Recent Advances in Diagnosis and Treatment of Neuroblastoma

A special issue of Children (ISSN 2227-9067). This special issue belongs to the section "Oncology and Hematology".

Deadline for manuscript submissions: closed (1 July 2018) | Viewed by 57627

Special Issue Editor


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Guest Editor
1. Department of Pediatrics, Division of Hematology/Oncology, University of Washington, Seattle, WA, USA
2. Cancer & Blood Disorders Center, Seattle Children's Hospital, Seattle, WA, USA
Interests: pediatric solid tumor; neuroblastoma; cancer immunotherapy;pediatric pharmacology; sarcoma

Special Issue Information

Dear Colleagues,

Neuroblastoma is a tumor marked by wide biologic variability and is disproportionately represented in childhood cancer deaths. This Special Issue will highlight recent advances in our understanding of the biologic basis of various neuroblastoma subtypes, recent improvements in the standard of care treatment of high-risk neuroblastoma and spotlight exciting new therapies for patients with relasped and refractory neuroblastoma.

Dr. Navin Pinto
Guest Editor

Manuscript Submission Information

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Keywords

  • Neuroblastoma
  • cancer immunotherapy
  • MYCN
  • ALK
  • alternative lengthening of telomeres (ALT)
  • circulating tumor cells
  • cellular immunotherapy
  • MIBG therapy

Published Papers (7 papers)

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Research

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12 pages, 205 KiB  
Article
Non-High-Risk Neuroblastoma: Classification and Achievements in Therapy
by Holly J. Meany
Children 2019, 6(1), 5; https://doi.org/10.3390/children6010005 - 08 Jan 2019
Cited by 18 | Viewed by 5500
Abstract
Neuroblastoma, a tumor of the sympathetic nervous system, is the most common extra-cranial neoplasm of childhood. Variables with prognostic significance in patients with neuroblastoma, including age at diagnosis, disease stage, tumor histology, MYCN gene amplification, tumor cell ploidy, and the presence of segmental [...] Read more.
Neuroblastoma, a tumor of the sympathetic nervous system, is the most common extra-cranial neoplasm of childhood. Variables with prognostic significance in patients with neuroblastoma, including age at diagnosis, disease stage, tumor histology, MYCN gene amplification, tumor cell ploidy, and the presence of segmental chromosomal aberrations are utilized to classify patients based on risk of disease recurrence. Patients with non-high-risk neuroblastoma, low- and intermediate-risk categories, represent nearly half of all newly diagnosed cases. This group has an excellent event-free and overall survival with current therapy. Over time, the objective in treatment of non-high-risk neuroblastoma has been reduction of therapy intensity to minimize short- and long-term adverse events all the while maintaining excellent outcomes. Full article
(This article belongs to the Special Issue Recent Advances in Diagnosis and Treatment of Neuroblastoma)

Review

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12 pages, 1203 KiB  
Review
The Evolution of Risk Classification for Neuroblastoma
by Elizabeth Sokol and Ami V. Desai
Children 2019, 6(2), 27; https://doi.org/10.3390/children6020027 - 11 Feb 2019
Cited by 75 | Viewed by 10785
Abstract
Neuroblastoma is a tumor with great clinical heterogeneity. Patients in North America are risk-stratified using a number of features including age at diagnosis, disease stage, tumor histology, MYCN status (amplified versus nonamplified), and tumor cell ploidy. In this paper, we review the evidence [...] Read more.
Neuroblastoma is a tumor with great clinical heterogeneity. Patients in North America are risk-stratified using a number of features including age at diagnosis, disease stage, tumor histology, MYCN status (amplified versus nonamplified), and tumor cell ploidy. In this paper, we review the evidence for utilizing these features in the risk classification of neuroblastic tumors. Additionally, we review the clinical and biologic criteria used by various cooperative groups to define low, intermediate, and high-risk disease populations in clinical trials, highlighting the differences in risk classification internationally. Finally, we discuss the development of the International Neuroblastoma Risk Group classification system, designed to begin worldwide standardization of neuroblastoma pretreatment risk classification and allow comparison of clinical trials conducted through different cooperative groups. Full article
(This article belongs to the Special Issue Recent Advances in Diagnosis and Treatment of Neuroblastoma)
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27 pages, 292 KiB  
Review
Novel Therapies for Relapsed and Refractory Neuroblastoma
by Peter E. Zage
Children 2018, 5(11), 148; https://doi.org/10.3390/children5110148 - 31 Oct 2018
Cited by 41 | Viewed by 8079
Abstract
While recent increases in our understanding of the biology of neuroblastoma have allowed for more precise risk stratification and improved outcomes for many patients, children with high-risk neuroblastoma continue to suffer from frequent disease relapse, and despite recent advances in our understanding of [...] Read more.
While recent increases in our understanding of the biology of neuroblastoma have allowed for more precise risk stratification and improved outcomes for many patients, children with high-risk neuroblastoma continue to suffer from frequent disease relapse, and despite recent advances in our understanding of neuroblastoma pathogenesis, the outcomes for children with relapsed neuroblastoma remain poor. These children with relapsed neuroblastoma, therefore, continue to need novel treatment strategies based on a better understanding of neuroblastoma biology to improve outcomes. The discovery of new tumor targets and the development of novel antibody- and cell-mediated immunotherapy agents have led to a large number of clinical trials for children with relapsed neuroblastoma, and additional clinical trials using molecular and genetic tumor profiling to target tumor-specific aberrations are ongoing. Combinations of these new therapeutic modalities with current treatment regimens will likely be needed to improve the outcomes of children with relapsed and refractory neuroblastoma. Full article
(This article belongs to the Special Issue Recent Advances in Diagnosis and Treatment of Neuroblastoma)
16 pages, 229 KiB  
Review
Molecularly Targeted Therapy for Neuroblastoma
by Emily G. Greengard
Children 2018, 5(10), 142; https://doi.org/10.3390/children5100142 - 15 Oct 2018
Cited by 34 | Viewed by 5068
Abstract
Neuroblastoma is the most common extra-cranial solid tumor encountered in childhood and accounts for 15% of pediatric cancer-related deaths. Although there has been significant improvement in the outcomes for patients with high-risk disease, the therapy needed to achieve a cure is quite toxic [...] Read more.
Neuroblastoma is the most common extra-cranial solid tumor encountered in childhood and accounts for 15% of pediatric cancer-related deaths. Although there has been significant improvement in the outcomes for patients with high-risk disease, the therapy needed to achieve a cure is quite toxic and for those that do experience a disease recurrence, the prognosis is very dismal. Given this, there is a tremendous need for novel therapies for children with high-risk neuroblastoma and the molecular discoveries over recent years provide hope for developing new, less toxic, and potentially more efficacious treatments. Here I discuss many of the molecular aberrations identified thus far in neuroblastoma, as well as the agents in development to target these changes. The progress made in both the preclinical arena and in early phase drug development provide much promise for the future of precision medicine in neuroblastoma. Full article
(This article belongs to the Special Issue Recent Advances in Diagnosis and Treatment of Neuroblastoma)
18 pages, 592 KiB  
Review
Genetic Predisposition to Neuroblastoma
by Erin K. Barr and Mark A. Applebaum
Children 2018, 5(9), 119; https://doi.org/10.3390/children5090119 - 31 Aug 2018
Cited by 48 | Viewed by 6240
Abstract
Neuroblastoma is the most common solid tumor in children under the age of one. It displays remarkable phenotypic heterogeneity, resulting in differences in outcomes that correlate with clinical and biologic features at diagnosis. While neuroblastoma accounts for approximately 5% of all cancer diagnoses [...] Read more.
Neuroblastoma is the most common solid tumor in children under the age of one. It displays remarkable phenotypic heterogeneity, resulting in differences in outcomes that correlate with clinical and biologic features at diagnosis. While neuroblastoma accounts for approximately 5% of all cancer diagnoses in pediatrics, it disproportionately results in about 9% of all childhood deaths. Research advances over the decades have led to an improved understanding of neuroblastoma biology. However, the initiating events that lead to the development of neuroblastoma remain to be fully elucidated. It has only been recently that advances in genetics and genomics have allowed researchers to unravel the predisposing factors enabling the development of neuroblastoma and fully appreciate the interplay between the genetics of tumor and host. In this review, we outline the current understanding of familial neuroblastoma and highlight germline variations that predispose children to sporadic disease. We also discuss promising future directions in neuroblastoma genomic research and potential clinical applications for these advances. Full article
(This article belongs to the Special Issue Recent Advances in Diagnosis and Treatment of Neuroblastoma)
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7 pages, 763 KiB  
Review
High-Risk Neuroblastoma Treatment Review
by Valeria Smith and Jennifer Foster
Children 2018, 5(9), 114; https://doi.org/10.3390/children5090114 - 28 Aug 2018
Cited by 147 | Viewed by 15558
Abstract
Neuroblastoma is the most common extracranial solid tumor in children. One subset, high-risk neuroblastoma, is very difficult to treat and requires multi-modal therapy. Intensification of therapy has vastly improved survival rates, and research is focused on novel treatments to further improve survival rates. [...] Read more.
Neuroblastoma is the most common extracranial solid tumor in children. One subset, high-risk neuroblastoma, is very difficult to treat and requires multi-modal therapy. Intensification of therapy has vastly improved survival rates, and research is focused on novel treatments to further improve survival rates. The current treatment schema is divided into three stages—induction, consolidation, and maintenance. This review serves as an overview of the current treatment for high-risk neuroblastoma and a glimpse at current research for future therapy. Full article
(This article belongs to the Special Issue Recent Advances in Diagnosis and Treatment of Neuroblastoma)
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15 pages, 786 KiB  
Review
Late Effects and Survivorship Issues in Patients with Neuroblastoma
by Danielle Novetsky Friedman and Tara O. Henderson
Children 2018, 5(8), 107; https://doi.org/10.3390/children5080107 - 06 Aug 2018
Cited by 34 | Viewed by 5262
Abstract
Over the past two decades, marked progress has been made in understanding the biology of neuroblastoma; this has led to refined risk stratification and treatment modifications with resultant increasing 5-year survival rates for children with neuroblastoma. Survivors, however, remain at risk for a [...] Read more.
Over the past two decades, marked progress has been made in understanding the biology of neuroblastoma; this has led to refined risk stratification and treatment modifications with resultant increasing 5-year survival rates for children with neuroblastoma. Survivors, however, remain at risk for a wide variety of potential treatment-related complications, or “late effects”, which may lead to excess morbidity and premature mortality in this cohort. This review summarizes the existing survivorship literature on long-term health outcomes for survivors of neuroblastoma, focusing specifically on potential injury to the endocrine, sensory, cardiovascular, pulmonary, and renal systems, as well as survivors’ treatment-related risk for subsequent neoplasms and impaired quality of life. Additional work is needed to assess the potential late effects of newer multimodality therapies with the aim of optimizing long-term medical and psychosocial outcomes for all survivors of neuroblastoma. Full article
(This article belongs to the Special Issue Recent Advances in Diagnosis and Treatment of Neuroblastoma)
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