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Experience with ponatinib in paediatric patients with leukaemia

Rossoff, J.; Huynh, V.; Rau, R. E.; Macy, M. E.; Sulis, M. L.; Schultz, K. R.; Burke, M. J.; Athale, U.; O'Brien, M. M.; Gregory, J. J., Jr.; van der Sluis, I. M.; Keller, F. G.; Zwaan, C. M.; Suttorp, M.; Hijiya, N.

Br J Haematol. 2020 Jan 25; 189(2):363-368


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.

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