New Treatment for Low Platelet Levels in CMML

Chronic myelomonocytic leukemia (CMML) is a blood cancer that occurs when the bone marrow makes too many monocytes, a type of white blood cell. The median age at diagnosis is 72 years, and it's more common in males than females. The most common medications used to treat CMML are: azacitidine (Vidaza), decitabine (Dacogen) and Inqovi (a combination of decitabine and cedazuridine). In CMML, it is common to have low counts of other blood cell types, such as platelets, which cause symptoms like the risk of bleeding, bruising and difficulty in healing wounds. 

What Is Thrombocytopenia?

Thrombocytopenia (low platelet levels) and platelet dysfunction contribute to bleeding complications and can be life-threatening in severe cases. The most common symptoms are bleeding or bruising easily, even after minor injuries. Bleeding can occur inside the body, under the skin, or on the surface of the skin.

A New Medication To Improve Platelet Levels 

Eltrombopag is a thrombopoietin receptor agonist that stimulates platelet production in the bone marrow.  ​It is FDA approved for treatment of non-chemotherapy related disorders of low platelets like chronic immune thrombocytopenia (ITP).

Eltrombopag treats low platelet counts (thrombocytopenia) in CMML patients, as well as in adults and children with chronic immune thrombocytopenic purpura and severe aplastic anemia.

What Are the Side Effects of Eltrombopag?

Some side effects that may occur with this use of this treatment include:

• Swelling of the face, hand, lower legs or feet
• Fatigue
• Chills
• Cough
• Fever
• Nausea
• Loss of appetite
• Rash

A New Clinical Trial

A recent  phase II trial enrolled 29 patients across 15 centers who had CMML and lower-risk disease. 11 patients had had at least one prior therapy, including steroids or intravenous immunoglobulins (IVIG).

This trial lasted for 12 weeks:

• Patients received eltrombopag at a starting dose of 100 mg once daily, which was later changed to 50 mg/day.
• The Dose was adjustable up to 300 mg/day. 
• Patients responding well could continue the treatment for up to 24 months.
• This group had an overall survival rate of 47% and a two-year progression-free survival rate of 23%.

Conclusion

For those patients with CMML who are experiencing low platelet level complications, eltrombopag appears to be a new and effective treatment option when other lines of therapy have not been successful.

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Source: 

• Eltrombopag in chronic myelomonocytic leukemia with severe thrombocytopenia. A Groupe Francophone des Myélodysplasies (GFM) study