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

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

Author information

  • 1Biostatistics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Rockville, MD 20852, USA. rosenbep@mail.nih.gov

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.

PMID:
20456363
[PubMed - indexed for MEDLINE]
PMCID:
PMC2906693
Free PMC Article
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