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High-dose dexamethasone versus prednisone for treatment of adult immune thrombocytopenia: a prospective multicenter randomized trial

Yu Wei, Xue-bin Ji, Ya-wen Wang, Jing-xia Wang, En-qin Yang, Zheng-cheng Wang, Yu-qi Sang, Zuo-mu Bi, Cui-ai Ren, Fang Zhou, Guo-qiang Liu, Jun Peng and Ming Hou

Key points

  • High-dose dexamethasone is a preferred strategy to conventional prednisone as first-line management of newly diagnosed adult primary ITP.

Abstract

This study compared the efficacy and safety of high-dose dexamethasone (HD-DXM) and conventional prednisone (PDN) on the largest cohort to date as first-line strategies for newly diagnosed adult primary immune thrombocytopenia (ITP). Patients enrolled were randomized to receive dexamethasone 40 mg/d for four days (n = 95, non-responders received an additional four-day course of dexamethasone), or prednisone 1.0 mg/kg daily for four weeks and then tapered (n = 97). One or two courses of HD-DXM resulted in a higher incidence of overall initial response (82.1% vs. 67.4%, P = 0.044) and complete response (50.5% vs. 26.8%, P = 0.001) compared to prednisone. Time to response was shorter in the HD-DXM arm (P < 0.001), and a baseline bleeding score ≥ 8 was associated with a decreased likelihood of initial response. Sustained response was achieved by 40.0% of patients in the HD-DXM arm and 41.2% in the PDN arm (P = 0.884). Initial complete response was a positive indicator of sustained response, whereas presence of anti-platelet autoantibodies was a negative indicator. High-dose dexamethasone was generally tolerated better. We concluded that high-dose dexamethasone could be a preferred corticosteroid strategy for first-line management of adult primary ITP. This study is registered at http://clinicaltrials.gov as NCT01356511.

  • Submitted July 22, 2015.
  • Accepted October 13, 2015.