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

Group Pieters

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

30 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

Philadelphia chromosome
acute lymphoblastic leukaemia
chronic myeloid leukaemia
paediatrics
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.",

keywords = "Philadelphia chromosome, acute lymphoblastic leukaemia, chronic myeloid leukaemia, paediatrics, ponatinib",

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",

note = "Publisher Copyright: {\textcopyright} 2020 British Society for Haematology and John Wiley \& Sons Ltd",

year = "2020",

month = apr,

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doi = "10.1111/bjh.16338",

language = "English",

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AU - Rossoff, Jenna

AU - Huynh, Van

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

PY - 2020/4/1

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N2 - 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.

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.

KW - Philadelphia chromosome

KW - acute lymphoblastic leukaemia

KW - chronic myeloid leukaemia

KW - paediatrics

KW - ponatinib

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SP - 363

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JO - British journal of haematology

JF - British journal of haematology

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ER -

Experience with ponatinib in paediatric patients with leukaemia

Abstract

Keywords

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