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

Hematology. 2016 May;21(4):257-62. doi: 10.1179/1607845415Y.0000000041. Epub 2015 Aug 7.

Abstract

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.

Keywords: Platelets; Remission; Thrombopoietin; Thrombopoietin receptor agonist.

Publication types

  • Randomized Controlled Trial

MeSH terms

  • Adult
  • Aged
  • Female
  • Follow-Up Studies
  • Humans
  • Male
  • Middle Aged
  • Purpura, Thrombocytopenic, Idiopathic / blood
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Receptors, Fc / administration & dosage*
  • Recombinant Fusion Proteins / administration & dosage*
  • Remission Induction
  • Thrombopoietin / administration & dosage*

Substances

  • Receptors, Fc
  • Recombinant Fusion Proteins
  • Thrombopoietin
  • romiplostim