Details

Autor(en) / Beteiligte

• Vince, Carolina Sgarioni Camargo
• Brassesco, Maria Sol
• Mançano, Bruna Minniti
• Gregianin, Lauro Jose
• Carbone, Edna Kakitani
• do Amaral E Castro, Adham
• Dwan, Viviane Sayuri Yamachira
• Menezes da Silva, Roberta Zeppini
• Mariano, Cassia Silvestre
• da Mata, Juliana França
• Silva, Marcelo Oliveira
• Caran, Eliana Maria Monteiro
• Macedo, Carla Donato
• Alves da Costa, Gildene
• Esteves, Tereza Cristina
• Silva, Luciana Nunes
• Ferman, Sima Esther
• Martins, Flavia Delgado
• Cristófani, Lilian Maria
• Odone-Filho, Vicente
• Silva, Marcelo Milone
• Reis, Rui Manuel
• Pianovski, Mara Albonei Dudeque
• Campregher, Paulo Vidal
• Kunii, Mayara Satsuki
• de Sá Rodrigues, Karla Emilia
• Carvalho Filho, Neviçolino Pereira
• Valera, Elvis Terci

Titel

Beyond Clinical Trials: Understanding Neurotrophic Tropomyosin Receptor Kinase Inhibitor Challenges and Efficacy in Real-World Pediatric Oncology

Ist Teil von

• JCO precision oncology, 2024-05, Vol.8 (8), p.e2300713

Ort / Verlag

United States

Erscheinungsjahr

2024

Quelle

MEDLINE

Beschreibungen/Notizen

• 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. 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. 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. 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.