Mycophenolate Mofetil Use in Pediatric Immune Thrombocytopenia Refractory to First-line Therapy: a Single-center Experience.

Most children treated for immune thrombocytopenia remit during the first year following diagnosis. For the ∼40% who develop persistent or chronic disease, second-line treatment options include immunomodulation and thrombomimetic agents. While immunomodulators target the underlying mechanism, prolonged immunosuppression may increase the risk of infection. We report the use of the reversible immunomodulating agent mycophenolate mofetil (MMF) in 16 pediatric patients with immune thrombocytopenia refractory to first-line treatment. Using escalating doses up to 2400 mg/m2/d, MMF treatment resulted in a 73% response rate. Adverse events were mostly mild and tolerable. Complete responders have been successfully tapered off MMF with sustained responses.