Case study of remission in adults with immune thrombocytopenia following cessation of treatment with the thrombopoietin mimetic romiplostim.

Overview

OBJECTIVES: In adults, immune thrombocytopenia (ITP), characterized by platelet counts <100 × 10(9)/l, is typically chronic, with remission reported infrequently ≥3 years post-diagnosis. The thrombopoietin mimetic romiplostim increases platelet counts and reduces use of concomitant ITP medications in chronic ITP. While often perceived as a long-term treatment, dose-adjustment rules allow romiplostim to be discontinued when hemostatic platelet counts are reached, as reported in Amgen trials. METHODS: Eight romiplostim trials were examined for remission, defined as ≥26 consecutive weeks of platelets ≥50 × 10(9)/l without treatment. RESULTS: Remission was identified in 27 patients; median (quartile 1 [Q1], quartile 3 [Q3]) ITP duration of 2.1 (0.5, 4.2) years, 17/27 (63%) having ITP for >1 year, mean baseline platelets 20.9 × 10(9)/l, median preremission maximum dose 3.0 µg/kg, 12/27 (44%) were splenectomized at baseline, and there were 40-276 cumulative weeks of romiplostim with median time to remission 7.1 months. DISCUSSION/CONCLUSION: No clear-cut predictors of remission were apparent; however, a number of patients had ITP for <1 year and received romiplostim for <1 year.