What is the role of the granulocyte colony-stimulating factor (GCSF) in the treatment of myelodysplastic syndrome (MDS)?

Updated: Jul 24, 2018
  • Author: Emmanuel C Besa, MD; Chief Editor: Koyamangalath Krishnan, MD, FRCP, FACP  more...
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Answer

In cases of the presence of ringed sideroblasts or an absence of response, the addition of granulocyte colony-stimulating factor (G-CSF; filgrastim, filgrastim-sndz, or tbo-filgrastim), 1–2 μg/kg 1–3 times per week should be considered. The combination of ESAs and G-CSF should be considered only for patients who are not heavily transfusion dependent (fewer than 2 RBC units per month), have serum erythropoietin levels < 500 mU/mL, and are not responding to ESAs alone. For patients with a serum EPO ≤500 mU/mL and ring sideroblasts < 15% who have no response to an ESA alone, the NCCN suggests. adding lenalidomide plus or minus G-CSF. [24]

Of MDS patients with neutropenia, 75% respond to G-CSF. [25] Of MDS patients with anemia and neutropenia, 75% respond to a combination of an ESA and G-CSF for their neutropenia, with a 50% increase in erythroid response. The addition of low doses of G-CSF synergistically enhances the erythroid response to ESAs—in particular, patients who have refractory anemia with ringed sideroblasts (RARS).

A re-analysis that used the World Health Organization classification demonstrated a significantly better response in RARS (75%) than in refractory cytopenia with multilineage dysplasia and ringed sideroblasts (RCMD-RS; 9%). This may reflect G-CSF's ability to strongly inhibit cytochrome c release and hence mitochondria-mediated apoptosis in RARS erythroblasts.


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