The 54th Congress of the International Society of Paediatric Oncology, Barcelona, Spain, 28 September - 01 October 2022, vol.167, no.1786, pp.225
Background and Aims
Relapsed or refractory (R/R) high-risk (HR) neuroblastoma (NB) has a dismal
prognosis. Anti-GD2-mediated chemo-immunotherapy has a considerable anti-tumor
activity in patients with R/R HR-NB. In the present study, we purposed to
investigate the impacts and adverse effects of the combination of immunotherapy
with dinutuximab beta (DB) and chemotherapy in patients with R/R HR-NB.
Methods
Patients of over 12 months with documentation of a HR-NB diagnosis were
eligible at relapse or designation of refractory disease status. Inclusion
criteria were as follows: relapsed or refractory, measurable by
contrast-enhanced magnetic resonance imaging (MRI) and/or computed tomography
(CT) or metaiodobenzylguanidine (MIBG)/ fluorodeoxyglucose (FDG) positron emission
tomography (PET)/CT evaluable disease and/or demonstrated by bone marrow
aspiration and biopsy. Chemotherapy scheme was irinotecan (IV, 50 mg/m² per
dose, on Days 0-4) and temozolomide (PO, 100 mg/m² per dose, on days 0-4).
Dinutuximab beta was administered intravenously for 10 days through continuous
infusion with 10 mg/m2 per day (on Days 1-10). The patients received 2 to 12
successive cycles with duration of 28 days each. Disease assessment was
performed after cycles 2, 4, and 6 and every 2 to 3 cycles thereafter.
Results
Between January 2020 and January 2022, nineteen (n=19) patients received a total of 116 cycles of DB+CT. Objective (complete or partial) responses were achieved in 12/19 (63%) patients, including complete remission (CR) in 6/19 and partial response (PR) in 6/19. Stable disease was observed in two patients. The remaining five patients developed bone/bone marrow and soft tissue progression after 2-4 cycles of treatment. The most common side effect was fever, which was more common in the first cycles of treatment. Grade ≥3 toxicities were leukopenia (62%), thrombocytopenia (27%), hypertransaminasemia (25%), fever (14%), and rash/itching (11%), respectively.
Conclusions
DB-based chemo-immunotherapy is suitable leading to an encouraging response
rate in patients with R/R HR-NB