Temozolomide in aggressive and metastatic pituitary tumors: a Brazilian multicenter real-world cohort study
Year: 2026
Type: article
Study design: Observational Study
Abstract: PURPOSE: To evaluate the real-world efficacy and safety of temozolomide (TMZ) in aggressive and metastatic pituitary neuroendocrine tumors in a Latin American setting, and to address whether TMZ achieves meaningful radiological and biochemical disease control with acceptable toxicity. METHODS: We conducted a retrospective multicenter study across Brazilian reference centers including patients with aggressive/metastatic pituitary adenomas treated with TMZ and followed for ≥ 6 months. The radiological response was assessed via RECIST 1.1. For functioning pituitary adenomas, biochemical response was assessed using prespecified hormonal criteria. Adverse events were collected from medical records. RESULTS: Thirty patients were included (mean age 29.5 years; 53% female). All the tumors were macroadenomas, and 56% were giant (> 4 cm). Twenty-one pituitary adenomas were functioning and four were metastatic. Ki-67 was > 3% in 73% of the patients. The mean time from diagnosis to TMZ initiation was 102 months. The radiological disease control rate (partial response or stable disease) was 93.3%. Among functioning tumors, the biochemical disease control rate was 81.2%, with an objective biochemical response rate (complete + partial response) of 68.8%. Adverse events, most commonly nausea and myelotoxicity, occurred in 66% of patients. CONCLUSION: In this multicenter Brazilian real-world cohort, TMZ provided high radiological and biochemical disease control with an acceptable safety profile, suggesting that TMZ is the preferred first-line systemic chemotherapy for aggressive/metastatic pituitary adenomas after the failure of standard therapies.
Source: Pituitary
Authors: Isabelle Pinheiro Amaro de Magalhães, Camila Regina Pereira Batista de Macedo, Luciana Ansaneli Naves, Mauro Antonio Czepielewski, Isabella Naves Rosa, Lidiana Bandeira de Santana, Tobias Skrebsky de Almeida, Mario Padula, Paula Condé Lamparelli Elias, Margaret de Castro, Leandro Kasuki, Monica Roberto Gadelha, Carolina Garcia Soares Leães Rech, Heraldo Mendes Garmes, Cesar Luiz Boguszewski, Raquel Soares Jallad, Marilena Nakaguma, Maria Candida Barisson Villares Fragoso, Marcio Carlos Machado, Andrea Glezer, Malebranche Berardo Carneiro da Cunha Neto, Olavo Feher, Rafael Loch Batista
Institutions: Universidade de São Paulo, Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, Universidade de Brasília, Universidade Federal do Rio Grande do Sul, Hospital de Clínicas de Porto Alegre, Universidade Federal do Rio de Janeiro, Hospital Universitário Clementino Fraga Filho, Santa Casa Hospital, Universidade Estadual de Campinas (UNICAMP), Universidade Federal do Paraná, Instituto do Câncer do Estado de São Paulo
Language: English
Subfield: Endocrinology, Diabetes and Metabolism
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $4,490
Award: G3561301455
OpenAlex ID: W7141320569