Increasing treatment intensity has improved outcomes for children with neuroblastoma. We performed a pilot study in the Children's Oncology Group to assess the feasibility and toxicity of a tandem myeloablative regimen without TBI supported by autologous CD34-selected peripheral blood stem cells. Forty-one patients with high-risk neuroblastoma were enrolled; eight patients did not receive any myeloablative consolidation procedure and seven received only one. Two patients out of 41 (4.9%) experienced transplant-related mortality. CD34 selection was discontinued after subjects were enrolled due to serious viral illness. From the time of study enrollment, the overall 3-year EFS and OS were 44.8 ± 9.6% and 59.2 ± 9.2% (N=41). These results demonstrate that tandem transplantation in the cooperative group setting is feasible and support a randomized comparison of single vs tandem myeloablative consolidation with PBSC support for high-risk neuroblastoma.
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http://dx.doi.org/10.1038/bmt.2012.276 | DOI Listing |
Cancers (Basel)
January 2025
Department of Haematology and Oncology, University Children's Hospital, Eberhard Karls University Tuebingen, 72076 Tuebingen, Germany.
Background: The incorporation of anti-GD2 antibodies such as ch14.18/SP2/0 into the multimodal treatment of high-risk neuroblastoma (HR-NB) patients has improved their outcomes. As studies assessing the long-term outcomes, long-term sequelae, and health-related quality of life (HRQoL) of this treatment are limited, this retrospective analysis aimed to explore these.
View Article and Find Full Text PDFCancers (Basel)
December 2024
Princess Máxima Center for Pediatric Oncology, 3584 CS Utrecht, The Netherlands.
Background: The 5-year prognosis of non-high-risk neuroblastomas is generally good (>90%). However, a proportion of patients show progression and succumb to their disease. We aimed to identify molecular aberrations (not incorporated in the current risk stratification) associated with overall survival (OS) and/or event-free survival (EFS) in patients diagnosed with non-high-risk neuroblastoma.
View Article and Find Full Text PDFRev Esp Patol
January 2025
Departamento de Patología, Universidad de Valencia, Hospital Clínico Universitario de Valencia, CIBERONC (ISCIII Madrid), INCLIVA, Valencia, Spain. Electronic address:
High-risk neuroblastoma continues to show a very high mortality, with a 5-year survival rate of 50%. While MYCN amplification is the main genetic alteration associated with high-risk tumours, other molecular mechanisms, such as alterations in ATRX and TERT, remain poorly understood. ATRX and TERT biomarkers, which are associated with a more aggressive neuroblastoma pattern, should be considered for accurate prognostic stratification.
View Article and Find Full Text PDFClin Nucl Med
January 2025
From the Department of Nuclear Medicine, Beijing Friendship Hospital, Capital Medical University, Beijing, China.
A 7-year-old boy with high-risk neuroblastoma underwent 123I-MIBG SPECT/CT to evaluate the therapy response. The scan revealed abnormal 123I-MIBG uptake in the left basal ganglion, indicating the possibility of brain metastasis. Subsequent contrast-enhanced brain MRI, however, did not show any abnormal signal intensity in the left basal ganglion.
View Article and Find Full Text PDFJ Proteome Res
January 2025
Department of Pediatric Surgery, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan 450052, China.
Neuroblastoma (NB) remains associated with high mortality and low initial response rate, especially for high-risk patients, thus warranting exploration of molecular markers for precision risk classifiers. Through integrating multiomics profiling, we identified a range of hub genes involved in cell cycle and associated with dismal prognosis and malignant cells. Single-cell transcriptome sequencing revealed that a subset of malignant cells, subcluster 1, characterized by high proliferation and dedifferentiation, was strongly correlated with the hub gene signature and orchestrated an immunosuppressive tumor microenvironment (TME).
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