First drug approved to treat thrombocytopenia in adults with chronic liver disease who are having a medical procedure

Patients who received the drug for five days had increased platelet counts compared to those taking placebo.

The FDA recently approved avatrombopag (Doptelet) tablets to treat adults with chronic liver disease who have thrombocytopenia and are scheduled to have a medical or dental procedure, the agency announced on May 21. The medication is the first to be approved by the FDA for this indication.

The drug, which received priority review, was studied in two trials of 435 patients with chronic liver disease and severe thrombocytopenia who were about to have a procedure that would typically require platelet transfusion. Compared to participants who received placebo, a higher proportion of those who received oral avatrombopag for five days had increased platelet counts and did not need platelet transfusion or any rescue therapy on the day of the procedure and up to seven days later.

The most common side effects of the drug were fever, abdominal pain, nausea, headache, fatigue, and edema. The drug may also increase the risk of blood clots in people with chronic liver disease or certain blood clotting conditions.