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Mycophenolate Mofetil for First-Line Treatment of Immune Thrombocytopenia
Charlotte A Bradbury1, Julie Pell1, Quentin Hill1
1From the Faculty of Translational Health Sciences (C.A.B.), University of Bristol (J.I., K.B., R.K.), and the Bristol Haematology and Oncology Centre (C.A.B.) and the Research Design Service (R.G.), University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, the Centre for Trials Research, Cardiff University (J.P., I.T.), and the Department of Haematology, Cardiff and Vale University Health Board (R.R.), Cardiff, Leeds Teaching Hospitals NHS Trust, Leeds (Q.H.), Glasgow Royal Infirmary, Glasgow (C.B.), the Department of Immunology and Inflammation, Imperial College London, London (N.C.), East Kent Hospitals University NHS Foundation Trust, Canterbury (G.E.), and Newcastle upon Tyne Hospitals NHS Trust, Newcastle upon Tyne (K.T.) - all in the United Kingdom.
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. (Funded by the U.K. National Institute for Health Research; FLIGHT ClinicalTrials.gov number, NCT03156452; EudraCT number, 2017-001171-23.).
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