title: Excellent outcome with reduced treatment for infants with disseminated neuroblastoma without MYCN gene amplification.
abstract: OBJECTIVE
  On the assumption that most infants with disseminated neuroblastoma without MYCN amplification (MYCNA) have a favorable prognosis, two concomitant prospective trials were started in which chemotherapy was limited to patients presenting life- or organ-threatening symptoms or overt metastases to skeleton, lung, or CNS. Surgery was to be performed only in the absence of surgical risk factors.
  
  
  METHODS
  One hundred seventy infants with disseminated neuroblastoma without MYCNA, diagnosed between June 1999 and June 2004 in nine European countries were eligible for either of the two studies. Trial 99.2 included all stage 4S infants and those with stage 4 with a primary tumor infiltrating across the midline or positive skeletal scintigraphy who were to be observed in absence of symptoms. Trial 99.3 included infants with overt metastases to the skeleton, lung, and CNS to be treated with a minimum of four chemotherapy courses.
  
  
  RESULTS
  The 125 infants treated on trial 99.2 had a 2-year overall survival (OS) of 97.6% with no difference between asymptomatic and symptomatic patients (97.7% v 97.3%), patients without or with unresectable primary tumors (96.8% v 100%), and patients without or with positive skeletal scintigraphy without radiologic abnormalities (97.2% v 100%). The 45 infants treated on trial 99.3 had a 2-year OS of 95.6%. No patients died of surgery- or chemotherapy-related complications.
  
  
  CONCLUSIONS
  Infants with disseminated disease without MYCNA have excellent survival with minimal or no treatment. Asymptomatic infants with an unresectable primary tumor or positive skeletal scintigraphy without radiologic abnormalities may undergo observation alone.
authors.affiliation: Department of Hematology-Oncology, Giannina Gaslini Children Hospital, Largo Gerolamo Gaslini 5, 16148 Genova, Italy. brunodebernardi@ospedale-gaslini.ge.it
authors.forename: Bruno
authors.initials: B
authors.lastname: De Bernardi
authors.forename: Mary
authors.initials: M
authors.lastname: Gerrard
authors.forename: Luca
authors.initials: L
authors.lastname: Boni
authors.forename: Hervé
authors.initials: H
authors.lastname: Rubie
authors.forename: Adela
authors.initials: A
authors.lastname: Cañete
authors.forename: Andrea
authors.initials: A
authors.lastname: Di Cataldo
authors.forename: Victoria
authors.initials: V
authors.lastname: Castel
authors.forename: Ana
authors.initials: A
authors.lastname: Forjaz de Lacerda
authors.forename: Ruth
authors.initials: R
authors.lastname: Ladenstein
authors.forename: Ellen
authors.initials: E
authors.lastname: Ruud
authors.forename: Benédicte
authors.initials: B
authors.lastname: Brichard
authors.forename: Jerome
authors.initials: J
authors.lastname: Couturier
authors.forename: Caroline
authors.initials: C
authors.lastname: Ellershaw
authors.forename: Caroline
authors.initials: C
authors.lastname: Munzer
authors.forename: Paolo
authors.initials: P
authors.lastname: Bruzzi
authors.forename: Jean
authors.initials: J
authors.lastname: Michon
authors.forename: Andrew D J
authors.initials: AD
authors.lastname: Pearson
mesh_terms: D018572:Disease-Free Survival; D005060:Europe / Q000453:epidemiology; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D053208:Kaplan-Meier Estimate; D008297:Male; D009365:Neoplasm Regression, Spontaneous; D009447:Neuroblastoma / Q000401:mortality* / Q000473:pathology / Q000628:therapy*; D011379:Prognosis; D011446:Prospective Studies; D015996:Survival Rate