Oprelvekin

Based on real-world research, we aimed to systematically evaluate the efficacy and safety of hetrombopag for the treatment of chemotherapy-induced thrombocytopenia (CIT) in patients with solid tumors. Patients with solid tumors who developed CIT (platelet count < 100 × 10⁹/L) and were treated with hetrombopag in a single hospital between February 2022 and September 2023 were included in the study. The primary outcome was complete response rate within 14 days, defined as the proportion of patients with platelet counts of ≥100 × 10⁹/L or platelet counts increased by at least 50 × 10⁹/L from baseline. Response rate within 21 days, the incidence of chemotherapy intensity reduction, the median time of response, and adverse events were reported. A total of 73 patients met the inclusion criteria and were subsequently included in the analysis. The complete response rate within 14 days was 79.5%. Within 21 days, the complete response rate was 91.8%. The incidence of chemotherapy intensity reduction was 21.9%. The median time to platelet response was 9.0 days (95% CI, 8.3-9.7 days). The baseline platelet count of ≥ 50 × 10⁹/L and the treatment regimen of hetrombopag combined with rhTPO/rhIL-11 were identified as independent favorable prognostic factors for platelet response time. Subgroup analyses demonstrated that patients receiving combination regimen exhibited a significantly reduced median time to platelet response with baseline platelet counts of ≥50 × 10⁹/L. Safety profile showed good tolerability of hetrombopag (monotherapy or combined with rhTPO/rhIL-11) in patients. Hetrombopag may be an effective and well-tolerated treatment option for CIT in patients with solid tumors.

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.

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/day or 60 mg/day, 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.

In the per-protocol set of 293 cases (66 patients of the treatment group, 79 female patients), the 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 of ≥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.

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.