title: Neoadjuvant/adjuvant treatment of high-risk retinoblastoma: a report from the German Retinoblastoma Referral Centre.
abstract: BACKGROUND
  Retinoblastoma can extend beyond the structures of the eye, where cells can enter the bloodstream and cause metastases. Various types of protocols for adjuvant treatment risk-adapted according to histopathological risk factors are used worldwide.
  
  
  METHODS
  Between 1997 and 2009, 420 children were diagnosed with retinoblastoma at the German Retinoblastoma Referral Centre and risk factors were assessed. Patients with post-laminar optic nerve infiltration or choroid or minor scleral invasion received six courses of adjuvant chemotherapy using vincristine, etoposide, carboplatin and cyclophosphamide (group 1). Patients with microscopic extension beyond the sclera to the resection margin of the optic nerve or potential spread due to vitrectomy received chemotherapy plus orbital radiotherapy (group 2). Neoadjuvant chemotherapy was performed in patients with local extraocular invasion detected on MRI.
  
  
  RESULTS
  Following this protocol, 42 of the 420 patients and 21 referred from other centres showed high-risk histopathological factors qualifying for adjuvant therapy (57 in group 1 and 6 in group 2). Seven of the 63 patients received neoadjuvant and adjuvant treatment. During a mean follow-up of 5.8 (range 0.4-15.4) years, one of six patients in group 2 developed metastases and died. No patients died from toxicity. The 5-year overall survival was 100% for group 1 and 80% for group 2.
  
  
  CONCLUSIONS
  This retrospective single-site study reveals a 10% incidence of high-risk features in children with retinoblastoma diagnosed at the German Retinoblastoma Referral Centre. Overall survival rates of 98.3% underline the safety of this adjuvant chemotherapy protocol and its efficiency in preventing metastasis.
authors.affiliation: Department of Pediatric Oncology and Hematology, University Hospital Essen, Essen, Germany.
authors.forename: Annette
authors.initials: A
authors.lastname: Künkele
authors.affiliation: Department of Human Genetics, University Hospital Essen, Essen, Germany.
authors.forename: Josephine
authors.initials: J
authors.lastname: Wilm
authors.affiliation: Department of Ophthalmology, University Hospital Essen, Essen, Germany.
authors.forename: Markus
authors.initials: M
authors.lastname: Holdt
authors.affiliation: Department of Human Genetics, University Hospital Essen, Essen, Germany.
authors.forename: Dietmar
authors.initials: D
authors.lastname: Lohmann
authors.affiliation: Department of Ophthalmology, University Hospital Essen, Essen, Germany.
authors.forename: Norbert
authors.initials: N
authors.lastname: Bornfeld
authors.affiliation: Department of Pediatric Oncology and Hematology, Charité Berlin, Berlin, Germany.
authors.forename: Angelika
authors.initials: A
authors.lastname: Eggert
authors.affiliation: Department of Pediatric Oncology and Hematology, University Hospital Essen, Essen, Germany.
authors.forename: Petra
authors.initials: P
authors.lastname: Temming
authors.affiliation: Department of Pediatric Oncology and Hematology, University Hospital Essen, Essen, Germany German Cancer Consortium (DKTK), Heidelberg, Germany Translational Neuro-Oncology, West German Cancer Center, University Hospital Essen, University Duisburg-Essen, Germany German Cancer Research Center (DKFZ), Heidelberg, Germany Centre for Medical Biotechnology, University Duisburg-Essen, Essen, Germany.
authors.forename: Johannes H
authors.initials: JH
authors.lastname: Schulte
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D001918:Brachytherapy; D016190:Carboplatin / Q000008:administration & dosage; D017024:Chemotherapy, Adjuvant; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage; D015353:Eye Enucleation; D005260:Female; D005858:Germany; D006801:Humans; D007223:Infant; D008297:Male; D020360:Neoadjuvant Therapy; D061766:Proton Therapy; D012017:Referral and Consultation; D019572:Retinal Neoplasms / Q000188:drug therapy* / Q000473:pathology; D012175:Retinoblastoma / Q000188:drug therapy* / Q000556:secondary; D012189:Retrospective Studies; D012307:Risk Factors; D015996:Survival Rate; D014750:Vincristine / Q000008:administration & dosage; D014821:Vitrectomy
chemical_list: D014750:Vincristine; D005047:Etoposide; D003520:Cyclophosphamide; D016190:Carboplatin
keywords: Child health (paediatrics); Drugs; Neoplasia