Risk stratification of high-risk metastatic neuroblastoma: A report from the HR-NBL-1/SIOPEN study.

Détails

ID Serval
serval:BIB_9982CD87F060
Type
Article: article d'un périodique ou d'un magazine.
Collection
Publications
Institution
Titre
Risk stratification of high-risk metastatic neuroblastoma: A report from the HR-NBL-1/SIOPEN study.
Périodique
Pediatric blood & cancer
Auteur⸱e⸱s
Morgenstern D.A., Pötschger U., Moreno L., Papadakis V., Owens C., Ash S., Pasqualini C., Luksch R., Garaventa A., Canete A., Elliot M., Wieczorek A., Laureys G., Kogner P., Malis J., Ruud E., Beck-Popovic M., Schleiermacher G., Valteau-Couanet D., Ladenstein R.
ISSN
1545-5017 (Electronic)
ISSN-L
1545-5009
Statut éditorial
Publié
Date de publication
11/2018
Peer-reviewed
Oui
Volume
65
Numéro
11
Pages
e27363
Langue
anglais
Notes
Publication types: Journal Article ; Research Support, Non-U.S. Gov't
Publication Status: ppublish
Résumé
Risk stratification is crucial to treatment decision-making in neuroblastoma. This study aimed to explore factors present at diagnosis affecting outcome in patients aged ≥18 months with metastatic neuroblastoma and to develop a simple risk score for prognostication.
Data were derived from the European high-risk neuroblastoma 1 (HR-NBL1)/International Society for Paediatric Oncology European Neuroblastoma (SIOPEN) trial with analysis restricted to patients aged ≥18 months with metastatic disease and treated prior to the introduction of immunotherapy. Primary endpoint was 5-year event-free survival (EFS). Prognostic factors assessed were sex, age, tumour MYCN amplification (MNA) status, serum lactate dehydrogenase (LDH)/ferritin, primary tumour and metastatic sites. Factors significant in univariate analysis were incorporated into a multi-variable model and an additive scoring system developed based on estimated log-cumulative hazard ratios.
The cohort included 1053 patients with median follow-up 5.5 years and EFS 27 ± 1%. In univariate analyses, age; serum LDH and ferritin; involvement of bone marrow, bone, liver or lung; and >1 metastatic system/compartment were associated with worse EFS. Tumour MNA was not associated with worse EFS. A multi-variable model and risk score incorporating age (>5 years, 2 points), serum LDH (>1250 U/L, 1 point) and number of metastatic systems (>1, 2 points) were developed. EFS was significantly correlated with risk score: EFS 52 ± 9% for score = 0 versus 6 ± 3% for score = 5 (P < 0.0001).
A simple score can identify an "ultra-high risk" (UHR) cohort (score = 5) comprising 8% of patients with 5-year EFS <10%. These patients appear not to benefit from induction therapy and could potentially be directed earlier to alternative experimental therapies in future trials.
Mots-clé
Age Factors, Biomarkers, Tumor/analysis, Child, Child, Preschool, Clinical Trials as Topic, Disease-Free Survival, Female, Ferritins/blood, Humans, Infant, Kaplan-Meier Estimate, L-Lactate Dehydrogenase/blood, Male, N-Myc Proto-Oncogene Protein/genetics, Neuroblastoma/mortality, Neuroblastoma/pathology, Prognosis, Progression-Free Survival, Proportional Hazards Models, Risk Factors, Sex Factors, lactate dehydrogenase, metastatic, neuroblastoma, relapse, risk stratification, ultra-high risk
Pubmed
Web of science
Création de la notice
31/07/2018 14:52
Dernière modification de la notice
20/08/2019 15:01
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