The application of avatrombopag in chronic liver disease patients with severe thrombocytopenia undergoing procedures in china: a prospective real-world cohort study

Background: Several guidelines recommend individualized treatment to correct severe thrombocytopenia before medium- to high-risk invasive procedures in patients with chronic liver disease, lowering the risk of bleeding. In this multi-center, prospective, non-randomized controlled cohort study, the efficacy and safety of Avatrombopag, proven safe and effective in two phase III trials, was further evaluated in China. Materials and Methods: This real-world study enrolled 341 chronic liver disease patients with severe thrombocytopenia scheduled for medium and high-risk invasive procedures across 20 sites between 10 June 2021, and 25 April 2023. Avatrombopag (40 mg/d or 60 mg/d, treatment group) or alternatives (rhTPO, rhIL-11, Chinese patent medicine or no medication treatment, control group) were administered based on the doctor’s and patient’s preferences. Homogeneity was compared between the two groups. Results: In the per-protocol set of 293 cases (66 patients of treatment group, 79 female patients), treatment group showed a higher proportion of patients not requiring transfusion or rescue treatment (90.9% vs. 63.9%, P < 0.001), exhibited more patients with a platelet count ≥50 × 10 9 /L (78.8% vs. 23.4%, P < 0.001) and higher platelet counts ([75.6 ± 42.9] × 10 9 /L vs. [46.4 ± 23.1] × 10 9 /L, P < 0.001) on the procedure day, and demonstrated lower transfusion rates (7.6% vs. 32.9%, P < 0.001), with fewer WHO ≥ 2-grade bleeding events and comparable adverse events. Conclusion: Avatrombopag effectively raises platelet counts, mitigating the need for transfusions and lowering the bleeding events in real-world scenarios of Chinese chronic liver disease patients with severe thrombocytopenia during the peri-procedure period. The study was registered at http://www.clinicaltrials.gov.