Abstract
BackgroundNeuroblastoma is the most common pediatric extracranial tumor with varied prognoses, but the survival of treated refractory or relapsing patients remains poor.ObjectiveThis analysis presents the outcomes of children with neuroblastoma undergoing MIBG therapy in Poland in 2006-2019.Study DesignA retrospective cohort of 55 patients with refractory or relapsed neuroblastoma treated with I-131 MIBG in Poland in 2006-2019 was analyzed. The endpoints were overall survival (OS), event-free survival (EFS), cumulative incidence (CI) of second cancers and CI of hypothyroidism. Survival curves were estimated using the Kaplan-Meier method and compared between the cohorts by the log-rank test. Cox modeling was adopted to estimate hazard ratios for OS and EFS, considering factors with P < 0.2.ResultsFifty-five patients with a median age of 78.4 months (range 18-193) with neuroblastoma underwent one or more (4 patients) courses of MIBG I-131 therapy. Fifteen patients were not administered chemotherapy, 3 children received standard-dose chemotherapy, and 37 patients were administered high-dose chemotherapy (HDCT) (busulfan-melphalan in 24 and treosulfan-based in 12 patients). Forty-six patients underwent stem cell transplantation, with autologous (35 patients), haploidentical (6), allogeneic (4), and syngeneic grafts (1). The median time from first MIBG therapy to SCT was 22 days. Children with relapsing tumors had inferior OS compared to those with primary resistant disease (21.2% vs 58.7%, p=0.0045). Survival was better in patients without MYCN gene amplification. MIBG therapy was never curative, except in patients further treated with HDCT with stem cell rescue irrespective of the donor type. 31 patients were referred for immune therapy after MIBG therapy, and the 5-year OS in this group was superior to the untreated children (55.2% vs 32.7%, p=0.003), but the difference in the 5-year EFS was not significant (25.6% vs 32.9%, p=ns). In 3 patients, a second malignancy was diagnosed. In 19.6% of treated children, hypothyroidism was diagnosed within 5 years after MIBG therapy.ConclusionMIBG therapy can be incorporated into the therapeutic strategy of relapsed or resistant neuroblastoma patients as preconditioning with HDCT rather than stand-alone therapy. Follow-up is required due to the incidence of thyroid failure and risk of second cancers.
Highlights
Neuroblastoma, which is derived from sympathoadrenal progenitor cells within the neural crest, is the most common extracranial tumor in children and shows hallmarks of neural tissue, such as the production of catecholamines and the expression of neurotrophin receptors [1]
The idea of megatherapy is based on the administration of high-dose chemotherapy (HDCT) aimed at eradicating the primary malignancy, and bone marrow damage manifesting as myeloablation or myelosuppression must be subsequently managed by hematopoietic stem cell transplantation from autologous sources or allogeneic or haploidentical donors
MIBG therapy was never curative (5-year overall survival (OS) and 5-year event-free survival (EFS) 0%), except in patients further treated with HDCT with stem cell rescue irrespective of their donor type (p
Summary
Neuroblastoma, which is derived from sympathoadrenal progenitor cells within the neural crest, is the most common extracranial tumor in children and shows hallmarks of neural tissue, such as the production of catecholamines and the expression of neurotrophin receptors [1]. Because high-risk neuroblastoma patients benefit from consolidation megatherapy, many attempts have been made to improve the efficacy of this treatment. The results of chemotherapy and HDCT in neuroblastoma still leave a room for improvement that can be achieved by exploiting biological characteristics of malignant cells. The role of MIBG therapy in neuroblastoma has been confirmed, but its place in the therapeutic strategies remains unclear, and different approaches have been studied over the years. This analysis presents the results of children with neuroblastoma undergoing MIBG therapy in Poland in 2006-2019. Neuroblastoma is the most common pediatric extracranial tumor with varied prognoses, but the survival of treated refractory or relapsing patients remains poor
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