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

ObjectiveDual checkpoint inhibitor therapy with nivolumab and ipilimumab has been FDA approved for a number of cancer sites. However, its role in the treatment of ACC and non-ACC salivary gland carcinomas (non-ACC SGC) is not well established.Methods and AnalysisWe performed 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 non-ACC SGC. The primary endpoint was median progression-free survival (PFS); secondary endpoints were overall response rate (ORR), overall survival (OS), and toxicity.ResultsPatient enrollment was prematurely terminated due to funding constraints. In total, 19 patients with ACC and 5 patients with non-ACC SGC were enrolled. The patients with ACC had a median OS of 30.0 months (95% CI 15.3-NR months), a median PFS of 8.3 months (95% CI 5.5-30.0 months), and a disease control rate (DCR) of 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 non-ACC SGC had a median OS of 10.4 months (95% CI 6.21-NR months), a median PFS of 6.21 months (95% CI 2.83-NR months), and a DCR of 40% (2/5). The ORR in this cohort was 0%.ConclusionIn patients with recurrent or metastatic ACC and non-ACC SGC, the combination of nivolumab with ipilimumab resulted in moderate disease control. Further studies are warranted to validate our findings. Trial number: NCT03146650.ConclusionIn patients with recurrent or metastatic ACC and non-ACC SGC, the combination of nivolumab with ipilimumab resulted in moderate disease control. Further studies are warranted to validate our findings. Trial number: NCT03146650.