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Myelodysplasia and acute myeloid leukaemia in cases of aplastic anaemia and congenital neutropenia following G‐CSF administration

Summary. Myelodysplasia and acute myeloid leukaemia (MDS/AML) developed in three cases of severe aplastic anaemia (SAA) and one case of congenital neutropenia (CN, Kostmann's disease) who received recombinant human granulocyte colony‐stimulating factor (G‐CSF) are reported. In these four MDS/AML cases, age at diagnosis of SAA/CN was 0–13 years, the cumulative dose of G‐CSF was 98 μg/kg to 10 mg/kg over 1–57 months, and the interval from initiation of G‐CSF to MDS/AML was 25, 23, 31 and 57 months, respectively. These results suggest a link between SAA/CN and MDS/AML in relation to G‐CSF administration; however, large studies are necessary to determine if such a risk is significant in patients with SAA/CN who are treated with G‐CSF.