Real-World Use of Dinutuximab Beta for High-Risk Neuroblastoma in Clinical Settings in France: The QUATUOR Study.

Dinutuximab beta (DB) is recommended for maintenance therapy in high-risk neuroblastoma (HR-NB) in Europe. In France, early access to DB was provided in the Temporary Use Authorization (ATU) program. The QUATUOR (Qarziba Use ATU Outcomes Research) study is an observational, multicenter, retrospective study that evaluated the effectiveness and safety of DB in HR-NB patients who received DB in maintenance between February 2017 and August 2018 in real-world clinical settings in France as part of the ATU program. Fourteen patients received DB in the frontline and 17 in the refractory/relapsed setting. In the frontline group, 2-year EFS and OS rates were 64.3% (95% CI: 34.3-83.3%) and 78.6% (95% CI: 47.3-92.5%), respectively, and in the relapsed/refractory group, 57.5% (95% CI: 30.7-77.2%) and 80.9% (95% CI: 51.3-93.5%), respectively. Complete or partial response of the metastatic sites was maintained by 23/31 evaluable patients at the end of treatment. Overall, DB was effective, with acceptable tolerability, in patients with HR-NB treated as part of the ATU program in France. This study demonstrates the importance of successful collaboration between national health authorities, pharmaceutical companies, physicians, and the neuroblastoma community to provide early access to innovative treatments.