Treatment and survival of childhood neuroblastoma: Evidence from a population-based study in the United States

Coughlan, Diarmuid; Gianferante, Matthew; Lynch, Charles F.; Stevens, Jennifer; Harlan, Linda C.

doi:10.1080/08880018.2017.1373315

Cited by 79 publications

(76 citation statements)

References 19 publications

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“…Neuroblastoma (NB) is the most common extracranial solid tumor of pediatric malignancies and originates from sympathetic nervous system, accounting for approximately 10% of all pediatric tumors [1][2][3]. Although substantial progresses have been made in therapy of NB, including chemotherapy, radiotherapy, surgery, hematopoietic stem cell transfusion, and immunotherapy, the 5-year survival rate of patients with high-risk NB remains still less than 50% [4][5][6][7][8][9]. Besides age of onset, primary location, and metastasis, MYCN amplification is widely considered to correlate with risk stratification, relapse, progressive disease, and unfavorable survival rate in NB patients [5,[10][11][12][13][14][15][16][17].…”

Section: Introductionmentioning

confidence: 99%

Implementation of the plasma MYCN/NAGK ratio to detect MYCN amplification in patients with neuroblastoma

Wang²,

Zhao

et al. 2020

Molecular Oncology

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Detection of amplification of the MYCN gene is essential for determining optimal treatment and estimating prognosis of patients with neuroblastoma (NB). DNA FISH with neuroblastoma tissues or patient-derived bone marrow cells is the standard clinical practice for the detection of MYCN amplification. As tumor cells may often be unavailable, we developed a method to detect MYCN amplification in the plasma of patients with neuroblastoma. Taking single-copy NAGK DNA as reference, we used real-time quantitative PCR (qPCR) to determine the MYCN/NAGK ratio in the plasma of 115 patients diagnosed with NB. An increased MYCN/NAGK ratio in the plasma was consistent with MYCN amplification as assessed by DNA FISH. The AUC for a MYCN/NAGK ratio equal to 6.965 was 0.943, with 86% sensitivity and 100% specificity. Beyond the threshold of 6.965, the MYCN/NAGK ratio correlated with a heavier tumor burden. Event-free and overall survival of two years were significantly shortened in stage 4 patients with a MYCN/NAGK ratio higher than 6.965. Plasma MYCN/NAGK ratios increased in patients with progressive disease and relapse. Thus, we conclude that the determination of the plasma MYCN/ NAGK ratio by qPCR is a noninvasive and reproducible method to measure MYCN amplification in patients with NB.

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Section: Introductionmentioning

confidence: 99%

Implementation of the plasma MYCN/NAGK ratio to detect MYCN amplification in patients with neuroblastoma

Wang²,

Zhao

et al. 2020

Molecular Oncology

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“…The present study selected patients with low‐risk tumors based on the internationally adopted COG classification—MYCN nonamplified localized tumors and stage 4S tumors. Furthermore, we included patients with stage 3 neuroblastomas without MYCN amplification that were determined to belong to the intermediate‐risk group according to COG classifications because such cases were associated with good prognoses in a former study …”

Section: Introductionmentioning

confidence: 99%

Results of a prospective clinical trial JN‐L‐10 using image‐defined risk factors to inform surgical decisions for children with low‐risk neuroblastoma disease: A report from the Japan Children's Cancer Group Neuroblastoma Committee

Iehara

Yoneda

Yokota

et al. 2019

Pediatric Blood & Cancer

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Background:The present study sought to reduce the incidence of treatment complications of lowrisk neuroblastoma by using image-defined risk factors (IDRFs) to inform the timing of surgical resection.Procedures: Eligible patients included children (<18 years of age) with stage 1 or 2 disease, children (<365 days of age) with stage 3 disease, and infants with stage 4S disease. In IDRF-negative cases, treatment was completed with surgical resection alone. In IDRF-positive cases, the timing of surgery was determined based on the IDRFs after low-dose chemotherapy with 2-3 of the following four drugs: vincristine, cyclophosphamide, pirarubicin, and carboplatin. The outcome measures were overall survival, progression-free survival, and adverse events. This study was registered with the UMIN Clinical Trials Registry (number 000004355). Results:Of the 60 patients screened between 2010 and 2013, 58 eligible patients were enrolled; 32 were identified as IDRF negative at diagnosis while 26 were identified as IDRF positive and underwent induction chemotherapy. The 3-year overall and progression-free survival rates of the

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“…However, due to lack of tissue specificity, CPPs such as TAT (transactivating transcriptional activator) peptide, penetratin and polyarginine are not as efficient when aiming to deliver expensive or toxic drugs [1]. One group of diseases which are difficult to treat and may benefit from targeted delivery methods are neural cell disorders such as neuroblastoma [2][3][4] and neurosarcoma [5].…”

mentioning

confidence: 99%

Design, stability and efficacy of a new targeting peptide for nanoparticulate drug delivery to SH-SY5Y neuroblastoma cells

Huey

Rathbone

McCarron

et al. 2019

Journal of Drug Targeting

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In recent years, rabies virus-derived peptide (RDP) has shown promise as a specific neural cell targeting ligand, however stability of the peptide in human serum was unknown. Herein, we report the molecular modelling and design of an optimised peptide sequence based on interactions of RDP with the 7 subunit of the nicotinic acetylcholine receptor (nAChR). The new sequence, named DAS, designed around a 5-mer sequence which demonstrated optimal nAChR binding in silico, showed greatly improved stability for up to 8 hours in human serum in comparison to RDP, which degraded within 2 hours at 37 °C. In vitro analysis using SH-SY5Y neuroblastoma cells showed that DAS-conjugated nanoparticles containing the cytotoxic drug doxorubicin (DAS-Dox-NP) displayed significantly enhanced cytotoxicity compared with untargeted doxorubicin-loaded nanoparticles (Dox-NP). DAS-Dox-NP had no significant effect on non-neural cell types, confirming its neural-specific targeting properties. This is the first time that In this manuscript, we report the design and testing of an optimised peptide ligand, conjugated to a nanoparticulate delivery vehicle and specifically targeted to neural cells., has been reported. Future impact of an innovative targeting peptide ligand combining the ability to selectively identify the target and facilitate cellular internalisation could enable the successful treatment of many neural cell disorders.

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Treatment and survival of childhood neuroblastoma: Evidence from a population-based study in the United States

Abstract: Most neuroblastoma patients are registered on a risk-based open/active clinical trial. Variation in modality, systemic agents and sequence of treatment reflects the heterogeneity of therapy received by these patients.

Cited by 79 publications

References 19 publications

Implementation of the plasma MYCN/NAGK ratio to detect MYCN amplification in patients with neuroblastoma

Implementation of the plasma MYCN/NAGK ratio to detect MYCN amplification in patients with neuroblastoma

Results of a prospective clinical trial JN‐L‐10 using image‐defined risk factors to inform surgical decisions for children with low‐risk neuroblastoma disease: A report from the Japan Children's Cancer Group Neuroblastoma Committee

Design, stability and efficacy of a new targeting peptide for nanoparticulate drug delivery to SH-SY5Y neuroblastoma cells

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