Stewart Goldman1, Ian F Pollack2, Regina I Jakacki2, Catherine A Billups3, Tina Y Poussaint4,5, Adekunle M Adesina6, Ashok Panigrahy7, Donald W Parsons8, Alberto Broniscer9, Giles W Robinson10, Nathan J Robison11, Sonia Partap12, Lindsay B Kilburn13, Arzu Onar-Thomas3, Ira J Dunkel14, Maryam Fouladi15. 1. Division of Hematology, Oncology, Neuro-Oncology, Stem Cell Transplantation, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois. 2. Department of Pediatric Neurosurgery, Children's Hospital of Pittsburgh, University of Pittsburgh, Pittsburgh, Pennsylvania. 3. Department of Biostatistics, St Jude's Children's Research Hospital, Memphis, Tennessee. 4. Department of Radiology, Boston Children's Hospital, Boston, Massachusetts. 5. Department of Radiology, Harvard Medical School, Boston, Massachusetts. 6. Department of Pathology, Baylor College of Medicine, Houston, Texas. 7. Department of Pediatric Radiology, Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania. 8. Texas Children's Cancer and Hematology Centers, Texas Medical Center, Houston, Texas. 9. Department of Radiology, Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania. 10. Division of Neuro-Oncology, St Jude's Children's Research Hospital, Memphis, Tennessee. 11. Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, Los Angeles, California. 12. Department of Neurology, Stanford University School of Medicine, Stanford, California. 13. Department of Hematology and Oncology, Children's National Medical Center, Washington, DC. 14. Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, New York. 15. Department of Hematology and Oncology, Cincinnati Children's Hospital, Cincinnati, Ohio.
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.
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.
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