Experience with ponatinib in paediatric patients with leukaemia Journal Articles

abstract

• SummaryPonatinib 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.

authors

• 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
• van der Sluis, Inge M
• Keller, Frank G
• Zwaan, Christian M
• Suttorp, Meinolf
• Hijiya, Nobuko

status

• published 

publication date

• April 2020

has subject area

• 1102 Cardiorespiratory Medicine and Haematology (FoR)
• Adolescent (MeSH)
• Adult (MeSH)
• Antineoplastic Agents (MeSH)
• Child (MeSH)
• Female (MeSH)
• Humans (MeSH)
• Imidazoles (MeSH)
• Immunology (Science Metrix)
• Male (MeSH)
• Precursor Cell Lymphoblastic Leukemia-Lymphoma (MeSH)
• Pyridazines (MeSH)
• Young Adult (MeSH)

published in

• British Journal of Haematology  Journal

keywords

• Adolescent
• Adult
• Antineoplastic Agents
• Child
• Female
• Humans
• Imidazoles
• Male
• Philadelphia chromosome
• Precursor Cell Lymphoblastic Leukemia-Lymphoma
• Pyridazines
• Young Adult
• acute lymphoblastic leukaemia
• chronic myeloid leukaemia
• paediatrics
• ponatinib

Digital Object Identifier (DOI)

• 10.1111/bjh.16338

PubMed ID

• 31975387

start page

• 363

end page

• 368

volume

• 189

issue

• 2