Experience with ponatinib in paediatric patients with leukaemia

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*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

3 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)363-368
Number of pages6
JournalBritish Journal of Haematology
Issue number2
StatePublished - Apr 1 2020


  • Philadelphia chromosome
  • acute lymphoblastic leukaemia
  • chronic myeloid leukaemia
  • paediatrics
  • ponatinib

ASJC Scopus subject areas

  • Hematology

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