LGG-64. A Phase II Study of Pegylated Interferon in Children with Recurrent or Refractory and Radiographically or Clinically Progressive Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas (NCT02343224). (3rd June 2022)

• Record Type: Journal Article
• Authors: Aguilera, Dolly; Mazewski, Claire; Janss, Anna; MacDonald, Tobey; Goldman, Adam; Leong, Tracy; Castellino, Robert Craig
• Is Part Of: Neuro-oncology. Volume 24(2022)Supplement 1
• Page Start: i103
• Page End: i103
• Publication Date: 2022-06-03
• Subjects: Brain Neoplasms -- Periodicals; Brain -- Tumors -- Periodicals; Brain -- Cancer -- Periodicals; Nervous system -- Cancer -- Periodicals; 616.99481
• DOI: 10.1093/neuonc/noac079.375
• Languages: English
• ISSNs: 1522-8517

Abstract

Abstract: Unresectable Juvenile Pilocytic Astrocytomas (JPA) and Optic Pathway gliomas (OPG) are chronic diseases that can have solid +/- cystic components. We wanted to evaluate the objective response to pegylated interferon Alpha2B in this group of patients in a prospective single arm Phase II clinical trial. Eligibility criteria: age 3 -25 years, patients with neurofibromatosis (NF) were eligible, evidence of measurable disease in MRI, no limitation in the number of prior therapies including chemotherapy and radiation. Exclusion criteria: prior pegylated interferon exposure, less than 2 years from radiation, active autoimmune disease. Subjects enrolled received pegylated interferon 1 mcg/kg/dose SQ weekly, to a max dose of 150mcg/dose in 28 day cycles for up to 2 years. The study design is a Simon two stage design. If no complete or partial responses among the first 9 patients, the study will terminate. Nine subjects enrolled: 4 females, 5 males, median age of 11years, 6 Caucasians, 3 African Americans. Two subjects with NF. Molecular findings KIAA-BRAF fusion (6), V600E mutation (1), CDK2A loss (1). Location: brain (7), brain and spine (2). We enrolled a heavily pre-treated population, patients with prior radiation ( 1), nine with prior chemotherapy, the average number of regimens 4 (range 2-6). No complete responses or partial responses were seen. Two patients with prolonged stable disease 75+months and 66+ months. At 12 and 24 month EFS 76.2% (95%CI52.1-100%). Median EFS has not been reached. The 12 and 24 months survival estimate 75% (95% CI 50.3-100%), median survival has not been reached. Side effects as expected mostly grade 1-2. No grade 4 event related to pegylated interferon were seen. This is the first report of pegylated interferon in OPG and JPA, two patients with prolonged stable disease suggesting that pegylated interferon can offer potential benefit in this population and additional studies are important.