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Hematol. Oncol. Clin. North Am. · Aug 2018
ReviewIntroduction to Acquired and Inherited Bone Marrow Failure.
- Colin A Sieff.
- Division of Hematology/Oncology, Dana-Farber and Boston Children's Cancer and Blood Disorders Center, Dana 3104, 450 Brookline Avenue, Boston, MA 02215, USA. Electronic address: Colin.Sieff@childrens.harvard.edu.
- Hematol. Oncol. Clin. North Am. 2018 Aug 1; 32 (4): 569-580.
AbstractAcquired aplastic anemia and inherited bone marrow failure syndromes both present with pancytopenia and must be distinguished because they have differences in treatment decisions and continued monitoring requirements. Advances in the genetic interrogation of patient samples have led to identification of inherited germline diseases and appreciation that patients with inherited bone marrow failure disorders may be normal in appearance with few expected clinical clues. Somatic mutations in aplastic anemia may have prognostic value. Hematopoietic stem cells from inherited marrow failure diseases can correct the proliferative defect and may develop further somatic mutations that progress to myelodysplastic syndrome or acute myeloid leukemia.Copyright © 2018 Elsevier Inc. All rights reserved.
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