A review of romiplostim mechanism of action and clinical applicability

Research output: Contribution to journalReview article


Thrombocytopenia results from a variety of conditions, including radiation, chemotherapy, autoimmune disease, bone marrow disorders, pathologic conditions associated with surgical procedures, hematopoietic stem cell transplant (HSCT), and hematologic disorders associated with severe aplastic anemia. Immune thrombocytopenia (ITP) is caused by immune reactions that accelerate destruction and reduce production of platelets. Thrombopoietin (TPO) is a critical component of platelet production pathways, and TPO receptor agonists (TPO-RAs) are important for the management of ITP by increasing platelet production and reducing the need for other treatments. Romiplostim is a TPO-RA approved for use in patients with ITP in the United States, European Union, Australia, and several countries in Africa and Asia, as well as for use in patients with refractory aplastic anemia in Japan and Korea. Romiplostim binds to and activates the TPO receptor on megakaryocyte precursors, thus promoting cell proliferation and viability, resulting in increased platelet production. Through this mechanism, romiplostim reduces the need for other treatments and decreases bleeding events in patients with thrombocytopenia. In addition to its efficacy in ITP, studies have shown that romiplostim is effective in improving platelet counts in various settings, thereby highlighting the versatility of romiplostim. The efficacy of romiplostim in such disorders is currently under investigation. Here, we review the structure, mechanism, pharmacokinetics, and pharmacodynamics of romiplostim. We also summarize the clinical evidence supporting its use in ITP and other disorders that involve thrombocytopenia, including chemotherapy-induced thrombocytopenia, aplastic anemia, acute radiation syndrome, perisurgical thrombocytopenia, post-HSCT thrombocytopenia, and liver disease.


  • James B. Bussel
  • Gerald Soff
  • Adriana Balduzzi
  • Nichola Cooper
  • Tatiana Lawrence
  • John W. Semple
External organisations
  • Weill Cornell Medical College
  • Memorial Sloan-Kettering Cancer Center
  • Hammersmith Hospital
  • Amgen Inc.
  • University of Toronto
  • San Gerardo Hospital
Research areas and keywords

Subject classification (UKÄ) – MANDATORY

  • Hematology


  • Immune thrombocytopenia, Pharmacodynamics, Pharmacokinetics, Structure, Thrombopoietin receptor agonist
Original languageEnglish
Pages (from-to)2243-2268
Number of pages26
JournalDrug Design, Development and Therapy
Publication statusPublished - 2021
Publication categoryResearch