Clinical overview and practical considerations for optimizing romiplostim therapy in patients with immune thrombocytopenia

Blood Rev. 2021 Sep:49:100811. doi: 10.1016/j.blre.2021.100811. Epub 2021 Feb 27.


The fundamental treatment goal for patients with immune thrombocytopenia (ITP) is reduced or ameliorated bleeding. Although various treatment options exist for the management of ITP, recent advances have led to the approval of three thrombopoietin receptor agonists (TPO-RAs; romiplostim, eltrombopag, and avatrombopag) in the United States and European Union. Current treatment guidelines for ITP indicate that medical therapy is preferred over surgical therapy and support the use of TPO-RAs as early as 3 months after disease onset. More recent data are available on the use of romiplostim in patients who have had ITP for <1 year, and romiplostim is now indicated for the treatment of adults who have not responded adequately to initial treatment, as well as children aged ≥1 year who have had ITP for ≥6 months. Here we review the role of romiplostim in the management of ITP, with a focus on efficacy and safety data, emerging data on early use (beginning within 3 months of disease onset) and treatment-free remission, and practical considerations for optimal management of ITP.

Keywords: ITP; Immune thrombocytopenia; Romiplostim; Thrombopoietin; Thrombopoietin receptor agonist.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Clinical Trials as Topic
  • Humans
  • Protein Conformation
  • Purpura, Thrombocytopenic, Idiopathic / drug therapy*
  • Receptors, Fc / administration & dosage
  • Receptors, Fc / chemistry
  • Receptors, Fc / therapeutic use*
  • Recombinant Fusion Proteins / administration & dosage
  • Recombinant Fusion Proteins / adverse effects
  • Recombinant Fusion Proteins / chemistry
  • Recombinant Fusion Proteins / therapeutic use*
  • Thrombopoietin / administration & dosage
  • Thrombopoietin / adverse effects
  • Thrombopoietin / chemistry
  • Thrombopoietin / therapeutic use*
  • Treatment Outcome


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