Le Pellerin

Researchers are evaluating treatments for children and young adults with high-risk neuroblastoma, a type of cancer affecting nerve tissue. This study focuses on patients with advanced stages of the disease, including those with tumors that have an amplified MYCN gene, which is linked to a higher risk of relapse. The trial is part of the European SIOP Neuroblastoma Group (SIOPEN) and aims to improve outcomes using various chemotherapy and immunotherapy strategies. Participants receive a rapid, intensive induction chemotherapy regimen called Rapid COJEC, followed by peripheral blood stem cell collection and surgery to remove the primary tumor. Depending on their response, some patients may receive additional chemotherapy cycles. Consolidation treatment involves high-dose chemotherapy with busulfan and melphalan (BuMel MAT), stem cell rescue, and radiotherapy. The study also tests immunotherapy using the ch14.18/CHO antibody (Dinutuximab beta, Qarziba®) given as a continuous infusion, with or without subcutaneous aldesleukin (IL-2), alongside isotretinoin (13-cis-RA). Patients diagnosed after June 2017 receive immunotherapy without IL-2 as standard care outside trials. During the study, participants undergo various assessments including tumor response evaluations and laboratory tests to monitor their health and treatment effects. The main outcomes measured are event-free survival up to three years and complete metastatic response within about 95 days after induction therapy. Safety and long-term effects are followed regularly over at least five years. The study collects biological tumor samples to assess prognostic factors and requires informed consent and ethical approval before participation.