title: Systemic neoadjuvant chemotherapy for Group B intraocular retinoblastoma (ARET0331): A report from the Children's Oncology Group.
abstract: OBJECTIVE
  To evaluate a chemoreduction regimen using systemic vincristine and carboplatin (VC) and local ophthalmic therapies to avoid external-beam radiotherapy (EBRT) or enucleation in patients with Group B intraocular retinoblastoma.
  
  
  METHODS
  Twenty-one patients (25 eyes) were treated with six cycles of VC, accompanied by local ophthalmic therapies after cycle 1. The primary study objective was to determine the 2-year event-free survival (EFS) where an event was defined as the use of systemic chemotherapy in addition to vincristine or carboplatin, EBRT, and/or enucleation.
  
  
  RESULTS
  All patients had tumor regression after the first cycle of VC and only two patients had progression during therapy. There were seven treatment failures within 2 years of study enrollment, resulting in 2-year EFS of 65% and early study closure in accordance with the statistical design. The 2-year cumulative incidence of enucleation was 15%; for external beam radiation therapy, it was 10%; and for chemotherapy to control progressive disease, it was 10%. All patients sustaining a treatment failure were salvaged with additional therapy.
  
  
  CONCLUSIONS
  For the majority of patients with Group B intraocular retinoblastoma, chemoreduction with VC, without etoposide, in conjunction with local therapy provides excellent opportunity for ocular salvage. Local therapy given with every chemotherapy cycle and incorporation of etoposide may provide improved ocular salvage rates. Central review of group at diagnosis is critical in assigning appropriate therapies.
authors.affiliation: Division of Pediatric Hematology/Oncology, Vanderbilt University School of Medicine, Nashville, Tennessee.|Vanderbilt-Ingram Cancer Center, Nashville, Tennessee.
authors.forename: Debra L
authors.initials: DL
authors.lastname: Friedman
authors.affiliation: University of Southern California, Los Angeles, California.|Children's Oncology Group, Monrovia, California.
authors.forename: Mark
authors.initials: M
authors.lastname: Krailo
authors.affiliation: Children's Oncology Group, Monrovia, California.
authors.forename: Doojduen
authors.initials: D
authors.lastname: Villaluna
authors.affiliation: MD Anderson Cancer Center, Houston, Texas.
authors.forename: Dan
authors.initials: D
authors.lastname: Gombos
authors.affiliation: University of Southern California, Los Angeles, California.|Children's Oncology Group, Monrovia, California.
authors.forename: Bryan
authors.initials: B
authors.lastname: Langholz
authors.affiliation: Children's Hospital of Los Angeles, Los Angeles, California.
authors.forename: Rima
authors.initials: R
authors.lastname: Jubran
authors.affiliation: Wills Eye Hospital, Philadelphia, Pennsylvania.
authors.forename: Carol
authors.initials: C
authors.lastname: Shields
authors.affiliation: University of Southern California, Los Angeles, California.|Children's Hospital of Los Angeles, Los Angeles, California.
authors.forename: Linn
authors.initials: L
authors.lastname: Murphree
authors.affiliation: University of Pennsylvania, Philadelphia, Pennsylvania.
authors.forename: Joan
authors.initials: J
authors.lastname: O'Brien
authors.affiliation: Imaging and Radiation Oncology Core, Lincoln, Rhode Island.
authors.forename: Sandra
authors.initials: S
authors.lastname: Kessel
authors.affiliation: St. Jude Children's Research Hospital, Memphis, Tennessee.
authors.forename: Carlos
authors.initials: C
authors.lastname: Rodriguez-Galindo
authors.affiliation: Texas Children's Hospital, Houston, Texas.|Baylor School of Medicine, Houston, Texas.
authors.forename: Murali
authors.initials: M
authors.lastname: Chintagumpala
authors.affiliation: The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
authors.forename: Anna T
authors.initials: AT
authors.lastname: Meadows
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D016190:Carboplatin / Q000008:administration & dosage; D017024:Chemotherapy, Adjuvant / Q000379:methods*; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D020360:Neoadjuvant Therapy / Q000379:methods*; D019572:Retinal Neoplasms / Q000188:drug therapy*; D012175:Retinoblastoma / Q000188:drug therapy*; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D014750:Vincristine; D016190:Carboplatin
keywords: ARET0331; Children's Oncology Group; Group B intraocular retinoblastoma; retinoblastoma; systemic neoadjuvant chemotherapy