Pediatric Solid Tumor: Precision Medicine and Long-Term Prognosis

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

Deadline for manuscript submissions: closed (20 February 2026) | Viewed by 1498

Special Issue Editors


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Guest Editor
Department of Radiology, Nemours Children’s Health, Wilmington, DE 19803, USA
Interests: tumor targeting; drug delivery; radiochemistry; drug quality control and nuclear medicine; molecular imaging; tissue engineering
Department of Radiology, Diagnostic and Research PET/MR Center, Nemours Children’s Health, Wilmington, DE 19803, USA
Interests: radiochemistry; neuroimaging; organic chemistry; nuclear medicine; molecular imaging
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Special Issue Information

Dear Colleagues,

Pediatric solid tumors represent a complex and heterogeneous set of cancers that demand highly tailored approaches to diagnosis, treatment, and long-term care. Precision medicine offers the potential for personalized therapies guided by molecular profiling, genomic alterations, and tumor biology; however, its adoption in pediatric oncology remains far from universal.

Key obstacles include inconsistent implementation across healthcare systems, a lack of robust evidence in varied patient populations, and persistent inequities in access. Predictive genomic risk models may underperform in underrepresented groups, while the routine use of multi-omics data is hampered by significant analytical and operational challenges. Overcoming these barriers is essential to achieving meaningful gains in survival, quality of life, and long-term health outcomes for all children.

This Special Issue welcomes original research, clinical studies, and reviews addressing the following:

  • Novel diagnostic tools and molecular targets;
  • Strategies for treatment optimization and overcoming resistance;
  • Methods for integrating multi-omics data into clinical decision-making;
  • Long-term monitoring and survivorship care;
  • Health equity, access, and implementation of science approaches;
  • Real-world evaluations of health outcomes.

We look forward to advancing this vital field together.

Dr. Hanieh Karimi
Dr. Xuyi Yue
Guest Editors

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Keywords

  • pediatric solid tumors
  • precision medicine
  • molecular profiling
  • targeted therapy
  • tumor biomarkers
  • treatment resistance
  • prognosis
  • survivorship
  • diagnostic tools
  • translational research

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Published Papers (2 papers)

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Research

14 pages, 851 KB  
Article
Non-Wilms Renal Tumours in Children: The Republic of Ireland Experience
by Kris Hughes, Charles Lee, Michael Capra, Jane Pears, Cormac Owens, Michael McDermott, Maureen O’Sullivan, Sri Paran and Israel Fernandez-Pineda
Children 2026, 13(4), 575; https://doi.org/10.3390/children13040575 - 21 Apr 2026
Viewed by 402
Abstract
Background: Non-Wilms renal tumours (NWRTs) are rare paediatric malignancies and account for a small but clinically significant proportion of childhood renal cancers. Due to their low incidence and biological heterogeneity, outcome data are limited, and management is largely extrapolated from international collaborative [...] Read more.
Background: Non-Wilms renal tumours (NWRTs) are rare paediatric malignancies and account for a small but clinically significant proportion of childhood renal cancers. Due to their low incidence and biological heterogeneity, outcome data are limited, and management is largely extrapolated from international collaborative protocols. No national data describing the incidence and outcomes of NWRTs in children in the Republic of Ireland (ROI) have previously been published. Objective: To determine the incidence, treatment strategies, and survival outcomes of NWRTs in children in the ROI. Methods: A retrospective cohort study was conducted of all children under 16 years of age with histologically confirmed renal tumours diagnosed and treated at Children’s Health Ireland (CHI) at Crumlin between January 2005 and December 2025. As CHI Crumlin is the single national paediatric oncology centre in the ROI, this cohort represents national case ascertainment for the study period. A total of 143 paediatric renal tumours were identified; Wilms tumours (n = 118) were excluded, leaving 25 children (17.48%) with NWRTs for analysis. No cases of bilateral renal tumours were identified. Histological subtypes included renal cell carcinoma (RCC), clear cell sarcoma of the kidney (CCSK), congenital mesoblastic nephroma (CMN), malignant rhabdoid tumour of the kidney (MRTK), and anaplastic sarcoma of the kidney. Demographic characteristics, treatment strategies, and survival outcomes were analysed. Results: Twenty-five children with NWRTs were identified: CCSK (n = 9), RCC (n = 7), CMN (n = 6), MRTK (n = 2), and anaplastic sarcoma of the kidney (n = 1). At a median follow-up of 107.9 months (range 4.5–181.3 months), overall survival for the cohort was 76%. Overall survival by histology was 100% for CMN, CCSK and anaplastic sarcoma, 43% for RCC, and 0% for MRTK. Treatment strategies varied by histology, with 68% undergoing upfront surgery, 32% receiving neoadjuvant chemotherapy, 60% receiving adjuvant systemic therapy, and 44% receiving radiotherapy. Tumour recurrence occurred in 4/25 patients (16%), confined to the RCC (3) and CMN (1) subgroups. Seven Event-Free Survival events were observed, comprising three RCC relapses and one RCC progression, one CMN relapse, and two MRTK progression-related deaths. No recurrences occurred in CCSK. Conclusions: NWRTs comprised 17.5% of all paediatric renal tumours diagnosed nationally during the study period and demonstrated marked heterogeneity in outcomes according to histological subtype. CMN showed excellent survival with six out of seven requiring surgery alone, whereas MRTK remained associated with dismal outcomes despite multimodal therapy. These national data support histology-driven, risk-adapted management and highlight the importance of continued international collaboration to improve outcomes in NWRTs. Full article
(This article belongs to the Special Issue Pediatric Solid Tumor: Precision Medicine and Long-Term Prognosis)
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15 pages, 1268 KB  
Article
Recent Trends in Clinical Trials for Pediatric Sarcoma in the United States: An Analysis of ClinicalTrials.gov
by Khaled Alkhawaldeh, Signe Thorpe, Sukjoo Cho, Alexandra Miller, Maua Alleyne, Jennifer Jones, Lynda Beaupin, Ajay Gupta and Jonathan Metts
Children 2026, 13(4), 455; https://doi.org/10.3390/children13040455 - 26 Mar 2026
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Abstract
Background/Objectives: Pediatric sarcomas are rare and heterogeneous malignancies for which clinical trials are essential to advance treatment and improve outcomes. However, the characteristics and trends of sarcoma clinical trials enrolling children in the United States have not been comprehensively described. This study [...] Read more.
Background/Objectives: Pediatric sarcomas are rare and heterogeneous malignancies for which clinical trials are essential to advance treatment and improve outcomes. However, the characteristics and trends of sarcoma clinical trials enrolling children in the United States have not been comprehensively described. This study aimed to characterize U.S.-based sarcoma clinical trials enrolling pediatric patients and to evaluate trends over time. Methods: ClinicalTrials.gov was searched for interventional sarcoma trials conducted in the United States that enrolled patients ≤ 17 years of age and were posted between 27 September 2007 and 11 January 2023. Trials were categorized as pediatric (maximum eligible age ≤ 21 years) or pediatric/adult (>21 years). Trial characteristics, including phase, intervention type, funding source, geographic scope, and reasons for early termination, were analyzed. Results: A total of 273 eligible trials were identified, of which 79% enrolled both pediatric and adult patients. Most studies were early phase (Phase 1, 2, or 1/2; 59%) and primarily evaluated drug or biologic therapies (73%). Trials involving mixed cancer types were most common (26%). The majority were multi-institutional (66%), non-industry funded (57%) and conducted exclusively in the United States (75%). Trial activations increased over time (p-value = 0.01), with a higher proportion of industry-funded studies initiated between 2016 and 2022 (p-value = 0.009). Twenty-three trials (8.4%) were terminated early, most commonly due to slow accrual (39%). Conclusions: Most sarcoma clinical trials enrolling pediatric patients continue to include both adult and pediatric populations, which may limit the development of therapies tailored to the unique biology of pediatric sarcomas. Improving outcomes will require greater emphasis on pediatric-focused research, enhanced collaboration across institutions, and increased awareness of clinical and regulatory frameworks to support the initiation of industry-funded trials. Full article
(This article belongs to the Special Issue Pediatric Solid Tumor: Precision Medicine and Long-Term Prognosis)
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