Phase II study of peginterferon alpha-2b for patients with unresectable or recurrent craniopharyngiomas: a Pediatric Brain Tumor Consortium report. Issue 11 (12th May 2020)

• Record Type: Journal Article
• Authors: Goldman, Stewart; Pollack, Ian F; Jakacki, Regina I; Billups, Catherine A; Poussaint, Tina Y; Adesina, Adekunle M; Panigrahy, Ashok; Parsons, Donald W; Broniscer, Alberto; Robinson, Giles W; Robison, Nathan J; Partap, Sonia; Kilburn, Lindsay B; Onar-Thomas, Arzu; Dunkel, Ira J; Fouladi, Maryam
• Is Part Of: Neuro-oncology. Volume 22:Issue 11(2020)
• Page Start: 1696
• Page End: 1704
• Publication Date: 2020-05-12
• Subjects: craniopharyngioma -- interferon -- pediatric brain tumor -- peginterferon alpha-2b; Brain Neoplasms -- Periodicals; Brain -- Tumors -- Periodicals; Brain -- Cancer -- Periodicals; Nervous system -- Cancer -- Periodicals; 616.99481
• DOI: 10.1093/neuonc/noaa119
• Languages: English
• ISSNs: 1522-8517

Abstract

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.