Background Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disorder characterised by low platelet count and may be responsible for episodes of mucocutaneous bleeding of varying severity.

Purpose The study evaluates the effectiveness and safety of rituximab in patients who have not responded to first-line treatment.

Materials and Methods We performed a retrospective observational study, between 2009–2011, in adult patients who had not responded to first-line treatment (high-dose corticosteroids or non-specific IV immunoglobulins), or who were intolerant to such alternatives. Both splenectomised and non-splenectomised patients were included. The dose employed was 375 mg/m² q7d for four weeks. We considered it a partial response if the platelet count exceeded 50 × 109 cells/L, and a complete response if the count was greater than 100 × 109 cells/L. Previous duration of thrombocytopenia, platelet counts before treatment and after 4 weeks, percentage of patients having a satisfactory response, median time to response, duration, occurrence of petechiae and mucocutaneous bleeding and tolerability of the infusion were examined.

Results We recruited 22 patients, 12 men and 10 women. The mean age of the sample was 55.6 years (range: 19–88) and a previous mean duration of ITP of 7.5 years (range 0.35 to 41). The mean platelet count before treatment was 32.8 × 109 cells/L (range: 6–70), and increased to 120x109 cells/L (range: 23–591) after completion within four weeks.

10 patients (45.5%) experienced a complete response and in 8 patients (36.3%) the response was partial, while 4 (18.2%) patients experienced no response. The mean time to get some response was 2.3 weeks (range 1–4 weeks), and it continued a median of 10.5 months (range: 1–25). Among patients who had some response, in 2 cases petechiae and bleeding were detected again, while in 3 the platelet count fell below 50x10⁹ cells/L. The other 13 patients who responded continue today with a platelet count within the target range and without clinical symptoms. The infusion was well tolerated in all cases.

Conclusions Rituximab seems an effective and well-tolerated alternative in patients with refractory ITP who require chronic treatment. This study and literature show that more than 50% of patients respond to treatment, and it may be an alter native to splenectomy. However, further prospective studies are required to define the optimal position of rituximab in the treatment of ITP.

No conflict of interest.