Excellent outcome with reduced treatment for infants with disseminated neuroblastoma without MYCN gene amplification.
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.
De Bernardi
Bruno
B
Department of Hematology-Oncology, Giannina Gaslini Children Hospital, Largo Gerolamo Gaslini 5, 16148 Genova, Italy. brunodebernardi@ospedale-gaslini.ge.it
Gerrard
Mary
M
Boni
Luca
L
Rubie
Hervé
H
Cañete
Adela
A
Di Cataldo
Andrea
A
Castel
Victoria
V
Forjaz de Lacerda
Ana
A
Ladenstein
Ruth
R
Ruud
Ellen
E
Brichard
Benédicte
B
Couturier
Jerome
J
Ellershaw
Caroline
C
Munzer
Caroline
C
Bruzzi
Paolo
P
Michon
Jean
J
Pearson
Andrew D J
AD
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