HR-NBL2/SIOPEN

In this protocol the term high-risk neuroblastoma refers to children with either:
● Stage M disease over the age of 12 months, any MYCN status
● L2, M or Ms neuroblastoma with MYCN amplification, any age
High-risk neuroblastoma represents the largest neuroblastoma subgroup. The prognosis of these patients has been progressively improved over the years through an intensified induction regimen, surgery of the primary tumor, high-dose chemotherapy (HDC) followed by autologous stem cell rescue (ASCR), radiotherapy and immunotherapy. As a result of this strategy, the current 3-year event-free survival (EFS) is now around 40% from date of randomization and 55% for those patients who complete all the different parts of the treatment. However, a further improvement in patient outcome is warranted.

Study primary aims:
● R-I: Comparison of the EFS rate of 2 induction regimens, GPOH and RAPID COJEC, in patients with high-risk neuroblastoma.
● R-HDC: Comparison of the EFS rate of single HDC with busulphan and melphalan (Bu-Mel) versus tandem HDC with Thiotepa followed by Bu-Mel in patients with high-risk neuroblastoma.
● R-RTx: Comparison of the EFS rate of 21.6 Gy radiotherapy to the preoperative tumor bed versus 21.6 Gy radiotherapy and a sequential boost up to 36 Gy to the residual tumor in patients with macroscopic residual disease after HDC and surgery.

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