Phase 2 study of monotherapy with pembrolizumab for advanced adrenocortical carcinoma

Brenda Chahla; Bettzy Stephen; Juhee Song; Vania Balderrama-Brondani; Feyza Yaylaci; Matthew T. Campbell; Mouhammed Amir Habra

doi:10.1530/endoabs.107.006

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Endocrine Abstracts (2024) 107 006 | DOI: 10.1530/endoabs.107.006

IACS9 9th International Adrenal Cancer Symposium 2024 Abstracts (18 abstracts)

Phase 2 study of monotherapy with pembrolizumab for advanced adrenocortical carcinoma

Brenda Chahla ¹ , Bettzy Stephen ² , Juhee Song ³ , Vania Balderrama-Brondani ¹ , Feyza Yaylaci ¹ , Matthew T. Campbell ⁴ & Mouhammed Amir Habra ¹

Author affiliations

¹Department of Endocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA ²Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA ³Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA ⁴Department of Genitourinary Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA
Mouhammed Amir Habra, MD, Department of Endocrine Neoplasia and Hormonal Disorders, Unit 1461, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Blvd, Houston, TX 77030, USA. Email: [email protected]

Background: Adrenocortical carcinoma (ACC) is a rare cancer with suboptimal response to chemotherapy. The role of immunotherapy in ACC management is evolving.

Methods: An investigator-initiated, open-label, phase 2 clinical trial was performed to ascertain the activity and safety of monotherapy with pembrolizumab (a humanized monoclonal anti-programmed cell death protein 1 antibody) in patients with advanced ACC. This study was part of a basket clinical trial (clinical trial registration ID: NCT02721732). Study participants were enrolled from August 15, 2016, till December 7, 2020. Pembrolizumab (200 mg) was administered intravenously every 3 weeks. The primary endpoint was the non-progression rate (being alive without progression) at 27 weeks, which was objectively assessed by an independent radiology team based on the immune-related Response Evaluation Criteria in Solid Tumors. Secondary endpoints consisted of adverse events assessed using the Common Terminology Criteria for Adverse Events (version 4.03).

Results: We enrolled 23 ACC patients (13 female [57%]) with a median age of 54 years (range, 31-78 years). Four cases were cortisol producing (17%). The median follow-up calculated by reverse Kaplan-Meier is 66.9 months (95% CI, 44.7-87.3 months). The median progression-free survival time was 4.0 months (95% CI, 2-6 months), and the median overall survival time was 15.5 months (95% CI, 6-23 months). Among 20 patients with evaluable response, 6 (30%) were alive without progression at 27 weeks. We saw no complete responses but did see partial responses in 4 patients (20%) with median duration of response of 20.9 months (95% CI non-evaluable). The clinical benefit rate was 30% (complete response, partial response, and stable disease at 27 weeks). Three treatment-related adverse events were grade 3 or higher and were potential side effects of pembrolizumab. No treatment-related deaths occurred.

Conclusions: Single-agent pembrolizumab in treatment of advanced ACC has potential for durable responses and a manageable safety profile.Keywordspembrolizumab, immunotherapy, adrenocortical carcinoma, adverse events