Experience with ponatinib in paediatric patients with leukaemia. Br J Haematol 2020 Apr;189(2):363-368
Date
01/25/2020Pubmed ID
31975387DOI
10.1111/bjh.16338Scopus ID
2-s2.0-85078788320 (requires institutional sign-in at Scopus site) 22 CitationsAbstract
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 NAuthor
Michael James Burke MD Professor in the Pediatrics department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
AdolescentAdult
Antineoplastic Agents
Child
Female
Humans
Imidazoles
Male
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Pyridazines
Young Adult
