title: A phase 2 study of pegylated interferon α-2b (PEG-Intron(®)) in children with diffuse intrinsic pontine glioma.
abstract: BACKGROUND
  Interferon-α is a cytokine that has demonstrated activity in patients with supratentorial gliomas, but its ideal dose and schedule of administration is unknown. Studies suggest that low-dose, continuous exposure is more efficacious than intermittent, high doses. The authors performed a phase 2 study of recombinant interferon α-2b with monomethoxy polyethylene glycol (PEG-Intron(®)) in children with diffuse intrinsic pontine glioma (DIPG), a population with dismal survival despite decades of clinical investigation. The primary objective was to compare 2-year survival with a historic cohort that received radiation therapy alone.
  
  
  METHODS
  Patients received weekly subcutaneous PEG-Intron(®) at a dose of 0.3 μg/kg beginning 2 to 10 weeks after the completion of radiation therapy until they developed disease progression. Patients were evaluated clinically and radiographically at regular intervals. Serum and urine were assayed for biomarkers before each cycle. Quality-of-life (QOL) evaluations were administered at baseline and before every other cycle of therapy to the parents of patients ages 6 to 18 years.
  
  
  RESULTS
  Thirty-two patients (median age, 5.3 years; range, 1.8-14.8 years) were enrolled and received a median of 7 cycles of therapy (range, from 1 cycle to ≥70 cycles). PEG-Intron(®) was well tolerated, and no decrease in QOL scores was noted in the subset of patients tested. The 2-year survival rate was 14%, which was not significantly improved compared with the historic cohort. However, the median time to progression was 7.8 months, which compared favorably with recent trials reporting a time to progression of 5 months in a similar population.
  
  
  CONCLUSIONS
  Although low-dose PEG-Intron(®) therapy did not significantly improve 2-year survival in children with DIPG compared with an historic control population, it did delay the time to progression.
authors.affiliation: Pediatric Oncology Branch, National Cancer Institute, Bethesda, MD 20892, USA. warrenk@mail.nih.gov
authors.forename: Katherine
authors.initials: K
authors.lastname: Warren
authors.forename: Robyn
authors.initials: R
authors.lastname: Bent
authors.forename: Pamela L
authors.initials: PL
authors.lastname: Wolters
authors.forename: Alisa
authors.initials: A
authors.lastname: Prager
authors.forename: Ryan
authors.initials: R
authors.lastname: Hanson
authors.forename: Roger
authors.initials: R
authors.lastname: Packer
authors.forename: Joanna
authors.initials: J
authors.lastname: Shih
authors.forename: Kevin
authors.initials: K
authors.lastname: Camphausen
mesh_terms: D000293:Adolescent; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D020295:Brain Stem Neoplasms / Q000188:drug therapy* / Q000523:psychology / Q000532:radiotherapy; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D018572:Disease-Free Survival; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D007223:Infant; D000077190:Interferon alpha-2; D016898:Interferon-alpha / Q000627:therapeutic use*; D008297:Male; D011092:Polyethylene Glycols / Q000627:therapeutic use*; D011788:Quality of Life; D011994:Recombinant Proteins / Q000627:therapeutic use
chemical_list: D000970:Antineoplastic Agents; D000077190:Interferon alpha-2; D016898:Interferon-alpha; D011994:Recombinant Proteins; D011092:Polyethylene Glycols; C417083:peginterferon alfa-2b
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