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Mycophenolate mofetil for first-line treatment of immune thrombocytopenia

Bradbury, Charlotte A., Pell, Julie, Hill, Quentin, Bagot, Catherine, Cooper, Nichola, Ingram, Jenny, Breheny, Katie, Kandiyali, Rebecca, Rayment, Rachel, Evans, Gillian, Talks, Kate, Thomas, Ian and Greenwood, Rosemary 2021. Mycophenolate mofetil for first-line treatment of immune thrombocytopenia. New England Journal of Medicine 385 (10) , pp. 885-895. 10.1056/NEJMoa2100596

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BACKGROUND Immune thrombocytopenia is a rare autoimmune disorder with associated bleeding risk and fatigue. Recommended first-line treatment for immune thrombocytopenia is high-dose glucocorticoids, but side effects, variable responses, and high relapse rates are serious drawbacks. METHODS In this multicenter, open-label, randomized, controlled trial conducted in the United Kingdom, we assigned adult patients with immune thrombocytopenia, in a 1:1 ratio, to first-line treatment with a glucocorticoid only (standard care) or combined glucocorticoid and mycophenolate mofetil. The primary efficacy outcome was treatment failure, defined as a platelet count of less than 30×109 per liter and initiation of a second-line treatment, assessed in a time-to-event analysis. Secondary outcomes were response rates, side effects, occurrence of bleeding, patient-reported quality-of-life measures, and serious adverse events. RESULTS A total of 120 patients with immune thrombocytopenia underwent randomization (52.4% male; mean age, 54 years [range 17 to 87]; mean platelet level, 7×109 per liter) and were followed for up to 2 years after beginning trial treatment. The mycophenolate mofetil group had fewer treatment failures than the glucocorticoid-only group (22% [13 of 59 patients] vs. 44% [27 of 61 patients]; hazard ratio, 0.41; range, 0.21 to 0.80; P=0.008) and greater response (91.5% of patients having platelet counts greater than 100×109 per liter vs. 63.9%; P<0.001). We found no evidence of a difference between the groups in the occurrence of bleeding, rescue treatments, or treatment side effects, including infection. However, patients in the mycophenolate mofetil group reported worse quality-of-life outcomes regarding physical function and fatigue than those in the glucocorticoid-only group. CONCLUSIONS 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.

Item Type: Article
Date Type: Published Online
Status: Published
Schools: Medicine
Centre for Trials Research (CNTRR)
Publisher: Massachusetts Medical Society
ISSN: 0028-4793
Funders: NIHR
Last Modified: 31 Jan 2022 08:12

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