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

Abstract 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 Simon’s 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. Trial number: NCT03146650