Phase II study of nivolumab and ipilimumab for treatment of metastatic/recurrent adenoid cystic carcinoma (ACC) of all anatomic sites of origin and other malignant salivary gland tumors

Objective: Dual checkpoint inhibitor therapy with nivolumab and ipilimumab has been FDA-approved for a number of cancer sites. However, their role in the treatment of ACC and other salivary gland carcinomas is not well established. Methods and analysis: We performed a Simons two-stage prospective single-institution phase II clinical trial of nivolumab with ipilimumab. Two cohorts were analyzed: patients with metastatic/recurrent ACC and patients with other salivary gland cancers. The primary endpoint was median progression-free survival (PFS); secondary endpoints were overall response rate (ORR), overall survival (OS), and toxicity. Results: Patients with ACC (n=19) and other salivary gland carcinomas (total n=5) were enrolled. The patients with ACC had median OS 30.0 months (95% CI 15.3-NR months), median PFS 8.3 months (95% CI 5.5-30.0 months), and disease control rate (DCR) 53% (10/19). The ORR in the ACC group was 5% (CR 0%, n=0; confirmed PR 5%, n=1), with one patient having continued stable disease at the time of trial conclusion. The patients with other salivary gland cancers had median OS 10.4 months (95% CI 6.21-NR months), median PFS 6.21 months (95% CI 2.83-NR months), and DCR 40% (2/5). The ORR in this cohort was 0%. Conclusion: In patients with recurrent or metastatic ACC and other salivary gland neoplasms, combination nivolumab with ipilimumab resulted in moderate disease control. Further studies are warranted to validate our findings.