| Institution: | 1. Biostatistics Branch, National Cancer Institute, Rockville, MD, USA;2. These authors contributed equally.;3. Kinderklinik, Medizinische Hochschule, Carl‐Neuberg‐Str. 1, Hannover, Germany;4. Department of Medicine, University of Washington, Seattle, WA;5. Clinical Genetics Branch, National Cancer Institute, Rockville, MD;6. St. Joseph’s Children’s Hospital, Pediatric Hematology Oncology, Paterson NJ;7. University of Michigan, Pediatric Hematology/Oncology, Ann Arbor, MI, USA;8. Hospital for Sick Children, The University of Toronto, Toronto, ON, Canada;9. Paediatric Haematology, St. James’s University Hospital, Leeds, UK;10. Washington University School of Medicine, Division of Bone Marrow Transplantation, St. Louis, MO;11. University of Massachusetts Medical School, Worcester, MA;12. Fred Hutchinson Cancer Research Center, Seattle, WA, USA |
| 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. |
