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. 2020 Apr;189(2):363-368.
doi: 10.1111/bjh.16338. Epub 2020 Jan 24.

Experience with ponatinib in paediatric patients with leukaemia

Jenna Rossoff  1 , Van Huynh  2 , Rachel E Rau  3 , Margaret E Macy  4 , Maria L Sulis  5 , Kirk R Schultz  6 , Michael J Burke  7 , Uma Athale  8 , Maureen M O'Brien  9 , John J Gregory Jr  10 , Inge M van der Sluis  11 , Frank G Keller  12 , Christian M Zwaan  11 , Meinolf Suttorp  13 , Nobuko Hijiya  1
Affiliations

Experience with ponatinib in paediatric patients with leukaemia

Jenna Rossoff et al. Br J Haematol. 2020 Apr.

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.

Keywords: Philadelphia chromosome; acute lymphoblastic leukaemia; chronic myeloid leukaemia; paediatrics; ponatinib.

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