Doptelet

DOPTELET (avatrombopag) is a second generation, once daily, orally administered TPO receptor agonist approved for the treatment of thrombocytopenia in adult patients with CLD who are scheduled to undergo a procedure. DOPTELET is designed to mimic the effects of TPO, the primary regulator of normal platelet production. Two global Phase 3, double-bli... nd, placebo-controlled trials (ADAPT-1 [N=231] and ADAPT-2 [N=204]), conducted in adults with thrombocytopenia (platelet count of less than 50,000/µL) and CLD, supported the FDA approval. Patients were assigned to either 40 mg or 60 mg of avatrombopag daily for five days based on their Baseline platelet counts (40 to 50,000/µmL or 40,000/µmL, respectively). Avatrombopag was shown to be superior to placebo in increasing the proportion of patients not requiring platelet transfusions or rescue procedures for bleeding up to seven days following a scheduled procedure in both trials in both the 40 mg (ADAPT-1, 88% vs. 38%, p 0.0001; ADAPT-2, 88% vs. 33%; p0.0001), and 60 mg (ADAPT-1, 66% vs. 23%, p 0.0001; ADAPT-2, 69% vs. 35%; p=0.0006) treatment groups. Avatrombopag was also superior to placebo at the two secondary efficacy endpoints in each trial. In the avatrombopag treatment groups, there was an increased proportion of patients achieving the target platelet count of 50,000/µmL on procedure day, and a greater magnitude of the change in mean platelet count from baseline to procedure day; all treatment differences between the avatrombopag and placebo treatment groups for each secondary endpoint were highly statistically significant with p values 0.0001. The most common adverse reactions with avatrombopag included pyrexia, abdominal pain, nausea, headache, fatigue and edema peripheral. Portal vein thromboses have been reported in patients with CLD and in patients receiving TPO receptor agonists. One treatment-emergent event of portal vein thrombosis was reported in the ADAPT trials in an avatrombopag-treated patient.Read More