Mycophenolate Mofetil Added to Steroids for Immune Thrombocytopenia

Steroids are typical first-line choices for the treatment of immune thrombocytopenia; however, adverse events and treatment failures are common, and alternative front-line therapies are needed. In the multicenter U.K. FLIGHT trial, 120 patients with a platelet count <30×10⁹/L were randomized to mycophenolate mofetil with or without steroids (prednisolone or dexamethasone). The primary endpoint was treatment failure, defined as platelet count <30×10⁹/L and need for a second-line treatment.

Mean age was 54 years, 54% were male, and mean platelet count was 7×10⁹/L. Prednisolone was the most commonly used steroid in both treatment groups and was dosed at 1 mg/kg for 4 days, followed by 40 mg daily for 2 weeks, with further tapering (2 weeks each of 20 mg, 10 mg, and 5 mg daily, then 5 mg every other day for 2 weeks). Mycophenolate was initiated at 500 mg twice daily for 2 weeks, then increased, if tolerated, to 750 mg twice daily and eventually 1000 mg twice daily.

Treatment failures were significantly less frequent in the mycophenolate group than in the steroid-only group (22% vs. 44%; adjusted hazard ratio, 0.41; P=0.006). Treatment-related side effects were similar in the two groups. However, patients in the mycophenolate group reported worse quality of life.