Mycophenolate mofetil treats immune thrombocytopenia

For adults with immune thrombocytopenia, the addition of mycophenolate mofetil to a glucocorticoid for first-line treatment is associated with fewer treatment failures and higher response rates than glucocorticoid-only treatment, according to a study published in the Sept. 2 issue of the New England Journal of Medicine.

Charlotte A. Bradbury, M.D., Ph.D., from the University of Bristol in the United Kingdom, and colleagues conducted an open-label, randomized trial involving adults with immune thrombocytopenia. One hundred twenty participants were randomly assigned to first-line treatment either with a glucocorticoid only or with a glucocorticoid plus mycophenolate mofetil (61 and 59 patients, respectively). Participants were followed for up to two years after beginning trial treatment.

The researchers found that fewer treatment failures occurred in the mycophenolate mofetil group versus the glucocorticoid-only group (22 versus 44 percent; hazard ratio, 0.41), and the response was greater (91.5 versus 63.9 percent of patients had platelet counts greater than 100 x 109/liter). There was no evidence of a between-group difference in the occurrence of bleeding, rescue treatment, or treatment side effects, including infection. Worse quality-of-life outcomes relating to physical function and fatigue were seen for patients in the mycophenolate mofetil group versus the glucocorticoid-only group.

“A note of caution is needed: quality-of-life studies showed greater fatigue and poorer physical health in the mycophenolate mofetil group, an important reminder that immune thrombocytopenia is not just about the platelet count and that randomized trials can produce unexpected findings that warrant further research,” write the authors of an accompanying editorial.