Recombinant human thrombopoietin for chronic liver disease-associated thrombocytopenia with or without concomitant infections: a real-world retrospective study

Objectives Recombinant human thrombopoietin (rhTPO) has been shown to improve platelet (PLT) counts in chronic liver disease (CLD)-associated thrombocytopenia (TP). However, whether concomitant infections influence its efficacy remains unclear. Methods We conducted a retrospective study of 259 patients with CLD-associated TP who received rhTPO at Henan Provincial People’s Hospital from January 2021 to October 2023. Patients were divided into concomitant infection (n = 178) and non-concomitant infection (n = 81) groups. Primary endpoints were the response rate and time to response. Secondary endpoints included the platelet transfusion requirements, PLT recovery differences and factors influencing rhTPO efficacy. Propensity score matching (PSM) was applied to adjust for baseline confounders. Results After 1:1 PSM (n = 132), the overall response rate was 57.6% and the median time to response was 10 days, with no serious adverse events observed. Response rates and response time were comparable between concomitant infection and non-concomitant infection groups (59.1% vs. 56.1%, P = 0.725; 10 days vs. 11 days, P = 0.442). rhTPO significantly increased PLT counts from Day 3 in patients without concomitant infection and from Day 5 in those with concomitant infection. Both groups maintained elevated levels at 14 days post-discontinuation and remained above baseline until 28 days after discontinuation. No significant differences between the two groups were observed at any time point. Patients with concomitant infection had a significant higher platelet transfusion rate than those without infections (22.7% vs. 7.6%, P = 0.015). Sensitivity analysis excluding these transfused patients showed consistent efficacy of rhTPO regardless of infection status. Multivariable analyses identified Child–Pugh C and cirrhosis as independent factors associated with response and time to response. Conclusion rhTPO was associated with improved PLT counts in CLD-associated TP without serious adverse events. This efficacy appeared comparable regardless of concomitant infection status. Liver function reserve may be the major determinant of efficacy. Prospective multicenter studies are needed to confirm these findings.