Abstract
Background: Treatment of HR-NB comprise induction, consolidation with autologous stem cell transplant (ASCT) followed by anti-GD2 immunotherapy and isotretinoin. Childrens Oncology Group and SIOPEN studies used dinutuximab and cytokines to treat patients in complete remission or refractory Bone/Bone marrow (B/BM) disease after ASCT. Methods: HR-NB patients referred to Hospital Sant Joan de Déu for anti-GD2 immunotherapy were eligible for two consecutive studies (dinutuximab for EudraCT 2013–004864–69 and naxitamab for 017–001829–40) and naxitamab/Sargramostim CU with or without prior ASCT. Patients enrolled in first complete remission or with primary refractory B/BM disease. We accrued a study population of two groups whose therapy, aside from ASCT, was similar. This is a retrospective analysis of their outcome calculated from study entry. Results: From December 2014–2019, 67 patients were treated with dinutuximab and cytokines (n = 21) in the Hospital Sant Joan de Déu-HRNB-Ch14.18 study or with naxitamab and Sargramostim either in the Ymabs study 201 (n = 12) or CU (n = 34). 23 patients were treated with primary refractory disease in the B/BM (11 with dinutuximab and 12 with naxitamab), and 44 in first CR (10 with dinutuximab and 34 with naxitamab). Study patients included 13 (19.4%) treated following single ASCT and 54 following conventional chemotherapy. Median follow-up for all patients is 16.2 months. Two-year rates for ASCT and non-ASCT patients were, respectively, EFS 64.1% vs. 54.2% (p = 0.28), and OS 66.7% vs. 84.1% (p = 0.81). For the 44 pts in first CR, 2-years rates for ASCT and non-ASCT patients were, respectively, EFS 65.5% vs. 58.7% (p = 0.48), and OS 71.4% vs. 85.4% (p = 0.63). Conclusions: In this retrospective, single center study, ASCT did not provide survival benefit when anti-GD2 immunotherapy was used after induction chemotherapy.
Highlights
High-risk neuroblastoma is defined by metastatic disease over the age of 18 months or MYCN amplification at any age (Fletcher et al, 2018)
HR-NB patients referred to Hospital Sant Joan de Déu for anti-GD2 immunotherapy were eligible for two consecutive studies and naxitamab/Sargramostim CU with or without prior autologous stem cell transplant (ASCT)
From December 2014–2019, 67 patients were treated with dinutuximab and cytokines (n 21) in the Hospital Sant Joan de Déu-HRNB-Ch14.18 study or with naxitamab and Sargramostim either in the Ymabs study 201 (n 12) or CU (n 34). 23 patients were treated with primary refractory disease in the Bone/Bone marrow (B/BM) (11 with dinutuximab and 12 with naxitamab), and 44 in first CR (10 with dinutuximab and 34 with naxitamab)
Summary
High-risk neuroblastoma is defined by metastatic disease over the age of 18 months or MYCN amplification at any age (Fletcher et al, 2018). Treatment approaches within the major international cooperative groups (GPOH, COG and SIOP) comprise a backbone of intensive induction, consolidation with high dose chemotherapy and ASCT, and isotretinoin as maintenance therapy. The most recent report from a cooperative group study with this classical approach (pre anti-GD2 immunotherapy era) is from the GPOH trial NB 2004 (Berthold et al, 2020). In 2010 the results of the COG ANBL0032 trial were published demonstrating that the 2-years EFS and OS of patients with HR-NB achieving major responses (after standard induction and consolidation -including ASCT-) and receiving anti-GD2 immunotherapy (dinutuximab and cytokines: GM-CSF and IL-2) in addition to isotretinoin were significantly higher (2-y EFS 66 ± 5% and OS 86 ± 4%) compared to those patients receiving isotretinoin alone (2-y EFS 46 ± 5% and OS 75 ± 5%) (Yu et al, 2010). Childrens Oncology Group and SIOPEN studies used dinutuximab and cytokines to treat patients in complete remission or refractory Bone/Bone marrow (B/BM) disease after ASCT
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