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Pediatric patients with refractory central nervous system tumors: experiences of a clinical trial combining bevacizumab and temsirolimus. Anticancer Res 2014 Apr;34(4):1939-45

Date

04/03/2014

Pubmed ID

24692729

Pubmed Central ID

PMC5685166

Scopus ID

2-s2.0-84902246311 (requires institutional sign-in at Scopus site)   24 Citations

Abstract

BACKGROUND: Pre-clinical findings suggest that combination treatment with bevacizumab and temsirolimus could be effective against malignant pediatric central nervous system (CNS) tumors.

PATIENTS AND METHODS: Six pediatric patients were treated as part of a phase I trial with intravenous temsirolimus 25 mg on days 1, 8, 15, and bevacizumab at 5, 10, or 15 mg/kg on day 1 of each 21-day cycle until disease progression or patient withdrawal.

RESULTS: The median patient age was six years (range=3-14 years). The primary diagnoses were glioblastoma multiforme (n=2), medullobalstoma (n=2), pontine glioma (n=1) and ependymoma (n=1). All patients had disease refractory to standard-of-care (2-3 prior systemic therapies). Grade 3 toxicities possibly related to drugs used occurred in two patients: anorexia, nausea, and weight loss in one, and thrombocytopenia and alanine aminotransferase elevation in another. One patient with glioblastoma multiforme achieved a partial response (51% regression) and two patients (with medulloblastoma and pontine glioma) had stable disease for four months or more (20 and 47 weeks, respectively). One other patient (with glioblastoma multiforme) showed 18% tumor regression (duration=12 weeks).

CONCLUSION: The combination of bevacizumab with temsirolimus was well-tolerated and resulted in stable disease of at least four months/partial response in three out of six pediatric patients with chemorefractory CNS tumors.

Author List

Piha-Paul SA, Shin SJ, Vats T, Guha-Thakurta N, Aaron J, Rytting M, Kleinerman E, Kurzrock R

Author

Razelle Kurzrock MD Center Associate Director, Professor in the Medicine department at Medical College of Wisconsin




MESH terms used to index this publication - Major topics in bold

Adolescent
Angiogenesis Inhibitors
Antibodies, Monoclonal, Humanized
Antineoplastic Combined Chemotherapy Protocols
Bevacizumab
Brain Neoplasms
Central Nervous System Neoplasms
Child
Child, Preschool
Humans
Magnetic Resonance Imaging
Protein Kinase Inhibitors
Sirolimus
Treatment Outcome