Medical College of Wisconsin
CTSICores SearchResearch InformaticsREDCap

Experience with ponatinib in paediatric patients with leukaemia. Br J Haematol 2020 Apr;189(2):363-368



Pubmed ID





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.

Author List

Rossoff J, Huynh V, Rau RE, Macy ME, Sulis ML, Schultz KR, Burke MJ, Athale U, O'Brien MM, Gregory JJ Jr, van der Sluis IM, Keller FG, Zwaan CM, Suttorp M, Hijiya N


Michael James Burke MD Professor in the Pediatrics department at Medical College of Wisconsin

jenkins-FCD Prod-482 91ad8a360b6da540234915ea01ff80e38bfdb40a