Real-world efficacy and safety of avatrombopag in Japanese patients with chronic liver disease and severe thrombocytopenia.

BACKGROUND

This multicenter retrospective study evaluated the efficacy and safety of avatrombopag (AVA) in Japanese patients with chronic liver disease (CLD) and severe thrombocytopenia in a real-world clinical setting.

METHODS

The study included CLD patients with platelet counts < 50,000/μL, who received AVA before invasive procedures. Platelet-increasing effect and associated factors were examined, and adverse events were investigated.

RESULTS

Sixty-three patients were included, with 10 repeat registrations for different procedure. Thus, the efficacy of AVA was evaluated in 53 unique patients at initial registration, while the comparison based on prior thrombopoietin receptor agonist (TPO-RA) treatment history was conducted in all 63 patients. Patients were divided by baseline platelet count (BPC) into Low BPC (< 40,000/μL; n = 24) and High BPC (≥ 40,000/μL; n = 29) groups. The median platelet counts at baseline and before procedures were 43,000/μL and 75,000/μ, respectively. A platelet increase of ≥ 20,000/μL from baseline was observed in 37 patients (69.8%), and no significant differences were found between the Low BPC and High BPC groups. In addition, a platelet count of ≥ 50,000/μL was achieved in 49 (92.5%) before procedures and 51 (96.2%) avoided platelet transfusions. A multivariate analysis identified body weight > 68 kg as the only significant factor associated with platelet count increase < 20,000/μL. Platelet-increasing effects did not differ by TPO-RA administration history. No adverse events, including portal vein thrombosis or hemorrhagic events, occurred.

CONCLUSIONS

While interpretation requires caution due to the small sample size, in Japanese real-world practice, AVA was relatively safe and effective in preventing perioperative bleeding and avoiding platelet transfusions.