Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Observational Study
. 2025 Dec;72(12):e32080.
doi: 10.1002/pbc.32080. Epub 2025 Oct 3.

Chemo-Immunotherapy Rescue for High-Risk Neuroblastoma Patients With Progressive Disease Before High-Dose Chemotherapy: Real-World Data From the SACHA-France Study

Claudia Pasqualini  1 , Stéphanie Proust  2 , Gudrun Schleiermacher  3 , Marion Gambart  4 , Sarah Jannier  5 , Arnaud Petit  6 , Chrystelle Dupraz  7 , Estelle Thebaud  8 , Yves Reguerre  9 , Lee Aymar Ndounga-Diakou  10 , Salim Laghouat  10 , Anne Sophie Defachelles  11 , Pablo Berlanga  1
Affiliations
Observational Study

Chemo-Immunotherapy Rescue for High-Risk Neuroblastoma Patients With Progressive Disease Before High-Dose Chemotherapy: Real-World Data From the SACHA-France Study

Claudia Pasqualini et al. Pediatr Blood Cancer. 2025 Dec.

Abstract

Background: Patients with high-risk neuroblastoma (HR-NBL) who experience disease progression (PD) during first-line treatment prior to high-dose chemotherapy (HDC) represent a rare and understudied subgroup, for whom treatment strategies are poorly defined and prognosis appears to be extremely poor.

Aims: We report real-world data on the off-label use of chemo-immunotherapy for HR-NBL patients with PD before HDC. The primary endpoint of our analysis is the best response during the chemo-immunotherapy treatment.

Methods: The SACHA-France registry prospectively documents safety and efficacy data on compassionate/off-label treatments for patients of ≤25 years old (NCT04477681).

Results: Between January 2020 and September 2024, 13 patients with HR-NBL received chemo-immunotherapy due to PD before HDC, and were included in SACHA-France. They had a median age of 3.4 years (1.3-8.0). Six patients had NMYC-amplified disease. Five PD occurred during/end-of-induction chemotherapy, and eight during temozolomide-based chemotherapy after initial insufficient metastatic response or toxicity. All but one had metastatic progression. The chemo-immunotherapy consisted of topotecan-cyclophosphamide (n = 9) or temozolomide-irinotecan (n = 4), combined with dinutuximab beta (dB) for a maximum of six cycles. Objective responses (ORs) were seen in five of 13 patients (38%)-four partial responses (PR) and one complete response (CR). Three out of the five patients with PD during/end of induction had PR, including two with NMYC-amplified tumors. Overall, six patients underwent tandem HDC, with two remaining progression-free after 1.7 and 2.1 years, and one remaining disease-free at 3.4 years.

Conclusion: Chemo-immunotherapy can benefit HR-NBL patients with PD before HDC, including those with progression during/at the end of the induction chemotherapy and NMYC amplification. These findings support its early inclusion in HR-NBL trials.

Keywords: chemo‐immunotherapy; high‐dose chemotherapy; high‐risk neuroblastoma; progressive disease; real‐world data.

PubMed Disclaimer

References

    1. A. Garaventa, U. Pötschger, D. Valteau‐Couanet, et al., “Randomized Trial of Two Induction Therapy Regimens for High‐Risk Neuroblastoma: HR‐NBL1.5 International Society of Pediatric Oncology European Neuroblastoma Group Study,” Journal of Clinical Oncology 39, no. 23 (2021): 2552–2563.
    1. J. R. Park, S. G. Kreissman, W. B. London, et al., “Effect of Tandem Autologous Stem Cell Transplant vs Single Transplant on Event‐Free Survival in Patients with High‐Risk Neuroblastoma: A Randomized Clinical Trial,” JAMA 322, no. 8 (2019): 746.
    1. N. Pinto, A. Naranjo, E. Hibbitts, et al., “Predictors of Differential Response to Induction Therapy in High‐Risk Neuroblastoma: A Report From the Children's Oncology Group (COG),” European Journal of Cancer 112 (2019): 66–79.
    1. A. L. Yu, A. L. Gilman, M. V. Ozkaynak, et al., “Anti‐GD2 Antibody With GM‐CSF, Interleukin‐2, and Isotretinoin for Neuroblastoma,” New England Journal of Medicine 363, no. 14 (2010): 1324–1334.
    1. R. Ladenstein, U. Pötschger, D. Valteau‐Couanet, et al., “Interleukin 2 With Anti‐GD2 Antibody ch14.18/CHO (dinutuximab beta) in Patients With High‐Risk Neuroblastoma (HR‐NBL1/SIOPEN): A Multicentre, Randomised, Phase 3 Trial,” Lancet Oncology 19, no. 12 (2018): 1617–1629.

Publication types

Associated data