Tandem Transplant for High-Risk Neuroblastoma: Next Steps in the Era of Precision Medicine
Mené sur 355 patients atteints d'un neuroblastome à haut risque de récidive, cet essai randomisé compare l'efficacité, du point de vue de la survie sans événement, et la toxicité d'une greffe autologue de cellules souches et d'une greffe autologue en tandem (intervention répétée quelques mois après la première greffe)
Neuroblastoma, a malignancy of the sympathetic nervous system that arises in cells derived from the neural crest, predominantly affects young children. Although survival rates have improved significantly,1-3 neuroblastoma remains challenging due, in part, to its biological and clinical heterogeneity. Subsets of children fare well with minimal therapy, with cure rates of close to 100% for children with specific disease characteristics; however, for high-risk patients the cure rates are approximately 50%.1,2 Current risk stratification systems consider patient age and stage as well as tumor histology and genomic features, permitting delivery of therapy that is based on the likelihood of disease recurrence. Only observation is required for some patients, whereas for other patients, maximally intensive multimodality therapy is necessary.
JAMA 2019