Although the majority of congenital neutropenia patients respond to daily granulocyte colony stimulating factor, approximately 15 % do not respond to this cytokine at doses up to 50 μg/kg/day and approximately 15 % of patients will develop myelodysplasia or acute myeloid leukemia.
Thus, some decision makers may determine that filgrastim-sndz and infliximab-dyyb be used for the off-label indications of treating symptomatic anemia in patients with myelodysplasia and immune-mediated colitis, respectively.
Children with severe congenital neutropenia chronically receive pharmacologic doses of GCSF, and ∼30% will develop myelodysplasia/acute myeloid leukemia (AML) associated with GCSFR truncation mutations.