Which medications in the drug class Hematopoietic Growth Factors are used 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

Hematopoietic Growth Factors

Eltrombopag has gained FDA approval for severe aplastic anemia. It is indicated for first-line treatment, in combination with standard immunosuppressive therapy, and as second-line treatment in patients who fail to respond adequately to at least 1 prior immunosuppressive therapy.

Several preliminary studies have demonstrated that the addition of cytokines (eg, granulocyte colony-stimulating factor [G-CSF], granulocyte-macrophage colony-stimulating factor [GM-CSF]) may hasten the neutrophil recovery and that these agents may improve response rate and survival, although long-term use may increase the risk of clonal evolution.

Eltrombopag (Promacta)

Eltrombopag is a thrombopoietin (TPO)-receptor agonist that interacts with human TPO receptor transmembrane domain of human TPO-receptor. It initiates signaling cascades that induce proliferation and differentiation of megakaryocytes from bone marrow progenitor cells. It is indicated for severe aplastic anemia in patients who fail to respond adequately to at least 1 prior immunosuppressive therapy.

Sargramostim (Leukine)

A recombinant human GM-CSF, sargramostim can stimulate production of neutrophils and activate mature granulocytes and macrophages. The dose and frequency of administration vary with the investigational protocol.

Filgrastim (Neupogen)

Filgrastim is a G-CSF that activates and stimulates the production, maturation, migration, and cytotoxicity of neutrophils.


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