Pembrolizumab therapy in a patient with advanced adrenocortical carcinoma and high tumor mutational burden (27.1/Mb) resulted in transient tumor shrinkage before death at 9 months.
Case Report (n=1)
Pembrolizumab may have therapeutic activity in advanced adrenocortical carcinoma with high tumor mutational burden, though pseudo-progression can complicate response evaluation.
ABSTRACT Introduction Adrenocortical carcinoma (ACC) is a rare and aggressive malignancy. Recent studies suggest that immune checkpoint inhibitors exhibit antitumor activity in selected ACC patients. Case Presentation We report a case of a 49‐year‐old man with advanced ACC and pulmonary metastasis treated with EDP‐M chemotherapy, followed by pembrolizumab based on high tumor mutational burden (TMB, 27.1/Mb). Despite suspected pseudo‐progression, transient tumor shrinkage was observed. However, the patient died of liver failure 9 months after diagnosis. Conclusion This case suggests a therapeutic role for ICIs in ACC. Moreover, it highlights the importance of TMB and immune RECIST criteria in evaluating treatment response.
Shimoda et al. (2026) conducted a case report in Advanced adrenocortical carcinoma (n=1). Pembrolizumab was evaluated. Pembrolizumab therapy in a patient with advanced adrenocortical carcinoma and high tumor mutational burden (27.1/Mb) resulted in transient tumor shrinkage before death at 9 months.