Mycophenolate Mofetil for First-Line Treatment of Immune Thrombocytopenia

In the September 2, 2021 article of the New England Journal of Medicine Charlotte Bardbury and colleagues report on the results of mycophenolate mofetil for first-line treatment of immune thrombocytopenia


ITP is a rare autoimmune disorder leading to not only excessive bleeding and bruising but fatigue as well. Steroids are first-line treatment but are associated with numerous adverse events. The purpose of the study was to assess the efficacy as well as adverse event profile of mycophenolate in the first-line setting for ITP.


The trial was a multicenter, open-label, randomized, controlled study conducted in the UK, where adult patients with ITP were signed in a 1:1 fashion, to first-line treatment with a glucocorticoid-only therapy or combined glucocorticoid -therapy plus mycophenolate mofetil. The primary outcome was treatment failure, which investigators defined as a platelet count of less than 30×10^9 / L and initiation of a second-line therapy, assessed in a time-to-event analysis. Secondary outcomes were response rate, side effect profile, bleeding occurrence, patient-reported QOL measures, and serious adverse events.


120 patients underwent randomization (52.4% male; mean age, 54 years [range 17 – 87]; mean platelet level, 7×10^9 per liter) and were followed for up to 2 years after beginning treatment. The mycophenolate mofetil group had fewer treatment failures than the glucocorticoid-only group (22% [13 / 59 patients] vs. 44% [27 / 61 patients]; HR , 0.41; range, 0.21 – 0.80; p=0.008) and higher response (91.5% of patients with platelet counts > 100×10^9 / L vs. 63.9%; p<0.001). The investigators found no evidence of a difference between the treatment groups with regards to bleeding occurrence, rescue treatments, or treatment side effects, including infection. The investigators also noted that patients in the mycophenolate treatment arm reported worse QOL outcomes regarding physical function and fatigue than those in the steroid treatment arm.


The investigators concluded “the addition of mycophenolate mofetil to a glucocorticoid for first-line treatment of immune thrombocytopenia resulted in greater response and a lower risk of refractory or relapsed immune thrombocytopenia, but with somewhat decreased quality of life.”