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High-Risk Neuroblastoma and Rhabdoid Tumors: New Clinical Insights and Challenges (Second Edition)

A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Oncology".

Deadline for manuscript submissions: 25 May 2026 | Viewed by 200

Special Issue Editors


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Guest Editor
1. Senior Professor, Department of Hematology/Oncology, Children’s Hospital, University Hospital Tübingen, 72076 Tübingen, Germany
2. George and Jennifer Yeo Endowed Chair in Pediatric Oncology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Singapore
Interests: stem cell transplantation; immunotherapy
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Guest Editor
Director, Department of Pediatric Oncology/Hematology, Children’s Hospital, University Hospital Tübingen, 72076 Tübingen, Germany
Interests: pediatric oncology; neuroblastoma
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Neuroblastoma is the most common extracranial solid tumor in young children and typically presents at an average age of 1 to 2 years. Occasionally, newborns can present with metastatic neuroblastoma, and adolescents or younger adults can also be affected. In newborns, even disseminated neuroblastoma can spontaneously regress without any therapy, whereas metastasized neuroblastoma in toddlers and older patients is highly aggressive and associated with a poor prognosis. Approximately half of the toddlers and older children with a neuroblastoma present with a local tumor (low risk) and a good prognosis and the other half with a disseminated tumor (high risk) and a poor prognosis. These features make neuroblastoma a tumor with one of the highest spontaneous regression rates, on the one hand. and one of the most aggressive tumors, on the other, due to its tendency to recur after initial treatment. The biology behind these features is slowly beginning to be understood, but there are still major challenges, especially in the treatment of high-risk patients. Current therapies comprise intensive chemotherapy, radiation, autologous or even allogeneic stem cell transplantation, targeted therapies, tumor vaccination approaches, monoclonal antibodies, chimeric antigen receptor (CAR) T-cells, and combinations of these methods.

Carrying on from the success of the first edition of the Special Issue “High-Risk Neuroblastoma: New Clinical Insights and Challenges”, this second edition welcomes authors to submit original papers or reviews on current and future new diagnostic and treatment strategies which will hopefully improve the currently poor prognosis of children with high-risk neuroblastoma.

In this second edition, we also invite original research papers regarding new treatment strategies for rhabdoid tumors. Despite the discovery of some underlying mechanisms of tumor development, such as mutations in the SMARCB1 gene resulting in the inactivation of the tumor-suppressive SWI/SNF protein complex, the outcome for patients is still poor despite aggressive standard therapy including surgery, chemotherapy, and irradiation. A better understanding of the biology of these tumors might lead to more effective therapies including targeted therapy and/or immunotherapy.

Prof. Dr. Rupert Handgretinger
Prof. Dr. Johannes Hubertus Schulte
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 250 words) can be sent to the Editorial Office for assessment.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Journal of Clinical Medicine is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2600 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • neuroblastoma
  • rhabdoid tumor
  • autologous stem cell transplantation
  • allogeneic stem cell transplantation immunotherapy
  • anti-GD2 antibody
  • targeted therapy
  • CAR T-cells

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Published Papers (1 paper)

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Review

18 pages, 439 KB  
Review
Neuroblastoma in Childhood: Biological Insights, Risk Stratification, and Advances in Multimodal Therapy
by Amina De Bona, Martina Barbieri, Nicole Rinaldi and Susanna Esposito
J. Clin. Med. 2026, 15(3), 1101; https://doi.org/10.3390/jcm15031101 - 30 Jan 2026
Abstract
Neuroblastoma is the most common extracranial solid tumor of childhood and remains a leading cause of cancer-related mortality in pediatric patients. Characterized by marked clinical and biological heterogeneity, the disease ranges from spontaneously regressing tumors in infants to highly aggressive, treatment-resistant malignancies in [...] Read more.
Neuroblastoma is the most common extracranial solid tumor of childhood and remains a leading cause of cancer-related mortality in pediatric patients. Characterized by marked clinical and biological heterogeneity, the disease ranges from spontaneously regressing tumors in infants to highly aggressive, treatment-resistant malignancies in older children. Advances in molecular biology and genomics have significantly improved understanding of neuroblastoma pathogenesis, revealing the critical role of genetic and epigenetic alterations—such as MYCN amplification, ALK mutations, and chromosomal aberrations—in disease behavior and prognosis. Contemporary risk stratification systems now integrate clinical, biological, and molecular features to guide therapy more precisely. Management strategies have evolved toward risk-adapted, multimodal approaches. Low- and intermediate-risk patients often achieve excellent outcomes with surgery alone or limited chemotherapy, whereas high-risk neuroblastoma requires intensive multimodal treatment including induction chemotherapy, surgical resection, high-dose chemotherapy with autologous stem cell rescue, radiotherapy, and maintenance therapy. The incorporation of immunotherapeutic approaches, particularly anti-GD2 monoclonal antibodies, has significantly improved survival in high-risk disease. Emerging therapies such as targeted agents, radiopharmaceuticals, and cellular immunotherapies are further expanding the therapeutic landscape. Despite these advances, high-risk and relapsed neuroblastoma remain associated with substantial morbidity and mortality. Ongoing challenges include treatment resistance, long-term toxicity, and disparities in access to advanced therapies. Continued progress will depend on integrating molecular profiling into clinical decision-making, refining risk-adapted treatment strategies, and expanding international collaborative research efforts. This narrative review summarizes current knowledge on neuroblastoma epidemiology, biology, staging, and treatment, highlighting recent advances and future directions aimed at improving outcomes for affected children. Full article
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