title: Stable long-term risk of leukaemia in patients with severe congenital neutropenia maintained on G-CSF therapy.
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.
authors.affiliation: Biostatistics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Rockville, MD 20852, USA. rosenbep@mail.nih.gov
authors.forename: Philip S
authors.initials: PS
authors.lastname: Rosenberg
authors.forename: Cornelia
authors.initials: C
authors.lastname: Zeidler
authors.forename: Audrey A
authors.initials: AA
authors.lastname: Bolyard
authors.forename: Blanche P
authors.initials: BP
authors.lastname: Alter
authors.forename: Mary A
authors.initials: MA
authors.lastname: Bonilla
authors.forename: Laurence A
authors.initials: LA
authors.lastname: Boxer
authors.forename: Yigal
authors.initials: Y
authors.lastname: Dror
authors.forename: Sally
authors.initials: S
authors.lastname: Kinsey
authors.forename: Daniel C
authors.initials: DC
authors.lastname: Link
authors.forename: Peter E
authors.initials: PE
authors.lastname: Newburger
authors.forename: Akiko
authors.initials: A
authors.lastname: Shimamura
authors.forename: Karl
authors.initials: K
authors.lastname: Welte
authors.forename: David C
authors.initials: DC
authors.lastname: Dale
mesh_terms: D004812:Epidemiologic Methods; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use*; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000209:etiology*; D009190:Myelodysplastic Syndromes / Q000209:etiology; D009503:Neutropenia / Q000150:complications* / Q000151:congenital* / Q000188:drug therapy
chemical_list: D016179:Granulocyte Colony-Stimulating Factor
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