title: Results of a multicenter prospective study on the postoperative treatment of unilateral retinoblastoma after primary enucleation.
abstract: OBJECTIVE
  The objective of this prospective study was to assess overall survival and event-free survival in patients with intraocular unilateral retinoblastoma (Reese-Ellsworth group V) treated by primary enucleation with or without adjuvant therapy depending on histopathologic risk factors.
  
  
  METHODS
  Patients (n = 123) were divided into three groups on the basis of risk factors for extraocular relapse and metastasis assessed on centralized histologic examination of enucleated eyes. Group 1 (n = 70) had minimal or no choroidal involvement and/or prelaminar or no optic nerve involvement and received no adjuvant therapy. Group 2 (n = 52) had massive choroidal involvement and/or intra- or retrolaminar optic nerve involvement and/or anterior segment involvement and received four courses of adjuvant chemotherapy. Group 3 (n = 1) had invasion of the surgical margin of the optic nerve and/or microscopic extrascleral involvement and received six courses of adjuvant chemotherapy with intrathecal thiotepa, consolidation chemotherapy, and autologous stem-cell rescue. Genetic testing was also performed.
  
  
  RESULTS
  Median follow-up for the 123 patients was 71 months. No disease progression, relapse, or distant metastasis occurred during follow-up. No second malignancies occurred. This requires confirmation with longer follow-up. Secondary bilateralization occurred in two patients with identified RB1 germline mutation. Adjuvant chemotherapy was well tolerated, with limited toxicity. Molecular testing found constitutional RB1 gene mutations in only nine of 100 evaluated patients.
  
  
  CONCLUSIONS
  The survival rate of 100% was excellent, including 57% of patients who received no adjuvant therapy, suggesting that chemotherapy could be de-escalated in some patients, especially those with massive choroidal involvement.
authors: Department of Pediatric Oncology, Institut Curie-Hôpital, 26 rue d'Ulm 75248, Paris Cedex 05, France. isabelle.aerts@curie.net
authors: Isabelle
authors: I
authors: Aerts
authors: Xavier
authors: X
authors: Sastre-Garau
authors: Alexia
authors: A
authors: Savignoni
authors: Livia
authors: L
authors: Lumbroso-Le Rouic
authors: Estelle
authors: E
authors: Thebaud-Leculée
authors: Didier
authors: D
authors: Frappaz
authors: Carole
authors: C
authors: Coze
authors: Caroline
authors: C
authors: Thomas
authors: Marion
authors: M
authors: Gauthier-Villars
authors: Christine
authors: C
authors: Lévy-Gabriel
authors: Hervé J
authors: HJ
authors: Brisse
authors: Laurence
authors: L
authors: Desjardins
authors: François
authors: F
authors: Doz
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D017024:Chemotherapy, Adjuvant / Q000379:methods; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D015353:Eye Enucleation / Q000379:methods; D005260:Female; D005334:Fever / Q000139:chemically induced; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D053208:Kaplan-Meier Estimate; D008297:Male; D052016:Mucositis / Q000139:chemically induced; D009154:Mutation; D009503:Neutropenia / Q000139:chemically induced; D011182:Postoperative Care / Q000379:methods; D011446:Prospective Studies; D012160:Retina / Q000187:drug effects* / Q000473:pathology / Q000601:surgery; D019572:Retinal Neoplasms / Q000188:drug therapy* / Q000235:genetics / Q000601:surgery; D012175:Retinoblastoma / Q000188:drug therapy* / Q000235:genetics / Q000601:surgery; D016160:Retinoblastoma Protein / Q000235:genetics; D016896:Treatment Outcome
chemical_list: D016160:Retinoblastoma Protein