Experience with ponatinib in paediatric patients with leukaemia.

British journal of haematology 2020 Apr

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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. © 2020 British Society for Haematology and John Wiley & Sons Ltd.

Citation

Jenna Rossoff, Van Huynh, Rachel E Rau, Margaret E Macy, Maria L Sulis, Kirk R Schultz, Michael J Burke, Uma Athale, Maureen M O'Brien, John J Gregory, Inge M van der Sluis, Frank G Keller, Christian M Zwaan, Meinolf Suttorp, Nobuko Hijiya. Experience with ponatinib in paediatric patients with leukaemia. British journal of haematology. 2020 Apr;189(2):363-368