Nivolumab in Metastatic Adrenocortical Carcinoma: Results of a Phase 2 Trial

Benedito A. Carneiro, Bhavana Konda, Rubens B. Costa, Ricardo L.B. Costa, Vinay Sagar, Demirkan B. Gursel, Lawrence S. Kirschner, Young Kwang Chae, Sarki A. Abdulkadir, Alfred Rademaker, Devalingam Mahalingam, Manisha H. Shah, Francis J. Giles

Research output: Contribution to journalArticlepeer-review

50 Scopus citations


Context: Systemic treatment of metastatic adrenocortical carcinoma (ACC) remains limited to chemotherapy and mitotane. Preliminary evidence suggesting that antitumor immune responses can be elicited in ACC has fostered interest in checkpoint inhibitors such as anti-PD-1 nivolumab. Objective: The primary endpoint was objective response rate according to the response evaluation criteria in solid tumors. Secondary endpoints were progression-free survival (PFS), overall survival, and safety. Design: Single-arm, multicenter, phase 2 clinical trial with two-stage design. Setting: Comprehensive cancer center. Patients: Ten adult patients with metastatic ACC previously treated with platinum-based chemotherapy and/or mitotane as well as patients who declined front-line chemotherapy. Intervention: Nivolumab (240 mg) IV every 2 weeks. Results: Ten patients with metastatic ACC were enrolled between March and December 2016. The median number of doses of nivolumab administered was two. Three patients only received one treatment [one died of disease progression, one discontinued due to adverse events (AEs), one withdrew after beginning treatment]. The median PFS was 1.8 months. The median follow-up was 4.5 months (range, 0.1 to 25.6 months). Two patients had stable disease for a duration of 48 and 11 weeks, respectively. One patient had an unconfirmed partial response but discontinued the study due to an AE. Most AEs were grade 1/2. The most common grade 3/4 treatment-related AEs were aspartate aminotransferase and alanine aminotransferase elevations, mucositis, and odynophagia. Conclusion: Nivolumab demonstrated modest antitumor activity in patients with advanced ACC. The nivolumab safety profile was consistent with previous clinical experience without any unexpected AEs in this population.

Original languageEnglish (US)
Pages (from-to)6193-6200
Number of pages8
JournalJournal of Clinical Endocrinology and Metabolism
Issue number12
StatePublished - Dec 1 2019
Externally publishedYes

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Biochemistry
  • Endocrinology
  • Clinical Biochemistry
  • Biochemistry, medical


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