Updates on Diagnosis and Treatment for Pediatric Solid Tumors

A special issue of Current Oncology (ISSN 1718-7729). This special issue belongs to the section "Childhood, Adolescent and Young Adult Oncology".

Deadline for manuscript submissions: closed (30 June 2025) | Viewed by 4937

Special Issue Editor


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Guest Editor
Department of Neurological Surgery, Indiana University School of Medicine, Indianapolis, IN 46202, USA
Interests: neurooncology; precision medicine; translational oncology

Special Issue Information

Dear Colleagues,

We are excited to invite you to contribute to our upcoming Special Issue in Current Oncology, titled “Updates on Diagnosis and Treatment for Pediatric Solid Tumors”. This Special Issue aims to bridge critical gaps in the current landscape of pediatric solid tumor research and treatment. Given your expertise, your contribution would be invaluable in enhancing our collective understanding and advancing the field.

Despite significant strides being made in pediatric oncology, challenges persist, particularly in developing precise diagnostic tools and treatments tailored to children's unique physiology. The complexity of tumor heterogeneity and its role in differential treatment responses adds another layer of intricacy. Furthermore, orphan diseases and rare tumors, often lacking standard care protocols, meaning that they present unique challenges. The long-term effects and survivorship issues are under-explored, and the psychosocial impact on patients and families often receives insufficient attention. Additionally, disparities in access to advanced treatments and the economic burden on families and healthcare systems underscore the urgent need for innovative research and comprehensive studies that address these multifaceted challenges.

To address these critical gaps, for this Special Issue, we welcome papers which focus on a range of topics, each providing a unique perspective and potential solutions:

  1. Emerging Diagnostic and Therapeutic Technologies: Advances in less invasive diagnostic methods and targeted treatments.
  2. Personalized Medicine and Pharmacogenomics: Tailoring treatments based on genetic profiles and individual responses to therapy.
  3. Targeted Therapy and Translational Medicine: Development of preclinical models for testing new therapies and translating research findings into clinical practice.
  4. Long-term Effects and Survivorship: Studies focusing on the after-effects of cancer treatment and strategies for long-term care.
  5. Psychosocial Impact: Research into the psychological, emotional, and social challenges faced by pediatric patients and their families.
  6. Economic and Access Disparities: Investigations into disparities in healthcare access and the economic impact of treatments.
  7. Multidisciplinary Care Approaches: Evaluations of the effectiveness of coordinated and comprehensive care models.
  8. Early Detection and Screening: Development of screening methods and early detection strategies for pediatric solid tumors.
  9. Clinical Trials and Emerging Treatments: Reports on new therapies and their transition from clinical trials to clinical practice.
  10. Epidemiology and Risk Factors: Insights into changing trends and new risk factors in pediatric solid tumors.
  11. Healthcare Policies in Pediatric Oncology: Analysis of policies affecting treatment access and healthcare delivery for pediatric cancer patients.

We encourage submissions of original research, reviews, case studies, highly novel case reports, retrospective studies, or perspectives that align with these themes. Your insights could help significantly shape future directions and improve outcomes for pediatric solid tumor patients.

Dr. Nur P. Damayanti
Guest Editor

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Keywords

  • pediatric tumors
  • diagnostic innovation
  • targeted therapy
  • personalized oncology
  • pediatric pharmacogenomics
  • translational oncology
  • pediatric rare tumor
  • cancer survivorship
  • early detection
  • pediatric clinical trial

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Published Papers (3 papers)

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Research

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10 pages, 483 KiB  
Article
Germline TP53 p.R337H and XAF1 p.E134* Variants: Prevalence in Paraguay and Comparison with Rates in Brazilian State of Paraná and Previous Findings at the Paraguayan–Brazilian Border
by Edith Falcon-de Legal, Marta Ascurra, Rosa Vega-Paredes, Elis Sade, Magna Monteiro, Mariana Paraízo, Magali Colman, Angeles Gutierrez Florentin, Cesar Ojeda, Horacio Legal-Ayala and Andreas Ries
Curr. Oncol. 2025, 32(6), 333; https://doi.org/10.3390/curroncol32060333 - 6 Jun 2025
Viewed by 802
Abstract
Adrenal cortex carcinoma (ACC) in children is a rare tumor that is probably of multifactorial origin and is mainly associated with genetic and environmental alterations. In the south and part of the southeast of Brazil, as well as in the Paraguayan region bordering [...] Read more.
Adrenal cortex carcinoma (ACC) in children is a rare tumor that is probably of multifactorial origin and is mainly associated with genetic and environmental alterations. In the south and part of the southeast of Brazil, as well as in the Paraguayan region bordering the Brazilian State of Paraná, ACC prevalence is higher than in any other country, which is associated with the high prevalence of the TP53 p.R337H variant in Paraná (0.30%), Santa Catarina (0.249%), cities around Campinas-SP (0.21%), and the Paraguayan cities on the border with Paraná (0.05%). Recent research suggests that the co-segregation of XAF1-E134* and TP53-R337H mutations leads to a more aggressive cancer phenotype than TP53-R337H alone. Breast cancer may be mildly influenced by co-segregation with XAF1 p.E134*, and this variant can also confer risk for sarcoma. Objectives: The objectives of this study were to (1) estimate the prevalence of the germline TP53 p.R337H and XAF1 p.E134* variants in Paraguay (excluding cities on the border with Paraná State, Brazil) and (2) estimate whether the ethnic origin of TP53 p.R337H carriers in Paraguay is similar to that of ethnic groups in Paraná (possible Portuguese/Spanish origin). Materials and methods: DNA tests for the identification of TP53 p.R337H were carried out from 2016 to 2019 at the Bio-Materials Laboratory of Facultad Politecnica, UNA, and at the Research Center in Biotechnology and Informatics (CEBIOTEC), Asunción, Paraguay. Polymerase chain reaction followed by restriction enzyme digestion (PCR-RFLP) was used to identify TP53 p.R337H, and real-time PCR (RT-PCR) was employed for XAF1 p.E134*. Peripheral blood samples from 40,000 Paraguayan newborns (NBs) were used for the TP53 p.R337H tests. The XAF1 p.E134* tests (RT-PCR) were performed on samples from 2000 Paraguayan newborns at the Pelé Pequeno Principe Research Institute, Curitiba, PR, Brazil. Results: The TP53 p.R337H variant was not found in any of the 14 Paraguayan departments investigated. A total of 12 of the 2000 Paraguayan NBs were positive for one XAF1 p.E134* allele. Conclusions: The hypothesis of Spanish immigrants carrying p.R337H to Paraguay was disproved. TP53 p.R337H neonatal testing in Paraguay is not recommended, except when there are families with Brazilian ancestry presenting cancer cases. Additional epidemiological studies are required to determine the likelihood of the identified prevalence of the XAF1 p.E134* allele (1/153) in NBs from Paraguay without TP53 p.R337H to present cancer risk. This study complements the first national initiative for the DNA screening of newborns aimed at mapping the TP53 p.R337H and XAF1 p.E134* variants in Paraguay (based on the regions of residence of the newborns). Full article
(This article belongs to the Special Issue Updates on Diagnosis and Treatment for Pediatric Solid Tumors)
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11 pages, 1172 KiB  
Article
Population Pharmacokinetics of Tamibarotene in Pediatric and Young Adult Patients with Recurrent or Refractory Solid Tumors
by Takuya Azechi, Yutaka Fukaya, Chika Nitani, Junichi Hara, Hiroshi Kawamoto, Tomoaki Taguchi, Kenichi Yoshimura, Akihiro Sato, Naoko Hattori, Toshikazu Ushijima and Toshimi Kimura
Curr. Oncol. 2024, 31(11), 7155-7164; https://doi.org/10.3390/curroncol31110527 - 14 Nov 2024
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Abstract
Tamibarotene is a synthetic retinoid that inhibits tumor cell proliferation and promotes differentiation. We previously reported on the safety and tolerability of tamibarotene in patients with recurrent or refractory solid tumors. Therefore, in this study, we aimed to evaluate the pharmacokinetic properties of [...] Read more.
Tamibarotene is a synthetic retinoid that inhibits tumor cell proliferation and promotes differentiation. We previously reported on the safety and tolerability of tamibarotene in patients with recurrent or refractory solid tumors. Therefore, in this study, we aimed to evaluate the pharmacokinetic properties of tamibarotene and construct a precise pharmacokinetic model. We also conducted a non-compartmental analysis and population pharmacokinetic (popPK) analysis based on the results of a phase I study. Targeted pediatric and young adult patients with recurrent or refractory solid tumors were administered tamibarotene at doses of 4, 6, 8, 10, and 12 g/m2/day. Serum tamibarotene concentrations were evaluated after administration, and a popPK model was constructed for tamibarotene using Phoenix NLME. During model construction, we considered the influence of various parameters (weight, height, body surface area, and age) as covariates. Notably, 22 participants were included in this study, and 109 samples were analyzed. A two-compartment model incorporating lag time was selected as the base model. In the final model, the body surface area was included as a covariate for apparent total body clearance, the central compartment volume of distribution, and the peripheral compartment volume of distribution. Visual prediction checks and bootstrap analysis confirmed the validity and predictive accuracy of the final model as satisfactory. Full article
(This article belongs to the Special Issue Updates on Diagnosis and Treatment for Pediatric Solid Tumors)
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Review

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15 pages, 1170 KiB  
Review
CyberKnife in Pediatric Oncology: A Narrative Review of Treatment Approaches and Outcomes
by Costanza M. Donati, Federica Medici, Arina A. Zamfir, Erika Galietta, Silvia Cammelli, Milly Buwenge, Riccardo Masetti, Arcangelo Prete, Lidia Strigari, Ludovica Forlani, Elisa D’Angelo and Alessio G. Morganti
Curr. Oncol. 2025, 32(2), 76; https://doi.org/10.3390/curroncol32020076 - 29 Jan 2025
Cited by 1 | Viewed by 1803
Abstract
Pediatric cancers, while rare, pose unique challenges due to the heightened sensitivity of developing tissues and the increased risk of long-term radiation-induced effects. Radiotherapy (RT) is a cornerstone in pediatric oncology, but its application is limited by concerns about toxicity, particularly secondary malignancies, [...] Read more.
Pediatric cancers, while rare, pose unique challenges due to the heightened sensitivity of developing tissues and the increased risk of long-term radiation-induced effects. Radiotherapy (RT) is a cornerstone in pediatric oncology, but its application is limited by concerns about toxicity, particularly secondary malignancies, growth abnormalities, and cognitive deficits. CyberKnife (CK), an advanced robotic radiosurgery system, has emerged as a promising alternative due to its precision, non-invasiveness, and ability to deliver hypofractionated, high-dose RT while sparing healthy tissues. This narrative review explores the existing evidence on CK application in pediatric patients, synthesizing data from case reports, small series, and larger cohort studies. All the studies analyzed reported cases of tumors located in the skull or in the head and neck region. Findings suggest CK’s potential for effective tumor control with favorable toxicity profiles, especially for complex or inoperable tumors. However, the evidence remains limited, with the majority of studies involving small sample sizes and short follow-up periods. Moreover, concerns about the “dose-bath” effect and limited long-term data on stochastic risks warrant cautious adoption. Compared to Linac-based RT and proton therapy, CK offers unique advantages in reducing session numbers and enhancing patient comfort, while its real-time tracking provides superior accuracy. Despite these advantages, CK is associated with significant limitations, including a higher potential for low-dose scatter (often referred to as the “dose-bath” effect), extended treatment times in some protocols, and high costs requiring specialized expertise for operation. Emerging modalities like π radiotherapy further underscore the need for comparative studies to identify the optimal technique for specific pediatric cases. Notably, proton therapy remains the benchmark for minimizing long-term toxicity, but its cost and availability limit its accessibility. This review emphasizes the need for balanced evaluations of CK and highlights the importance of planning prospective studies and long-term follow-ups to refine its role in pediatric oncology. A recent German initiative to establish a CK registry for pediatric CNS lesions holds significant promise for advancing evidence-based applications and optimizing treatment strategies in this vulnerable population. Full article
(This article belongs to the Special Issue Updates on Diagnosis and Treatment for Pediatric Solid Tumors)
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