Stable long-term risk of leukaemia in patients with severe congenital neutropenia maintained on G-CSF therapy

Br J Haematol. 2010 Jul;150(2):196-9. doi: 10.1111/j.1365-2141.2010.08216.x. Epub 2010 Apr 29.

Abstract

In severe congenital neutropenia (SCN), long-term therapy with granulocyte colony-stimulating factor (G-CSF) has reduced mortality from sepsis, revealing an underlying predisposition to myelodysplastic syndrome and acute myeloid leukaemia (MDS/AML). We have reported the early pattern of evolution to MDS/AML, but the long-term risk remains uncertain. We updated a prospective study of 374 SCN patients on long-term G-CSF enrolled in the Severe Chronic Neutropenia International Registry. Long-term, the annual risk of MDS/AML attained a plateau (2.3%/year after 10 years). This risk now appears similar to, rather than higher than, the risk of AML in Fanconi anaemia and dyskeratosis congenita.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, N.I.H., Intramural

MeSH terms

  • Epidemiologic Methods
  • Granulocyte Colony-Stimulating Factor / therapeutic use*
  • Humans
  • Leukemia, Myeloid, Acute / etiology*
  • Myelodysplastic Syndromes / etiology
  • Neutropenia / complications*
  • Neutropenia / congenital*
  • Neutropenia / drug therapy

Substances

  • Granulocyte Colony-Stimulating Factor