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Aims of the study:
LOW RISK PATIENTS
● 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%.
Randomisation was closed in 2017 following IDMC recommendations.
● To maintain a 2 year EFS of at least 90% and an OS of at least 95% in L2 patients with LTS without SCA (study group 2).
● To maintain the 2 year EFS of 85% and an OS of at least 98% in Ms patients without SCA (study groups 4 and 5)
● To improve the 2 year EFS to at least 90% and maintain the OS of close to 100% in L2 patients with SCA (Study Group 3) and improve the 2 year EFS to over 70% in Ms patients with SCA (study group 6).
● To evaluate adherence to the protocol recommendations regarding LTS
● To reduce surgical morbidity by promoting strict adherence to IDRFs to determine surgical resectability
● To define the long term follow-up and natural history of the Stage L2 non-resected masses that have remained IDRF positive at the end of treatment (study groups 1-3).
● To collect tissue/biologic information and follow outcome in all LR patients.
● To estimate the incidence of NB risk groups across Europe and participating countries.
INTERMEDIATE RISK PATIENTS
● To improve the EFS to 70% with an OS of 90% of INRG stage L2 patients over the age of 18 months, with poorly differentiated or undifferentiated tumour histology (INPC criteria), by the addition of radiotherapy and 13-cis RA compared to historical conventional treatment (study group8).
● To confirm in a larger patient cohort the excellent OS of 95% in stage M neuroblastoma without MYCN amplification, less than 12 months of age, when treated with moderate therapy (study group 10).
● To maintain the excellent results of 3 year EFS of 90% and 3 year OS of 100% in stage L2 patients over the age of 18 months, with differentiating neuroblastoma or differentiating ganglioneuroblastoma nodular, despite a treatment reduction (study group 7).
● To improve the 3 year EFS to at least 50% and the 3 year OS to 80% in INSS stage I patients with MYCN amplified neuroblastoma by the addition of adjuvant treatment (study group 9).
● To evaluate the impact of the tumour genomic profile on patient outcome, in order to consider its role in the treatment stratification of these intermediate risk patients (all study groups).
● To collect tissue/biologic information and follow outcome in all IR patients.
● To estimate the incidence of NB risk groups across Europe and participating countries.

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