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Experience with ponatinib in paediatric patients with leukaemia
Author(s) -
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 year - 2020
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.16338
Subject(s) - ponatinib , medicine , chronic myeloid leukaemia , pediatrics , cohort , lymphoblastic leukemia , disease , leukemia , myeloid leukemia , dasatinib , imatinib
Summary 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.