Temozolomide in aggressive and metastatic pituitary tumors: a Brazilian multicenter real-world cohort study

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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.