Experience with ponatinib in paediatric patients with leukaemia.
Jenna RossoffVan HuynhRachel E RauMargaret E MacyMaria L SulisKirk R SchultzMichael J BurkeUma AthaleMaureen M O'BrienJohn J GregoryInge M van der SluisFrank G KellerChristian M ZwaanMeinolf SuttorpNobuko HijiyaPublished in: British journal of haematology (2020)
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.
Keyphrases
- chronic myeloid leukemia
- intensive care unit
- emergency department
- end stage renal disease
- newly diagnosed
- chronic kidney disease
- ejection fraction
- acute lymphoblastic leukemia
- liver failure
- bone marrow
- peritoneal dialysis
- acute myeloid leukemia
- dendritic cells
- respiratory failure
- electronic health record
- copy number
- gene expression
- machine learning
- immune response
- drug induced
- hepatitis b virus
- mechanical ventilation