Phase 1 study of pomalidomide in children with recurrent, refractory, and progressive central nervous system tumors: A Pediatric Brain Tumor Consortium trial.
IMiD agent
brain tumor
central nervous system tumor
pediatric
pomalidomide
Journal
Pediatric blood & cancer
ISSN: 1545-5017
Titre abrégé: Pediatr Blood Cancer
Pays: United States
ID NLM: 101186624
Informations de publication
Date de publication:
02 2021
02 2021
Historique:
received:
15
08
2020
revised:
22
09
2020
accepted:
23
09
2020
pubmed:
8
10
2020
medline:
17
6
2021
entrez:
7
10
2020
Statut:
ppublish
Résumé
Central nervous system (CNS) malignancies are the most common solid tumors among children, and novel therapies are needed to help improve survival. Pomalidomide is an immunomodulatory agent that displays antiangiogenic and cytotoxic activity, making it an appropriate candidate to explore in pediatric CNS tumors. A phase 1 first in pediatric trial of pomalidomide was conducted in children with recurrent, progressive, and refractory CNS tumors. The primary objective was to determine the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) when given orally once daily for 21 consecutive days of a 28-day cycle. Once the MTD was established, 12 additional patients were enrolled on expansion cohorts based on age and steroid use. Twenty-nine children were enrolled and 25 were evaluable for dose-limiting toxicity (DLT). The MTD was 2.6 mg/m The MTD of pomalidomide is 2.6 mg/m
Sections du résumé
BACKGROUND
Central nervous system (CNS) malignancies are the most common solid tumors among children, and novel therapies are needed to help improve survival. Pomalidomide is an immunomodulatory agent that displays antiangiogenic and cytotoxic activity, making it an appropriate candidate to explore in pediatric CNS tumors.
METHODS
A phase 1 first in pediatric trial of pomalidomide was conducted in children with recurrent, progressive, and refractory CNS tumors. The primary objective was to determine the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) when given orally once daily for 21 consecutive days of a 28-day cycle. Once the MTD was established, 12 additional patients were enrolled on expansion cohorts based on age and steroid use.
RESULTS
Twenty-nine children were enrolled and 25 were evaluable for dose-limiting toxicity (DLT). The MTD was 2.6 mg/m
CONCLUSIONS
The MTD of pomalidomide is 2.6 mg/m
Identifiants
pubmed: 33025730
doi: 10.1002/pbc.28756
pmc: PMC7757731
mid: NIHMS1651744
doi:
Substances chimiques
Angiogenesis Inhibitors
0
Antineoplastic Agents
0
Thalidomide
4Z8R6ORS6L
pomalidomide
D2UX06XLB5
Banques de données
ClinicalTrials.gov
['NCT03257631']
Types de publication
Clinical Trial, Phase I
Journal Article
Research Support, N.I.H., Extramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
e28756Subventions
Organisme : NCI NIH HHS
ID : P30 CA008748
Pays : United States
Organisme : NCI NIH HHS
ID : UM1 CA081457
Pays : United States
Organisme : NCI NIH HHS
ID : UMICA081457
Pays : United States
Organisme : NCI NIH HHS
ID : P30CA008748
Pays : United States
Informations de copyright
© 2020 Wiley Periodicals LLC.
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