Stable long-term risk of leukaemia in patients with severe congenital neutropenia maintained on G-CSF therapy
Department of Pediatrics
Medical Subject Headings
Epidemiologic Methods; Granulocyte Colony-Stimulating Factor; Humans; Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Neutropenia
Hematology | Oncology | Pediatrics
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.
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Citation: Br J Haematol. 2010 Jul;150(2):196-9. Epub 2010 Apr 29. Link to article on publisher's website