Document Detail


Malignancies and survival patterns in the National Cancer Institute inherited bone marrow failure syndromes cohort study.
MedLine Citation:
PMID:  20507306     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Fanconi anaemia (FA), dyskeratosis congenita (DC), Diamond-Blackfan anaemia (DBA), and Shwachman-Diamond syndrome (SDS) comprise major inherited bone marrow failure syndromes (IBMFS). Adverse events include severe bone marrow failure (BMF), myelodysplastic syndrome (MDS), acute myeloid leukaemia (AML), and solid tumours (ST). The natural history of FA is well characterised; hazard rates in the other syndromes have not yet been quantified. An open cohort was established at the National Cancer Institute (NCI) in 2002. Patients enrolled prior to December, 2007 were followed up to December, 2008. Diagnoses were confirmed with standard tests. Age-associated risks of adverse events were calculated. Most patients in each syndrome survived to young adulthood. Patients with FA had earlier onset of cancers, need for stem cell transplant, and death; followed by DC; DBA and SDS were mildest. While FA and DC patients had markedly increased risks of cancer, AML and MDS, there were no cases of leukaemia in DBA or SDS patients. The NCI cohort provides the first direct quantitative comparison of timing and magnitude of cancer risk in the IBMFS. The findings demonstrate that both FA and DC are major cancer susceptibility syndromes. The IBMFS, historically considered paediatric disorders, have important management implications for physicians treating adult patients.
Authors:
Blanche P Alter; Neelam Giri; Sharon A Savage; June A Peters; Jennifer T Loud; Lisa Leathwood; Ann G Carr; Mark H Greene; Philip S Rosenberg
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Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural; Research Support, N.I.H., Intramural     Date:  2010-04-30
Journal Detail:
Title:  British journal of haematology     Volume:  150     ISSN:  1365-2141     ISO Abbreviation:  Br. J. Haematol.     Publication Date:  2010 Jul 
Date Detail:
Created Date:  2010-07-19     Completed Date:  2010-09-01     Revised Date:  2011-08-01    
Medline Journal Info:
Nlm Unique ID:  0372544     Medline TA:  Br J Haematol     Country:  England    
Other Details:
Languages:  eng     Pagination:  179-88     Citation Subset:  IM    
Affiliation:
Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Rockville, MD 20852-7231, USA. alterb@mail.nih.gov
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MeSH Terms
Descriptor/Qualifier:
Adolescent
Adult
Age Distribution
Anemia, Diamond-Blackfan / complications,  genetics
Bone Marrow Diseases / complications*,  genetics
Child
Child, Preschool
Dyskeratosis Congenita / complications,  genetics
Epidemiologic Methods
Fanconi Anemia / complications,  genetics
Female
Genetic Diseases, Inborn / complications*,  genetics
Genetic Predisposition to Disease
Genotype
Humans
Male
Middle Aged
Neoplasms / etiology*,  genetics
Prognosis
Syndrome
Young Adult
Grant Support
ID/Acronym/Agency:
N02-CP-11019/CP/NCI NIH HHS; N02-CP-31003/CP/NCI NIH HHS; N02-CP-65501/CP/NCI NIH HHS; N02-CP-65504/CP/NCI NIH HHS; ZIA CP010144-12/CP/NCI NIH HHS
Comments/Corrections

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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