Article
Oncology
Chad R. Schultz, Matthew A. Swanson, Thomas C. Dowling, Andre S. Bachmann
Summary: The study examined the use of DFMO/probenecid in NB patient-derived xenografts in mice and found that probenecid, an OAT 1/3 inhibitor, reduced DFMO renal clearance and significantly increased its antitumor activity. The combination of probenecid as an adjuvant to DFMO therapy may decrease overall dose and improve drug efficacy in vivo.
CANCER CHEMOTHERAPY AND PHARMACOLOGY
(2021)
Article
Oncology
Jacqueline M. Kraveka, Elizabeth C. Lewis, Genevieve Bergendahl, William Ferguson, Javier Oesterheld, Elizabeth Kim, Abhinav B. Nagulapally, Karl J. Dykema, Valerie Brown, William D. Roberts, Deanna Mitchell, Don Eslin, Derek Hanson, Michael S. Isakoff, Randal K. Wada, Virginia L. Harrod, Jawhar Rawwas, Gina Hanna, William P. D. Hendricks, Sara A. Byron, Matija Snuderl, Jonathan Serrano, Jeffrey M. Trent, Giselle L. Saulnier Sholler
Summary: This study demonstrates the safety and feasibility of adding targeted agents to standard induction therapy and adding DFMO to immunotherapy for HRNB.
Article
Oncology
Laura Privitera, Dale J. Waterhouse, Alessandra Preziosi, Irene Paraboschi, Olumide Ogunlade, Chiara Da Pieve, Marta Barisa, Olumide Ogunbiyi, Gregory Weitsman, J. Ciaran Hutchinson, Kate Cross, Lorenzo Biassoni, Danail Stoyanov, Neil Sebire, Paul Beard, Paolo De Coppi, Gabriela Kramer-Marek, John Anderson, Stefano Giuliani
Summary: Fluorescence-guided surgery is important for pediatric tumor management, and shortwave infrared imaging (SWIR) offers advantages over conventional NIR-I imaging. In this study, NIR-I dyes (IRDye800CW and IR12) were conjugated and used for neuroblastoma surgery. The NIR-I/SWIR device showed high tumor-to-background ratio (TBR) for both dyes and enabled high-contrast delineation of tumor margins. This work demonstrates the potential of SWIR imaging in clinical applications.
Article
Oncology
Sara A. Mansfield, M. Beth McCarville, John T. Lucas, Matthew J. Krasin, Sara M. Federico, Victor M. Santana, Wayne L. Furman, Andrew M. Davidoff
Summary: The study showed that neoadjuvant chemotherapy can reduce the number of IDRFs in patients with high-risk neuroblastoma, and the number of IDRFs present after chemotherapy is positively correlated with the extent of tumor resection.
ANNALS OF SURGICAL ONCOLOGY
(2022)
Article
Oncology
Lijuan Feng, Ying Kan, Wei Wang, Chao Wang, Hui Zhang, Peng Xie, Jigang Yang
Summary: The purpose of this study was to develop and validate a nomogram for predicting survival in intermediate- and high-risk neuroblastoma patients, and to compare its accuracy with the Children's Oncology Group (COG) risk stratification. The nomogram was constructed based on Cox regression analysis to determine the factors associated with event-free survival (EFS) and overall survival (OS) in neuroblastoma. The nomogram showed good predictive performance and outperformed the COG risk stratification.
JOURNAL OF CANCER RESEARCH AND CLINICAL ONCOLOGY
(2023)
Article
Oncology
Ami Desai, Mark A. Applebaum, Theodore G. Karrison, Akosua Oppong, Cindy Yuan, Katherine R. Berg, Kyle MacQuarrie, Elizabeth Sokol, Anurekha G. Hall, Navin Pinto, Ian Wolfe, Rajen Mody, Suzanne Shusterman, Valeria Smith, Jennifer H. Foster, Michele Nassin, James L. LaBelle, Rochelle Bagatell, Susan L. Cohn
Summary: This multicenter retrospective study investigated the efficacy of different post-induction therapies in high-risk neuroblastoma patients. The results showed that there was no significant difference in survival rates before and after autologous stem cell transplantation (ASCT) for patients who received post-induction therapy. However, patients who did not receive post-induction therapy had lower survival rates.
Article
Pediatrics
Quan Sun, Yanmin Chen, Qianya Jin, Xiaojun Yuan
Summary: This study retrospectively analyzed data from intermediate and high-risk neuroblastoma patients and identified age at diagnosis, tumor localization, MYCN state, histologic subtype, and tumor capsule as independent risk factors for recurrence. A nomogram model was established to accurately predict the recurrence of neuroblastoma.
EUROPEAN JOURNAL OF PEDIATRICS
(2022)
Article
Genetics & Heredity
Gunes Gundem, Max F. Levine, Stephen S. Roberts, Irene Y. Cheung, Juan S. Medina-Martinez, Yi Feng, Juan E. Arango-Ossa, Loic Chadoutaud, Mathieu Rita, Georgios Asimomitis, Joe Zhou, Daoqi You, Nancy Bouvier, Barbara Spitzer, David B. Solit, Filemon Dela Cruz, Michael P. LaQuaglia, Brian H. Kushner, Shakeel Modak, Neerav Shukla, Christine A. Iacobuzio-Donahue, Andrew L. Kung, Nai-Kong V. Cheung, Elli Papaemmanuil
Summary: Patients with high-risk neuroblastoma often experience widely metastatic disease and relapse despite intensive therapy. Understanding the genetic and clonal dynamics of metastatic spread and disease progression has been limited. In this study, genomic profiling of sequential and spatially separated samples from 283 patients revealed subtype-specific genetic evolutionary trajectories and the clonal dynamics of metastases. Convergent evolution of mutations targeting shared pathways emerged as a predominant feature of disease progression.
Review
Oncology
Jaume Mora
Summary: This article reviews the history of high-dose chemotherapy in the field of oncology and finds that it has not shown significant benefits in the treatment of solid tumors in adults, except for high-risk neuroblastoma. However, new immunotherapy approaches have demonstrated significant survival benefits with lesser side effects.
Article
Radiology, Nuclear Medicine & Medical Imaging
Lijuan Feng, Shuxin Zhang, Chaoran Wang, Siqi Li, Ying Kan, Chao Wang, Hui Zhang, Wei Wang, Jigang Yang
Summary: The study constructed and validated a combined model based on axial skeleton radiomics of F-18-FDG PET/CT, which showed more accurate predictive performance for event-free survival in high-risk pediatric neuroblastoma patients, helping to design individualized treatment strategies and regular follow-ups.
ACADEMIC RADIOLOGY
(2023)
Article
Medicine, General & Internal
Francesca Del Bufalo, Biagio De Angelis, Ignazio Caruana, Giada Del Baldo, Maria A. De Ioris, Annalisa Serra, Angela Mastronuzzi, Maria G. Cefalo, Daria Pagliara, Matteo Amicucci, Giuseppina Li Pira, Giovanna Leone, Valentina Bertaina, Matilde Sinibaldi, Stefano Di Cecca, Marika Guercio, Zeinab Abbaszadeh, Laura Iaffaldano, Monica Gunetti, Stefano Iacovelli, Rossana Bugianesi, Stefania Macchia, Mattia Algeri, Pietro Merli, Federica Galaverna, Rachid Abbas, Maria C. Garganese, Maria F. Villani, Giovanna S. Colafati, Federico Bonetti, Marco Rabusin, Katia Perruccio, Veronica Folsi, Concetta Quintarelli, Franco Locatelli
Summary: Immunotherapy with GD2-CAR T cells expressing the inducible caspase 9 suicide gene is a feasible and safe treatment option for high-risk neuroblastoma patients, with a sustained antitumor effect and a significant improvement in overall survival and event-free survival.
NEW ENGLAND JOURNAL OF MEDICINE
(2023)
Letter
Medicine, General & Internal
Concetta Quintarelli, Francesca del Bufalo, Franco Locatelli
Summary: Del Bufalo et al. describe a treatment using GD2-CART01 T cells that target GD2 and express a suicide gene for patients with relapsed or refractory pediatric neuroblastoma. Although this treatment suppresses tumor growth, T cell reexpansion and tumor GD2 expression were not observed in relapsed patients.
NEW ENGLAND JOURNAL OF MEDICINE
(2023)
Article
Medicine, General & Internal
Aleksandra Wieczorek, Urszula Zebrowska, Marek Ussowicz, Agnieszka Sokol, Marzena Stypinska, Bozenna Dembowska-Baginska, Katarzyna Pawinska-Wasikowska, Walentyna Balwierz, Giuseppina Barutello
Summary: A retrospective study conducted in Poland demonstrated the feasibility and benefits of using Dinutuximab beta for maintenance treatment in patients with high-risk neuroblastoma. The overall survival and progression-free survival rates were high, and the treatment was generally well tolerated.
JOURNAL OF CLINICAL MEDICINE
(2023)
Article
Pharmacology & Pharmacy
Zhichao Liu, Xi Chen, Ruth Roberts, Ruili Huang, Mike Mikailov, Weida Tong
Summary: This study investigated the translational ability of gene fusions for patient stratification and treatment development for high-risk neuroblastoma patients. Gene fusion profiles could be utilized to redefine patient subgroups in high-risk NB, leading to improved survival curves. The study identified potential repositioning candidates for precision medicine-based drug repositioning implementation.
FRONTIERS IN PHARMACOLOGY
(2021)
Article
Oncology
Xiaofei Sun, Zijun Zhen, Ying Guo, Yuanhong Gao, Juan Wang, Yu Zhang, Jia Zhu, Suying Lu, Feifei Sun, Junting Huang, Ruiqing Cai, Yizhuo Zhang, Juncheng Liu, Zizheng Xiao, Sihui Zeng, Zhuowei Liu
Summary: The study found that oral metronomic MT can improve survival in high-risk neuroblastoma patients, especially those unable to undergo ASCT or anti-GD2 antibody therapy. The low toxicity of this treatment is a significant advantage.