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.

Authors

Institutions

Publication Details

Journal
Journal of Pediatric Hematology/Oncology
Published
2026-10-15
DOI
https://doi.org/10.1097/mph.0000000000003302
Primary Topic
Neuroblastoma Research and Treatments
Type
article
Field-Weighted Citation Impact
0.00
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

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

Sarah Benezech, M Deparis, Dominique Valteau‐Couanet, L Mansuy et al.
Journal of Pediatric Hematology/Oncology
Neuroblastoma Research and Treatments
article

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

Sarah Benezech, M Deparis, Dominique Valteau‐Couanet, L Mansuy, Dominique Plantaz, Estelle Thébaud, Anne Sophie Defachelles, Julie Tandonnet, Joy Benadiba, Gudrun Schleiermacher, Sarah Jannier, Carole Coze, Véronique Laithier, Stéphanie Proust, Chrystelle Dupraz
article en

Abstract

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.

Journal of Pediatric Hematology/Oncology
Université de Bordeaux (FR), Université Paris Sciences et Lettres (FR), Hotel Dieu Hospital (CA), Institut Gustave Roussy (FR), Centre Hospitalier Universitaire de Besançon (FR), Hôpital Couple Enfant (FR), Hospices Civils de Lyon (FR), Hôpital d'Enfants (FR), Bordeaux Population Health (FR), Centre Hospitalier Universitaire de Poitiers (FR), Centre Hospitalier Universitaire d'Angers (FR), Centre Hospitalier Universitaire de Caen Normandie (FR), Hôpital l'Archet (FR), Centre Oscar Lambret (FR), Hôpital d'Hautepierre (FR), Hôpital de la Timone (FR), Université de Caen Normandie (FR)
Good health and well-being
Openalex Percentile: Top 14%
Neuroblastoma Research and Treatments
DOIOpen Access PDFOpenAlex
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.