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Lassaletta A.AuthorRuano D.AuthorMadero L.Author

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October 28, 2024
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Safety and outcome of children, adolescents and young adults participating in phase I/II clinical oncology trials: a 9-year center experience

Publicated to:Frontiers in Pediatrics. 12 - 2024-01-01 12(), DOI: 10.3389/fped.2024.1423484

Authors: Pujol Manresa A; Buendía López S; Andión M; Herrero B; Lassaletta Á; Ramirez M; Ruano D; Hernández-Marqués C; Varo A; de Rojas T; Cortés Hernández M; Verdú-Amorós J; Martín Prado S; Artigas A; Redondo E; Ruiz Pato J; Herreros López P; Sevilla J; Madero L; Moreno L; Bautista Sirvent F; Rubio-San-Simón A

Affiliations

ACCELERATE - Author
Hospital Clinico Universitario de Valencia; Instituto de Investigación Sanitaria Fundación Para la Investigación del Hospital Clínico de Valencia - INCLIVA; Centro de Investigación Biomédica en Red de Cáncer - Author
Hospital Infantil Universitario Niño Jesús - Author
Hospital Infantil Universitario Niño Jesús; Hospital Universitari Vall d'Hebron - Author
Hospital Infantil Universitario Niño Jesús; La Princesa Research Institute - Author
Hospital Infantil Universitario Niño Jesús; Princess Máxima Center for Pediatric Oncology - Author
Hospital Regional Universitario Carlos Haya - Author
Hospital Universitari Vall d'Hebron - Author
Pediatric Cancer Center Barcelona - Author
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Abstract

Introduction: Enrolling children with cancer in early phase trials is crucial to access innovative treatments, contributing to advancing pediatric oncology research and providing tailored therapeutic options. Our objective is to analyze the impact of these trials on patient outcomes and safety, and to examine the evolution and feasibility of trials in pediatric cancer over the past decade. Methods: All patients recruited in pediatric anticancer phase I/II clinical trials from January 2014 to December 2022 were included. Clinical records and trial protocols were analyzed. Results: A total of 215 patients (median age 11.2 years, range 1–29.5) were included in 52 trials (258 inclusions). Patients with extracranial solid tumors (67%), central nervous system (CNS) tumors (24%), and leukemia (9%) were included. The most common investigational drugs were small molecules (28.3%) and antibodies (20.5%). Serious adverse events were experienced by 41% of patients, 4.4% discontinued treatment because of toxicity and two had toxic deaths. Median event-free survival was 3.7 months (95%CI: 2.8–4.5), longer in phase II trials than in phase I (2 vs. 6.3 months; p ≤ 0.001). Median overall survival was 12 months (95%CI: 9–15), higher in target-specific vs. non-target-specific trials (14 vs. 6 months; p ≤ 0.001). Discussion: A significant and increasing number of patients have been included in early clinical trials, suggesting that both oncologists and families consider it valuable to be referred to specialized Units to access new therapies. Moreover, our data suggests that participation in early clinical trials, although not without potential toxicities, might have a positive impact on individual outcomes.

Keywords

Access to innovationClinical researchClinical trialsDrug developmentPediatric hematology and oncology

Quality index

Impact and social visibility

From the perspective of influence or social adoption, and based on metrics associated with mentions and interactions provided by agencies specializing in calculating the so-called "Alternative or Social Metrics," we can highlight as of 2025-08-02:

  • The use, from an academic perspective evidenced by the Altmetric agency indicator referring to aggregations made by the personal bibliographic manager Mendeley, gives us a total of: 10.
  • The use of this contribution in bookmarks, code forks, additions to favorite lists for recurrent reading, as well as general views, indicates that someone is using the publication as a basis for their current work. This may be a notable indicator of future more formal and academic citations. This claim is supported by the result of the "Capture" indicator, which yields a total of: 10 (PlumX).

With a more dissemination-oriented intent and targeting more general audiences, we can observe other more global scores such as:

  • The Total Score from Altmetric: 0.75.
  • The number of mentions on the social network X (formerly Twitter): 2 (Altmetric).