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Experience with ponatinib in paediatric patients with leukaemia. Br J Haematol 2020 Apr;189(2):363-368

Date

01/25/2020

Pubmed ID

31975387

DOI

10.1111/bjh.16338

Scopus ID

2-s2.0-85078788320   12 Citations

Abstract

Ponatinib has proven to be effective in adults with Philadelphia chromosome-positive leukaemias, but data in paediatrics are scarce. Among paediatric patients with chronic myeloid leukaemia (n = 9) or acute lymphoblastic leukaemia (n = 12) treated with varying doses of ponatinib in 13 centres, 71% showed a decrease in disease burden after a median of three months. Ponatinib was well tolerated, with grade 3 toxicities occurring in 29% of patients. Toxicities were similar to those reported in adults, with the exception of arterial thrombotic events, which were not observed. Ponatinib has a favourable safety profile in this paediatric cohort, but dose-finding studies are needed.

Author List

Rossoff J, Huynh V, Rau RE, Macy ME, Sulis ML, Schultz KR, Burke MJ, Athale U, O'Brien MM, Gregory JJ Jr, van der Sluis IM, Keller FG, Zwaan CM, Suttorp M, Hijiya N

Author

Michael James Burke MD Professor in the Pediatrics department at Medical College of Wisconsin




MESH terms used to index this publication - Major topics in bold

Adolescent
Adult
Antineoplastic Agents
Child
Female
Humans
Imidazoles
Male
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Pyridazines
Young Adult