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

doi:10.1111/bjh.16338

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

Research output: Contribution to journal › Article › peer-review

24 Citations (Scopus)

Abstract

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 language	English
Pages (from-to)	363-368
Number of pages	6
Journal	British Journal of Haematology
Volume	189
Issue number	2
DOIs	https://doi.org/10.1111/bjh.16338
Publication status	Published - 1 Apr 2020

Keywords

acute lymphoblastic leukaemia
chronic myeloid leukaemia
paediatrics
Philadelphia chromosome
ponatinib

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10.1111/bjh.16338

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title = "Experience with ponatinib in paediatric patients with leukaemia",

abstract = "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.",

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author = "Jenna Rossoff and Van Huynh and Rau, {Rachel E.} and Macy, {Margaret E.} and Sulis, {Maria L.} and Schultz, {Kirk R.} and Burke, {Michael J.} and Uma Athale and O'Brien, {Maureen M.} and Gregory, {John J.} and {van der Sluis}, {Inge M.} and Keller, {Frank G.} and Zwaan, {Christian M.} and Meinolf Suttorp and Nobuko Hijiya",

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AU - Rau, Rachel E.

AU - Macy, Margaret E.

AU - Sulis, Maria L.

AU - Schultz, Kirk R.

AU - Burke, Michael J.

AU - Athale, Uma

AU - O'Brien, Maureen M.

AU - Gregory, John J.

AU - van der Sluis, Inge M.

AU - Keller, Frank G.

AU - Zwaan, Christian M.

AU - Suttorp, Meinolf

AU - Hijiya, Nobuko

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AB - 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.

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Experience with ponatinib in paediatric patients with leukaemia

Abstract

Keywords

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