Gemcitabine and oxaliplatin combination in patients with advanced adrenocortical carcinoma

Context: Adrenocortical carcinoma (ACC) is a rare, aggressive malignancy with limited treatment options beyond first-line therapy, underscoring the need for effective salvage regimens. Objective: To evaluate the clinical activity and safety of gemcitabine and oxaliplatin (GemOx) in advanced ACC. Design: Retrospective cohort study conducted from April 2023 to April 2024 with longitudinal follow-up. Setting: Single-center tertiary referral cancer center. Patients or Other Participants: Fourteen patients with histologically confirmed advanced ACC treated with GemOx were included. Patients were heavily pretreated, with a median of 3 prior systemic therapies (range, 2-9); 43% had hormonally functional tumors. Intervention(s): Gemcitabine (1000 mg/m² on days 1 and 8) and oxaliplatin (130 mg/m² on day 1) every 3 weeks until disease progression or unacceptable toxicity. Main Outcome Measure(s): Primary outcomes were progression-free survival (PFS) and overall survival (OS). Secondary outcomes included objective response rate per Response Evaluation Criteria In Solid Tumors 1.1 and treatment-related toxic effects. Results: After a median follow-up of 10.7 months (95% CI, 8.5-15.7), median PFS was 3.2 months (95% CI, 0.4-6.0) and OS was 13.0 months (95% CI, 3.6-22.5). Among 13 evaluable patients, 2 (15.4%) achieved partial response, 8 (61.5%) had stable disease, and 3 (23.1%) had progressive disease, yielding a disease control rate of 76.9%. No treatment-related deaths occurred. Conclusion: GemOx demonstrated modest clinical activity with manageable safety in heavily pretreated patients with advanced ACC. These findings suggest a potential role for GemOx as a salvage option, though validation in larger prospective studies is needed.