Stable long-term risk of leukaemia in patients with severe congenital neutropenia maintained on G-CSF therapy.
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.
Rosenberg
Philip S
PS
Biostatistics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Rockville, MD 20852, USA. rosenbep@mail.nih.gov
Zeidler
Cornelia
C
Bolyard
Audrey A
AA
Alter
Blanche P
BP
Bonilla
Mary A
MA
Boxer
Laurence A
LA
Dror
Yigal
Y
Kinsey
Sally
S
Link
Daniel C
DC
Newburger
Peter E
PE
Shimamura
Akiko
A
Welte
Karl
K
Dale
David C
DC
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
D016179:Granulocyte Colony-Stimulating Factor
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