Gemcitabine and oxaliplatin combination in patients with advanced adrenocortical carcinoma

Abstract Context Adrenocortical cancer (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. Conclusions 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.