Originally published as JCO Early Release 10.1200/JCO.2008.17.5877 on January 26 2009

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Excellent Outcome With Reduced Treatment for Infants With Disseminated Neuroblastoma Without MYCN Gene Amplification

From the Istituto Giannina Gaslini, and Istituto Nazionale Ricerca sul Cancro, Genova, Istituto Toscano Tumori, Firenze, and Università di Catania, Catania, Italy; Hôpital des Enfants, Toulouse, and Institut Curie, Paris, France; Hospital La Fe, Valencia, Spain; Instituto Português de Oncologia, Lisboa, Portugal; Rikshospitalet, Oslo, Norway; St Anna Kinderspital, Vienna, Austria; Saint Luc Cliniques Universitaires, Brussels, Belgium; Children's Cancer and Leukaemia Group Data Centre, University of Leicester, Sheffield Children's Hospital, and Institute of Cancer Research/Royal Marsden Hospital Sutton, United Kingdom.

Corresponding author: Bruno De Bernardi, MD, Department of Hematology-Oncology, Giannina Gaslini Children Hospital, Largo Gerolamo Gaslini 5, 16148 Genova, Italy; e-mail: brunodebernardi{at}ospedale-gaslini.ge.it.

Purpose 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.

Patients and 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.

Conclusion 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.

Supported by the Institut Curie, Association pour la Recherche sur le Cancer and Association Hubert Gouin, Paris, France; the Fondazione Italiana per la Lotta al Neuroblastoma, Finalizzata Ministeriale RF-IGG-2006-345206, and Istituto Giannina Gaslini, Genova, Italy; and AMGEN France.

Authors' disclosures of potential conflicts of interest and author contributions are found at the end of this article.

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