet counts, a condition called thrombocytopenia. Platelets help your blood clot, preventing excessive bleeding. When your platelet count drops below 150,000 per microliter (mcL), you're at a higher risk of bruising, bleeding episodes, and even internal bleeding or heart attack.

While anemia is more common in MDS, at least 10% of patients experience thrombocytopenia. Traditional treatments like platelet transfusions often provide only short-term relief, and patients can become resistant (disease recurrence) over time.

Eltrombopag, a medication currently undergoing clinical trials for low-risk MDS, offers new hope. It works by stimulating the production of platelets in your bone marrow. Studies have shown Eltrombopag to be safe and effective in:

• Rapidly increasing platelet count: This can significantly reduce your risk of bleeding and improve your quality of life.
• Not increasing bone marrow blasts: This is important because blasts are abnormal cells that can progress to leukemia.

Eltrombopag's clinical trial results

A Phase 2 clinical trial (NCT02912208) investigated Eltrombopag's effectiveness for thrombocytopenia in MDS patients. Here are some encouraging results:

• Nearly half (47.5%) of patients experienced a significant increase in platelet count.
• Only a small percentage (8.5%) had their disease progress to Acute Myeloid Leukemia (AML).
• Over half (59.3%) of patients achieved overall survival at 2 and 5 years.

These findings suggest that Eltrombopag can be a well-tolerated and effective treatment for low-platelet MDS.

Eltrombopag Side Effects

Eltrombopag is generally well-tolerated, but some side effects can occur. It's important to be aware of these and discuss them with your doctor.

Serious Side Effects:

• Liver problems: nausea, vomiting, dark urine, stomach pain, yellow eyes or skin.
• Blood clots: pain, swelling, and tenderness in your leg.
• Cataracts: Eltrombopag may cause or worsen cataracts.

Common Side Effects:

• Swelling in the face, arms, legs, or feet.
• Body aches, chills, or cough.
• Difficulty breathing, fever, or headache.
• Weight gain, sore throat, tiredness, or weakness.
• Tingling in hands and feet.

Eltrombopag offers a promising new approach for managing low platelet counts in MDS. Studies suggest it can effectively increase platelet production, reduce bleeding risk, and improve quality of life without worsening the underlying disease. Remember, Eltrombopag is still undergoing clinical trials, and more information may become available in the future. 

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Source: 

• Eltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS)