Stable long-term risk of leukaemia in patients with severe congenital neutropenia maintained on G-CSF therapy
Department of Pediatrics
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.
DOI of Published Version
Br J Haematol. 2010 Jul;150(2):196-9. Epub 2010 Apr 29. Link to article on publisher's website
British journal of haematology
Rosenberg, Philip S.; Zeidler, Cornelia; Bolyard, Audrey Anna; Alter, Blanche P.; Bonilla, Mary A.; Boxer, Laurence A.; Dror, Yigal; Kinsey, Sally; Link, Daniel C.; Newburger, Peter E.; Shimamura, Akiko; Welte, Karl; and Dale, David C., "Stable long-term risk of leukaemia in patients with severe congenital neutropenia maintained on G-CSF therapy" (2010). Hematology/Oncology. 131.