Background: Antinuclear antibodies (ANAs) can be detected in about 30% of patients with primary immune thrombocytopenia (ITP), yet their relationship with treatment response to rituximab remains elusive.Methods: we retrospectively reviewed the clinical records of hospitalized adult ITP patients who were treated with rituximab from three medical centers across China. Rituximab was given intravenously at 100 mg weekly for 4 weeks, or at a single dose of 375 mg/m2. All included patients had their ANAs tested before rituximab treatment.Results: A total of 287 patients fulfilled the inclusion criteria and were eligible for analysis. ANAs were positive in 98 (34.1%) of the included patients. The incidence of overall response and complete response (CR) in ANA-positive patients was significantly higher than that in ANA-negative patients (overall response: 76.5% vs. 55.0%, P < 0.001; CR: 46.9% vs. 29.1%, P = 0.003). However, sustained response (SR) rates in ANA-positive patients at 6, 12 and 24 months were all lower compared with ANA-negative patients (all P < 0.05). The overall duration of response (DOR) estimated by Kaplan-Meier analysis in ANA-negative patients was greater than that in ANA-positive patients (P < 0.001).Conclusion: ITP patients with positive ANA test were likely to achieve a better initial response to rituximab treatment, while their long-term outcome was unfavorable. Therefore, ANA test could be useful for predicting rituximab response in ITP.
Keywords: Antinuclear antibodies (ANAs); primary immune thrombocytopenia (ITP); rituximab.