2026-04-13T06:37:01Zhttps://recercat.cat/oai/request

oai:recercat.cat:11351/121592025-10-24T10:23:37Zcom_2072_378070com_2072_378040col_2072_378092

00925njm 22002777a 4500 dc Pujol Manresa, Anna author Buendía López, Susana author MAITANE, ANDION author Herrero, Blanca author Lassaletta, Álvaro author Ramirez Orellana, Manuel author Moreno, Lucas author 2024-10-31T07:17:02Z 2024-10-31T07:17:02Z 2024-09-04 Access to innovation; Drug development; Pediatric hematology and oncology Accés a la innovació; Desenvolupament de fàrmacs; Hematologia i oncologia pediàtrica Acceso a la innovación; Desarrollo de fármacos; Hematología y oncología pediátrica 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. http://hdl.handle.net/11351/12159 Càncer - Tractament Avaluació de resultats (Assistència sanitària) Assaigs clínics ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Diagnosis::Prognosis::Treatment Outcome DISEASES::Neoplasms Other subheadings::Other subheadings::Other subheadings::/drug therapy ANALYTICAL, DIAGNOSTIC AND THERAPEUTIC TECHNIQUES, AND EQUIPMENT::Investigative Techniques::Epidemiologic Methods::Epidemiologic Study Characteristics::Clinical Studies as Topic::Clinical Trials as Topic TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::diagnóstico::pronóstico::resultado del tratamiento ENFERMEDADES::neoplasias Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia TÉCNICAS Y EQUIPOS ANALÍTICOS, DIAGNÓSTICOS Y TERAPÉUTICOS::técnicas de investigación::métodos epidemiológicos::características de los estudios epidemiológicos::estudios clínicos como asunto::ensayos clínicos como asunto Safety and outcome of children, adolescents and young adults participating in phase I/II clinical oncology trials: a 9-year center experience