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Journal ArticleDOI

Children's Oncology Group's 2013 blueprint for research: Neuroblastoma

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TLDR
The incorporation of targeted radionuclide therapy prior to myeloablative chemotherapy into high‐risk treatment and druggable target(s) including mTOR inhibition and GD2‐directed therapy in combination with chemotherapy for patients with recurrent neuroblastoma, and ALK inhibition for those with ALK‐aberrant tumors are studied.
Abstract
Estimated 5-year survival rates for patients with non-high-risk and high-risk neuroblastoma are 90% and 50%, respectively. Recent clinical trials have shown excellent outcomes with reduced therapy for non-high-risk disease. For patients with high-risk neuroblastoma treated with chemoradiotherapy, surgery, and stem cell transplantation, the addition of anti-disialoganglioside (GD2) immunotherapy plus cytokines improves survival. Upcoming trials will study the incorporation of targeted radionuclide therapy prior to myeloablative chemotherapy into high-risk treatment. Phase 2 trials will investigate druggable target(s) including mTOR inhibition and GD2-directed therapy in combination with chemotherapy for patients with recurrent neuroblastoma, and ALK inhibition for those with ALK-aberrant tumors.

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Citations
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Journal ArticleDOI

Neuroblastoma

TL;DR: Neuroblastoma is a type of cancer that most often affects children and can spread to other parts of the body such as the bones, liver, or skin.
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Relapsed neuroblastomas show frequent RAS-MAPK pathway mutations

TL;DR: It is shown that RAS-MAPK pathway mutations may function as a biomarker for new therapeutic approaches to refractory disease and provide a rationale for genetic characterization of relapse neuroblastomas.
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Neuroblastoma: Paradigm for Precision Medicine

TL;DR: Current clinical trials have continued to reduce therapy for patients with non-high-risk NB, including the most favorable subsets who are often followed with observation approaches, and high-risk patients are treated aggressively with chemotherapy, radiation, surgery, and myeloablative and immunotherapies.
Journal ArticleDOI

Neuroblastoma: Molecular Pathogenesis and Therapy

TL;DR: Systematic classification of patients coupled with therapeutic advances point to a future of improved clinical outcomes for this biologically distinct and highly aggressive pediatric malignancy.
Journal ArticleDOI

Neuroblastoma: Clinical and Biological Approach to Risk Stratification and Treatment

TL;DR: The goal has been to decrease therapy for low-risk patients to avoid long-term complications while augmenting and targeting therapies for high- risk patients to improve overall survival.
References
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Journal ArticleDOI

Amplification of N-myc in untreated human neuroblastomas correlates with advanced disease stage

TL;DR: N-myc amplification is highly correlated with advanced stages of disease (P less than 0.001) and with the ability to grow in vitro as an established cell line, both of which are associated with a poor prognosis.
Journal ArticleDOI

Revisions of the international criteria for neuroblastoma diagnosis, staging, and response to treatment.

TL;DR: The International Neuroblastoma Staging System (INSS) as mentioned in this paper was proposed to establish an internationally accepted staging system for neuroblastoma, as well as consistent criteria for confirming the diagnosis and determining response to therapy.
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Association of multiple copies of the N-myc oncogene with rapid progression of neuroblastomas.

TL;DR: The results suggest that genomic amplification of N-myc may have a key role in determining the aggressiveness of neuroblastomas.
Journal ArticleDOI

Recent advances in neuroblastoma.

TL;DR: The author discusses recent advances in the understanding of neuroblastoma, an embryonal cancer of the autonomic nervous system, which has one of the highest rates of spontaneous and complete regression.
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