PD-1 Blockade in Advanced Adrenocortical Carcinoma

• The authors of this phase II study evaluated the activity of pembrolizumab in 39 patients with advanced adrenocortical carcinoma. The objective response rate was 29%, with the median duration of response not reached. The disease control rate was 52%. Responses were seen in patients with both microsatellite-high (MSI-H) and mismatch repair–deficient (MMR-D) tumors. PD-L1 expression and MSI-H/MMR-D status were not associated with an objective response. Median progression-free and overall survival were 2.1 months and 24.9 months, respectively.

• Pembrolizumab appears to be an effective therapy in patients with this rare, aggressive malignancy with few treatment options.

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