Mycophenolate mofetil for first-line treatment of immune thrombocytopenia

New England Journal of MedicineBradbury CA, Pell J, Hill Q, et al. | September 03, 2021


For first-line treatment of immune thrombocytopenia, administration of mycophenolate mofetil along with a glucocorticoid, vs glucocorticoid only, linked with a greater response and a lower risk of refractory or relapsed immune thrombocytopenia.

  • A multicenter, open-label, randomized, controlled trial.

  • A total of 120 patients with immune thrombocytopenia were assigned to receive first-line treatment with a glucocorticoid only or combined glucocorticoid and mycophenolate mofetil.

  • Fewer treatment failures (22% [13 of 59 patients] vs 44% [27 of 61 patients]) and greater response (91.5% of patients having platelet counts greater than 100×109 per liter vs 63.9%) were recorded in the mycophenolate mofetil group vs the glucocorticoid-only group.

  • However, patients described somewhat reduced quality of life regarding physical function and fatigue in correlation with receiving combination treatment.

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