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Transient abnormal myelopoiesis without constitutional Down syndrome

Abstract:
Transient abnormal myelopoiesis (TAM) is a unique entity that usually occurs in children with Down syndrome (DS) or with trisomy 21 mosaicism. The somatic GATA1 mutation is a distinct feature of TAM. At presentation, TAM can resemble congenital leukemia (CL), which unlike TAM has an extremely poor prognosis and requires prompt therapeutic interventions. Therefore, correct and timely distinction between the two entities is crucial. We report a case of a phenotypically normal infant diagnosed with CL during the first weeks of life that retrospectively was reassessed as TAM. No acute myeloid leukemia (AML) specific mutations were found except for trisomy 21 confined exclusively to leukemic blasts. Retrospectively GATA1 mutation was also detected in malignant cells, but somatic genome appeared to be intact.
Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.7363/090104

Authors

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Institution:
University of Oxford
Division:
MSD
Department:
RDM
Sub department:
RDM Strategic
Role:
Author
ORCID:
0000-0003-3931-0914


Publisher:
Hygeia Press di Corridori Marinella
Journal:
Journal of Pediatric and Neonatal Individualized Medicine More from this journal
Volume:
9
Issue:
1
Article number:
e090104
Publication date:
2019-12-23
Acceptance date:
2019-06-08
DOI:
EISSN:
2281-0692


Language:
English
Keywords:
Pubs id:
1119420
Local pid:
pubs:1119420
Deposit date:
2021-01-20
ARK identifier:

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