Survival in patients with High-Risk Neuroblastoma without autologous
stem cell transplant and dinutuximab
Abstract
Background: The majority of patients with high-risk neuroblastoma
(HR-NB) in low- and middle-income countries (LMIC) do not have access to
autologous stem cell transplant (ASCT) and dinutuximab. Consolidation
with non-myeloablative chemotherapy is not well-defined, and the
outcomes are variable. We report a single-center outcome of patients
with HR-NB, treated with non-myeloablative consolidation. A tabulated
compilation of similar reports is included. Procedure: A retrospective
chart review of patients with HR-NB was performed from January 2009 till
June 2016. Patients were treated on the backbone of HR-NBL1/SIOPEN
protocol. Treatment included induction with rapid-COJEC, surgery,
consolidation, radiotherapy to the primary tumor, and differentiation
therapy with isotretinoin. Consolidation included 4 cycles of topotecan,
vincristine, and doxorubicin (TVD) instead of ASCT. Infusion of
vincristine and doxorubicin were modified for ease and to enable
administration in daycare. Results: Over 7-½ years, 28 patients with
HR-NB were treated. Two (7%) patients had therapy-related mortality. A
relapse or disease progression occurred in 11 (39%) patients at a
median duration of 17 months (IQR: 5, 18). Treatment abandonment was
observed in 4 (14%) patients. The 4-year event-free survival was
29.3%. The median follow up of disease-free patients is 49 months (IQR:
45, 79). Patients with relapse were not treated further. Conclusions: A
4-year EFS of 29.3% was observed when 4-cycles of TVD were administered
instead of ASCT in patients with HR-NB. The study and the review will
aid stakeholders in LMIC for decision-making while considering the
options of treatment for HR-NB if access to ACST and dinutuximab is
lacking.