Abstract:
:Leukemia in infants is rare but generates tremendous interest due to its aggressive clinical presentation in a uniquely vulnerable host, its poor response to current therapies, and its fascinating biology. Increasingly, these biological insights are pointing the way toward novel therapeutic approaches. Using representative clinical case presentations, we review the key clinical, pathologic, and epidemiologic features of infant leukemia, including the high frequency of KMT2A gene rearrangements. We describe the current approach to risk-stratified treatment of infant leukemia in the major international cooperative groups. We highlight recent discoveries that elucidate the molecular biology of infant leukemia and suggest novel targeted therapeutic strategies, including modulation of aberrant epigenetic programs, inhibition of signaling pathways, and immunotherapeutics. Finally, we underscore the need for increased global collaboration to translate these discoveries into improved outcomes.
journal_name
Bloodjournal_title
Bloodauthors
Brown P,Pieters R,Biondi Adoi
10.1182/blood-2018-04-785980subject
Has Abstractpub_date
2019-01-17 00:00:00pages
205-214issue
3eissn
0006-4971issn
1528-0020pii
blood-2018-04-785980journal_volume
133pub_type
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