Experience with ponatinib in paediatric patients with leukaemia Academic Article uri icon

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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.


  • Rossoff, Jenna
  • Huynh, Van
  • Rau, Rachel E
  • Macy, Margaret E
  • Sulis, Maria L
  • Schultz, Kirk R
  • Burke, Michael J
  • Athale, Uma
  • O'Brien, Maureen M
  • Gregory, John J
  • Sluis, Inge M
  • Keller, Frank G
  • Zwaan, Christian M
  • Suttorp, Meinolf
  • Hijiya, Nobuko

publication date

  • April 2020