TY - JOUR
T1 - Chemo-immunotherapy with dinutuximab beta in patients with relapsed/progressive high-risk neuroblastoma
T2 - does chemotherapy backbone matter?
AU - Raiser, Patricia
AU - Schleiermacher, Gudrun
AU - Gambart, Marion
AU - Dumont, Benoit
AU - Defachelles, Anne Sophie
AU - Thebaud, Estelle
AU - Tandonnet, Julie
AU - Pasqualini, Claudia
AU - Proust, Stéphanie
AU - Entz-Werle, Natacha
AU - Aerts, Isabelle
AU - Ndounga-Diakou, Lee A.
AU - Petit, Arnaud
AU - Puiseux, Chloe
AU - Khanfar, Camille
AU - Rouger, Jeremie
AU - Mansuy, Ludovic
AU - Benadiba, Joy
AU - Millot, Frédéric
AU - Pluchart, Claire
AU - Laghouati, Salim
AU - Geoerger, Birgit
AU - Vassal, Gilles
AU - Valteau-Couanet, Dominique
AU - Berlanga, Pablo
N1 - Publisher Copyright:
© 2024 The Authors
PY - 2024/5/1
Y1 - 2024/5/1
N2 - Background: Addition of anti-GD2 antibodies to temozolomide-based chemotherapy has demonstrated increased antitumor activity and progression-free survival in patients with relapsed/progressive high-risk neuroblastoma. However, chemo-immunotherapy is not yet approved for this indication. This study presents the chemo-immunotherapy experience in patients with relapsed/progressive high-risk neuroblastoma treated within the off-label use program of the Neuroblastoma Committee of the French Society of Pediatric Oncology (SFCE). Methods: Dinutuximab beta (dB) was administered alongside temozolomide-topotecan (TOTEM) or temozolomide-irinotecan (TEMIRI) at first disease relapse/progression or topotecan-cyclophosphamide (TopoCyclo) at further relapse/progression. Real-world data on demographics, treatment, antitumor activity and safety was collected from all patients after inclusion in SACHA-France (NCT04477681), a prospective national registry, which documents safety and efficacy data on innovative anticancer therapies prescribed to patients ≤ 25 years old as compassionate or off-label use. Results: Between February 2021 and July 2023, 39 patients with confirmed relapsed/progressive high-risk neuroblastoma (median age 6 years, range 1–24) were treated with dB+TopoCyclo (n = 24) or dB+TOTEM/TEMIRI (n = 15) across 17 centers. In total, 163 chemo-immunotherapy cycles were administered, main toxicities were mild or moderate, with higher incidence of hematological adverse drug reactions with dB+TopoCyclo than dB+TOTEM/TEMIRI. Objective response rate was 42% for dB+TopoCyclo (CI95% 22–63%) and 40% for dB+TOTEM/TEMIRI (CI95% 16–68%). Conclusion: Similar objective response rates for dB+TopoCyclo and dB+TOTEM/TEMIRI in patients with relapsed/progressive high-risk neuroblastoma emphasize the importance of chemo-immunotherapy, irrespective of the chemotherapy backbone.
AB - Background: Addition of anti-GD2 antibodies to temozolomide-based chemotherapy has demonstrated increased antitumor activity and progression-free survival in patients with relapsed/progressive high-risk neuroblastoma. However, chemo-immunotherapy is not yet approved for this indication. This study presents the chemo-immunotherapy experience in patients with relapsed/progressive high-risk neuroblastoma treated within the off-label use program of the Neuroblastoma Committee of the French Society of Pediatric Oncology (SFCE). Methods: Dinutuximab beta (dB) was administered alongside temozolomide-topotecan (TOTEM) or temozolomide-irinotecan (TEMIRI) at first disease relapse/progression or topotecan-cyclophosphamide (TopoCyclo) at further relapse/progression. Real-world data on demographics, treatment, antitumor activity and safety was collected from all patients after inclusion in SACHA-France (NCT04477681), a prospective national registry, which documents safety and efficacy data on innovative anticancer therapies prescribed to patients ≤ 25 years old as compassionate or off-label use. Results: Between February 2021 and July 2023, 39 patients with confirmed relapsed/progressive high-risk neuroblastoma (median age 6 years, range 1–24) were treated with dB+TopoCyclo (n = 24) or dB+TOTEM/TEMIRI (n = 15) across 17 centers. In total, 163 chemo-immunotherapy cycles were administered, main toxicities were mild or moderate, with higher incidence of hematological adverse drug reactions with dB+TopoCyclo than dB+TOTEM/TEMIRI. Objective response rate was 42% for dB+TopoCyclo (CI95% 22–63%) and 40% for dB+TOTEM/TEMIRI (CI95% 16–68%). Conclusion: Similar objective response rates for dB+TopoCyclo and dB+TOTEM/TEMIRI in patients with relapsed/progressive high-risk neuroblastoma emphasize the importance of chemo-immunotherapy, irrespective of the chemotherapy backbone.
KW - Chemotherapy
KW - Dinutuximab beta
KW - Immunotherapy
KW - Neuroblastoma
KW - Real-world data
KW - Relapse
UR - http://www.scopus.com/inward/record.url?scp=85187987724&partnerID=8YFLogxK
U2 - 10.1016/j.ejca.2024.114001
DO - 10.1016/j.ejca.2024.114001
M3 - Article
C2 - 38489858
AN - SCOPUS:85187987724
SN - 0959-8049
VL - 202
JO - European Journal of Cancer
JF - European Journal of Cancer
M1 - 114001
ER -