ABSTRACT
PURPOSE: Our study aimed to explore real-world treatment scenarios for children and adolescents with neurotrophic tropomyosin receptor kinase (NTRK)-fused tumors, emphasizing access, responses, side effects, and outcomes. PATIENTS AND METHODS: Pooled clinical data from 17 pediatric cases (11 soft-tissue sarcomas, five brain tumors, and one neuroblastoma) treated with larotrectinib and radiologic images for 14 patients were centrally reviewed. Testing for gene fusions was prompted by poor response to treatment, tumor progression, or aggressiveness. RESULTS: Six different NTRK fusion subtypes were detected, and various payment sources for testing and medication were reported. Radiologic review revealed objective tumor responses (OR) in 11 of 14 patients: Complete responses: two; partial responses: nine; and stable disease: three cases. Grades 1 or 2 Common Terminology Criteria for Adverse Events adverse effects were reported in five patients. Regarding the entire cohort's clinical information, 15 of 17 patients remain alive (median observation time: 25 months): four with no evidence of disease and 11 alive with disease (10 without progression). One patient developed resistance to the NTRK inhibitor and died from disease progression while another patient died due to an unrelated cause. CONCLUSION: This real-world study confirms favorable agnostic tumor OR rates to larotrectinib in children with NTRK-fused tumors. Better coordination to facilitate access to medication remains a challenge, particularly in middle-income countries like Brazil.
Subject(s)
Protein Kinase Inhibitors , Pyrazoles , Humans , Child , Male , Female , Adolescent , Pyrazoles/therapeutic use , Child, Preschool , Protein Kinase Inhibitors/therapeutic use , Pyrimidines/therapeutic use , Receptor, trkA/genetics , Receptor, trkA/antagonists & inhibitors , Brain Neoplasms/drug therapy , Brain Neoplasms/genetics , Sarcoma/drug therapy , Sarcoma/genetics , Neuroblastoma/drug therapy , Neuroblastoma/genetics , Infant , Receptor, trkB/genetics , Receptor, trkC/genetics , Clinical Trials as TopicABSTRACT
Objetivo: relatar intervenções organizacionais vivenciadas pela equipe multiprofissional para a adaptação e manutenção de um serviço de oncologia pediátrica frente à pandemia do novo coronavírus. Método: Trata-se de um estudo descritivo exploratório junto a profissionais de saúde da oncologia pediátrica em parceria com enfermeiras do Serviço de Controle de Infecção Hospitalar e Educação Permanente em Saúde. Resultados: As adaptações e intervenções realizadas foram fundamentadas no Planejamento Comunicativo e organizado em três pilares: espaço físico; fluxo de atendimento; acolhimento e capacitação da equipe. Conclusão: As ações organizacionais para a adaptação e manutenção do serviço de oncologia pediátrica frente à pandemia, possibilitaram que os fluxos de atendimentos fossem mantidos com respeito às normas sociossanitárias e com direcionamento, que proporcionou assistência de qualidade às crianças e adolescentes
Objective: to report organizational interventions experienced by the multidisciplinary team for the adaptation and maintenance of a pediatric oncology service in the face of the new coronavirus pandemic. Method: This is an exploratory and descriptive study conducted with pediatric oncology health professionals in partnership with nurses from the Hospital Infection Control and Continuing Health Education Service. Results: The adaptations and interventions performed were based on Communicative Planning and organized into three pillars: physical space; service flow; reception and training of the team. Conclusion: The organizational actions for the adaptation and maintenance of the pediatric oncology service in the face of the pandemic allowed the flow of care to be maintained with respect to social and health standards and with guidance that provided quality care to children and adolescents.
Objetivo: informar de las intervenciones organizativas vividas por el equipo multidisciplinar para la adecuación y mantenimiento de un servicio de oncología pediátrica ante la nueva pandemia de coronavirus. Metodo: Este es un estudio descriptivo y exploratorio con los profesionales de la salud en oncología pediátrica en asociación con enfermeras del Servicio de Control de Infecciones Hospitalarias y Educación Continua en Salud. Resultados: Las adaptaciones e intervenciones realizadas se basaron en la Planificación Comunicativa y se organizaron en tres pilares: espacio físico; flujo de servicio; recepción y formación del equipo. Conclusión: Las acciones organizativas para la adecuación y mantenimiento del servicio de oncología pediátrica ante la pandemia permitieron mantener el flujo de atención con respecto a los estándares socio-sanitarios y con orientaciones que brinden una atención de calidad a la niñez y adolescencia