Phase II study of peginterferon alpha-2b for patients with unresectable or recurrent craniopharyngiomas: a Pediatric Brain Tumor Consortium report

Abstract

Background: Craniopharyngiomas account for approximately 1.2-4% of all CNS tumors. They are typically treated with a combination of surgical resection and focal radiotherapy. Unfortunately, treatment can lead to permanent deleterious effects on behavior, learning, and endocrine function.

Methods: The Pediatric Brain Tumor Consortium performed a multicenter phase 2 study in children and young adults with unresectable or recurrent craniopharyngioma (PBTC-039). Between December 2013 and November 2017, nineteen patients (median age at enrollment, 13.1 y; range, 2-25 y) were enrolled in one of 2 strata: patients previously treated with surgery alone (stratum 1) or who received radiation (stratum 2).

Results: Eighteen eligible patients (8 male, 10 female) were treated with weekly subcutaneous pegylated interferon alpha-2b for up to 18 courses (108 wk). Therapy was well tolerated with no grade 4 or 5 toxicities. 2 of the 7 eligible patients (28.6%) in stratum 1 had a partial response, but only one response was sustained for more than 3 months. None of the 11 stratum 2 patients had an objective radiographic response, although median progression-free survival was 19.5 months.

Conclusions: Pegylated interferon alpha-2b treatment, in lieu of or following radiotherapy, was well tolerated in children and young adults with recurrent craniopharyngiomas. Although objective responses were limited, progression-free survival results are encouraging, warranting further studies.

Keywords: craniopharyngioma; interferon; pediatric brain tumor; peginterferon alpha-2b.

© The Author(s) 2020. Published by Oxford University Press on behalf of the Society for Neuro-Oncology. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.

Figures

Fig. 1

Progression-free survival for eligible patients by stratum. PFS for stratum 1 and stratum 2 from start of peginterferon alpha-2b treatment.

Fig. 2

Imaging for an individual stratum 1 patient with a partial response. Pre-therapy sagittal T1 demonstrates a hyperintense lesion in the suprasellar region extending into the third ventricle (A). Axial T2 demonstrates a hypointense lesion in the third ventricle (B) and post-contrast axial T1 demonstrates minimal peripheral enhancement in the third ventricular mass (C). Post-therapy sagittal T1 2 years later demonstrates that the heterogeneous mass in the suprasellar region/inferior third ventricle is smaller in size (D). Axial T2 demonstrates the hypointense lesion with decrease in tumor size (E) and post-contrast axial T1 demonstrates ring enhancing lesion in the third ventricle, decreased in size (F).