Mycophenolate Mofetil for First-Line Treatment of Immune Thrombocytopenia

This open-label RCT (n=120) found the addition of mycophenolate to glucocorticoid in the first-line treatment of immune thrombocytopenia was associated with a lower risk of treatment failure (22% v 44%; HR 0.41; range 0.21-0.80; p=0.008) but decreased quality of life.

SPS commentary:

An accompanying editorial notes that the quality of life results suggesting the combination of mycophenolate and glucocorticoid were associated with greater fatigue and poorer physical health is an important reminder that immune thrombocytopenia is not just about the platelet count, and that such RCTs can produce unexpected findings that warrant further research.

Source:

New England Journal of Medicine

Resource links:

Editorial