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

Onderzoeksoutput: Bijdrage aan tijdschrift › Artikel › peer review

23 Citaten (Scopus)

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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.

Originele taal-2	Engels
Pagina's (van-tot)	363-368
Aantal pagina's	6
Tijdschrift	British Journal of Haematology
Volume	189
Nummer van het tijdschrift	2
DOI's	https://doi.org/10.1111/bjh.16338
Status	Gepubliceerd - 1 apr. 2020

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

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volume = "189",

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T1 - Experience with ponatinib in paediatric patients with leukaemia

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

Y1 - 2020/4/1

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 - acute lymphoblastic leukaemia

KW - chronic myeloid leukaemia

KW - paediatrics

KW - Philadelphia chromosome

KW - ponatinib

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SN - 0007-1048

VL - 189

SP - 363

EP - 368

JO - British Journal of Haematology

JF - British Journal of Haematology

IS - 2

ER -

Experience with ponatinib in paediatric patients with leukaemia

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