What is the role of romiplostim in the treatment of aplastic anemia?

Updated: Jan 29, 2021
  • Author: Sameer Bakhshi, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Romiplostim is a peptibody (Fc antibody fusion protein) with thrombopoietin receptor (also known as c-MPL) agonist activity that stimulates endogenous thrombopoietin production, promoting the proliferation and differentiation of megakaryocytes in the bone marrow. As an alternative to eltrombopag in frontline treatment for severe aplastic anemia, romiplostim is still under evaluation in ongoing trials (NCT03957694). In an open-label randomized phase 2 trial in refractory aplastic anemia patients previously treated with IST, romiplostim at dose of 10 mcg/kg/week showed responses in around 70% patients at 9 weeks [111] . Present studies are focusing on effectiveness of high-dose romiplostim (20 mcg/kg/week) in eltrombopag-refractory aplastic anemia. [112]


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