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 globalcollaboration to translate these discoveries into improved outcomes.

Brown, P., Pieters, R., Biondi, A. (2019). How I treat infant leukemia. BLOOD, 133(3), 205-214 [10.1182/blood-2018-04-785980].

How I treat infant leukemia

Biondi, A
2019

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 globalcollaboration to translate these discoveries into improved outcomes.
Articolo in rivista - Articolo scientifico
Biochemistry; Immunology; Hematology; Cell Biology
English
2019
133
3
205
214
reserved
Brown, P., Pieters, R., Biondi, A. (2019). How I treat infant leukemia. BLOOD, 133(3), 205-214 [10.1182/blood-2018-04-785980].
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10281/226172
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