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Life sciences · Journal article
iScience · October 23, 2026
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Systemic therapy for recurrent or metastatic adenoid cystic carcinoma remains limited, and durable disease control is difficult to achieve. We conducted a prospective, single-arm phase 2 trial of chidamide, a histone deacetylase inhibitor, plus cisplatin in 24 patients with recurrent or metastatic disease. Patients received a short chidamide lead-in followed by combination therapy for up to six cycles. The study did not meet its prespecified objective response target, with a confirmed objective response rate of 13.6% among 22 efficacy-evaluable patients. However, disease control was observed in 86.4% of patients, median progression-free survival was 17.9 months, and median overall survival was 40.3 months. Toxicity was manageable, with no treatment-related deaths. These findings support further evaluation of histone deacetylase inhibitor-platinum strategies as a disease-control approach in this rare cancer, while requiring validation in larger studies.