An abnormal clone with monosomy 7 and trisomy 21 in the bone marrow of a child with congenital agranulocytosis (Kostmann disease) treated with granulocyte colony-stimulating factor. Evolution towards myelodysplastic syndrome and acute basophilic leukemia

Cancer Genet Cytogenet. 1995 Oct 15;84(2):99-104. doi: 10.1016/0165-4608(95)00095-x.

Abstract

Cytogenetic analysis of bone marrow cells revealed an abnormal clone with monosomy 7 and trisomy 21 in a 12-year-old child with Kostmann disease (KD). The patient presented with anemia, thrombocytopenia, and splenomegaly after 5 years of treatment with granulocyte colony-stimulating factor (G-CSF). The bone marrow morphology was consistent with the diagnosis of myelodysplastic syndrome (MDS). Administration of G-CSF was discontinued at this point. Bone marrow studies 2 and 5 months later showed persistence of both myelodysplasia and the abnormal clone with monosomy 7 and trisomy 21. Monosomy 7 was also confirmed by fluorescence in situ hybridization (FISH). After 2 months of follow-up, the patient presented with acute basophilic leukemia, a very rare variant of acute myeloid leukemia (AML), expressing the same bone marrow chromosome abnormalities as observed earlier. This is a rare case of KD with prolonged survival and a cytogenetically abnormal clone evolving to MDS and acute basophilic leukemia. The significance of monosomy 7 and trisomy 21 in KD treated with G-CSF is discussed.

Publication types

  • Case Reports

MeSH terms

  • Agranulocytosis / congenital*
  • Agranulocytosis / therapy
  • Child
  • Chromosomes, Human, Pair 21*
  • Chromosomes, Human, Pair 7*
  • Down Syndrome / genetics*
  • Granulocyte Colony-Stimulating Factor / therapeutic use*
  • Humans
  • Karyotyping
  • Leukemia, Basophilic, Acute / genetics*
  • Male
  • Monosomy*
  • Myelodysplastic Syndromes / genetics*

Substances

  • Granulocyte Colony-Stimulating Factor