What are the side effects of eflornithine tablets?

Although eflornithine tablets (eflornithine) are a new type of targeted therapy drug, mainly used to treat neuroblastoma, it may still cause a series of adverse reactions related to the inhibition of polyamine metabolism during use. Its main mechanism is to interfere with cellular polyamine synthesis by irreversibly inhibiting ornithine decarboxylase (ODC). Although this mechanism effectively delays tumor growth, it may also affect the proliferation and function of normal cells, especially rapidly dividing cells, such as the hematopoietic system, gastrointestinal mucosa, and nervous system.

In clinical application, one of the most common adverse reactions of eflornithine is hematologic abnormalities, including neutropenia, anemia, and thrombocytopenia. This is because hematopoietic cells in the bone marrow are highly dependent on polyamines, and their hematopoietic ability is limited after drug intervention, which in turn leads to decreased immune function, increased risk of infection, and bleeding tendencies. Long-term use requires close monitoring of blood counts to assess the need for dose adjustment or treatment interruption.

Gastrointestinal reactions are also common, such as nausea, vomiting, diarrhea, loss of appetite, etc., which may be related to the obstruction of mucosal cell metabolism. These symptoms are usually mild, but if they persist or worsen, symptomatic treatment or intermittent use of medications should be considered. If patients experience weight loss or electrolyte imbalance, their nutritional status should be evaluated and timely intervention should be performed.

Neurological side effects may also occur in a small number of patients, especially hearing impairment, tinnitus, or mild paresthesia. This may be related toODC inhibiting polyamine levels that affect neurons. Although hearing problems have been reported with long-term use of eflornithine, the tablet version is generally less toxic than the intravenous version and the incidence is relatively manageable.

Reference materials:https://www.drugs.com/mtm/eflornithine.html