AUTHOR=Mora Jaume , Castañeda Alicia , Flores Miguel A. , Santa-María Vicente , Garraus Moira , Gorostegui Maite , Simao Margarida , Perez-Jaume Sara , Mañe Salvador 

TITLE=The Role of Autologous Stem-Cell Transplantation in High-Risk Neuroblastoma Consolidated by anti-GD2 Immunotherapy. Results of Two Consecutive Studies

JOURNAL=Frontiers in Pharmacology

VOLUME=Volume 11 - 2020

YEAR=2020

URL=https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2020.575009

DOI=10.3389/fphar.2020.575009

ISSN=1663-9812

ABSTRACT=Background: Treatment of HR-NB comprise induction, consolidation with ASCT followed by anti-GD2 immunotherapy and isotretinoin. COG and SIOPEN studies used dinutuximab and cytokines to treat patients in complete remission or refractory B/BM disease after ASCT.
Methods: HR-NB patients referred to HSJD for anti-GD2 immunotherapy were eligible for 2 consecutive studies (dinutuximab for EudraCT 2013-004864-69 and naxitamab for 017-001829-40) and naxitamab/GM-CSF CU with or without prior ASCT. Patients enrolled in first CR 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 HSJD-HRNB-Ch14.18 study or with naxitamab and GM-CSF 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-year 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.