European Low and Intermediate Risk Neuroblastoma Protocol
1. LOW RISK STUDY
The low risk group of patients includes NB patients without MYCN amplification with or
without life threatening symptoms in the following clinical situations:
- Children aged ≤ 18 months with localised neuroblastoma associated with image defined
risk factors precluding upfront surgery (stage INRG L2).
- Children aged ≤ 12 months with disseminated neuroblastoma without bone, pleura, lung or
CNS (Central Nervous System) disease (stage INRG Ms)
2) INTERMEDIATE RISK STUDY
The intermediate risk group of patients includes NB patients in the following clinical
- Children aged >18 months with localised neuroblastoma without MYCN amplification,
associated with image defined risk factors precluding upfront surgery (stage INRG L2).
- Children aged ≤12 months with disseminated neuroblastoma involving bone, pleura, lung
and/or CNS (stage INRG M), without MYCN amplification.
- Children with localised resected NB (stage INSS I) with MYCN amplification. NEONATAL
The incidence of suprarenal tumours/masses has increased in the last decade due to the
expanded use of prenatal ultrasonography in routine obstetric care and in the neonatal and
early infancy care. The differential diagnosis of these masses ranges from benign (adrenal
haemorrhage) to malignant processes (neuroblastoma, adrenal carcinoma). Knowledge on
perinatal suprarenal masses, although based on a relatively large literature, is scattered
amongst studies on very few cases with no methodical approach and often short follow up.
Therefore, the optimal management of these masses has not been clearly defined.
Neuroblastoma at this age is an intriguing entity with a very good prognosis in most cases.
The SIOPEN Group, based on their results in the first multicenter European Trial for infants
with neuroblastoma (INES) and the world-wide experience provided in the literature, is
launching this European surveillance study (Multi-centre, non-blinded, one armed prospective
trial) for these masses. Treatment: Observation
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary aim for Low Risk Neuroblastoma
To demonstrate through a randomisation between observation and chemotherapy that you can safely reduce treatment in a subgroup of L2 low risk patients (those without life threatening symptoms (LTS) and without any segmental chromosomal changes (SCA), i.e. study group 1) by giving less treatment than has been given historically while maintaining an excellent OS of 100%.
Adela Cañete, MD, PhD
Hospital Universitari i Politècnic La Fe, Valencia, Spain
Spain: Spanish Agency of Medicines