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

Stewart Goldman*, Ian F. Pollack, Regina I. Jakacki, Catherine A. Billups, Tina Y. Poussaint, Adekunle M. Adesina, Ashok Panigrahy, Donald W. Parsons, Alberto Broniscer, Giles W. Robinson, Nathan J. Robison, Sonia Partap, Lindsay B. Kilburn, Arzu Onar-Thomas, Ira J. Dunkel, Maryam Fouladi

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

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.

Original languageEnglish (US)
Pages (from-to)1696-1704
Number of pages9
JournalNeuro-oncology
Volume22
Issue number11
DOIs
StatePublished - Nov 1 2020

ASJC Scopus subject areas

  • Oncology
  • Clinical Neurology
  • Cancer Research

Fingerprint Dive into the research topics of 'Phase II study of peginterferon alpha-2b for patients with unresectable or recurrent craniopharyngiomas: A Pediatric Brain Tumor Consortium report'. Together they form a unique fingerprint.

Cite this