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

被引:105
作者
De Bernardi, Bruno
Gerrard, Mary
Boni, Luca
Rubie, Herve
Canete, Adela
Di Cataldo, Andrea
Castel, Victoria
de Lacerda, Ana Forjaz
Ladenstein, Ruth
Ruud, Ellen
Brichard, Benedicte
Couturier, Jerome
Ellershaw, Caroline
Munzer, Caroline
Bruzzi, Paolo
Michon, Jean
Pearson, Andrew D. J.
机构
[1] Ist Giannina Gaslini, I-16148 Genoa, Italy
[2] Ist Nazl Ric Canc, I-16132 Genoa, Italy
[3] Ist Toscano Tumori, Florence, Italy
[4] Univ Catania, Catania, Italy
[5] Hop Enfants, Toulouse, France
[6] Inst Curie, Paris, France
[7] Hosp La Fe, E-46009 Valencia, Spain
[8] Inst Portugues Oncol Francisco Gentil, Lisbon, Portugal
[9] Univ Oslo, Rikshosp, N-0027 Oslo, Norway
[10] St Anna Childrens Hosp, Vienna, Austria
[11] Clin Univ St Luc, B-1200 Brussels, Belgium
[12] Univ Leicester, Childrens Canc & Leukaemia Grp, Ctr Data, Sheffield Childrens Hosp, Leicester LE1 7RH, Leics, England
[13] Royal Marsden Hosp Sutton, Inst Canc Res, Sutton, Surrey, England
关键词
STAGE-IV-S; PEDIATRIC-ONCOLOGY-GROUP; CHILDRENS CANCER; PATHOLOGY CLASSIFICATION; METASTATIC NEUROBLASTOMA; LOCALIZED NEUROBLASTOMA; DIAGNOSIS; PROGNOSIS; CRITERIA; THERAPY;
D O I
10.1200/JCO.2008.17.5877
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
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. J Clin Oncol 27: 1034-1040. (C) 2009 by American Society of Clinical Oncology
引用
收藏
页码:1034 / 1040
页数:7
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