Monitoring Ponatinib in a Child with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia

Autor: Kazuki Tanimura, Kai Yamasaki, Yuki Okuhiro, Kota Hira, Chika Nitani, Keiko Okada, Hiroyuki Fujisaki, Kana Matsumoto, Junichi Hara
Jazyk: angličtina
Rok vydání: 2021
Předmět:
Zdroj: Case Reports in Oncology, Vol 14, Iss 1, Pp 24-28 (2021)
Druh dokumentu: article
ISSN: 1662-6575
DOI: 10.1159/000511071
Popis: Ponatinib is a third-generation tyrosine kinase inhibitor (TKI) reported to show a higher efficacy for adult Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL) than other TKIs. However, few studies describe ponatinib for pediatric Ph+ALL; therefore, the efficacy, safety, and optimal dosage have not been determined. Here, we report a 3-year-old girl with Ph+ALL treated by a ponatinib-containing regimen with therapeutic drug monitoring in the plasma and cerebrospinal fluid (CSF). In our case, a ponatinib-containing regimen was able to keep minimal residual disease negative, and the pharmacokinetics (PKs) of plasma ponatinib resembled that previously reported in adults. Penetration to the CSF was extremely limited. Thus, ponatinib was feasible and effective for a child with Ph+ALL, although the plasma concentration of ponatinib varied significantly throughout the treatment. The appropriate dosage should be confirmed in a prospective trial, including a detailed PK study.
Databáze: Directory of Open Access Journals