Sirolimus-based treatment regimens for antinuclear antibody (ANA)-positive immune thrombocytopenia: a retrospective single-center cohort study

OBJECTIVES: To evaluate the efficacy and safety of sirolimus-based treatment regimens in patients with antinuclear antibody (ANA)-positive immune thrombocytopenia (ITP). METHODS: This retrospective single-center study enrolled patients with a clear clinical diagnosis of ANA-positive ITP. They were divided into two groups based on different treatment regimens: the sirolimus monotherapy group (SRL group) and the sirolimus plus dexamethasone combination group (combination group). RESULTS: 0.05); additionally, in the early treatment phase (Week 1, Week 4), there were statistically significant differences in efficacy between the two groups, while no statistically significant difference in efficacy was observed during subsequent follow-up. Regarding treatment safety, all adverse events were Grade 1-2; no Grade 3 or higher adverse events were reported and no treatment-related deaths occurred. The most frequently observed adverse events were oral ulcers, gastrointestinal distress, and elevated liver enzymes. There was no statistically significant difference in the total incidence of adverse events between the two groups. CONCLUSION: In this retrospective single-center study, sirolimus-based regimens showed favorable efficacy and acceptable safety in ANA-positive ITP. Adding dexamethasone to sirolimus led to faster initial responses versus sirolimus alone, though no significant difference was seen in follow-up. These findings support the feasibility and potential efficacy of sirolimus-based regimens in ANA-positive ITP, warranting prospective controlled studies.