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Original Article
Differential Efficacy of Alpelisib by PIK3CA Mutation Site in Head and Neck Squamous Cell Carcinoma: An Analysis from the KCSG HN 15-16 TRIUMPH Trial
Kyoo Hyun Kim1orcid , Shinwon Hwang2, Min Kyoung Kim3, Keon-Uk Park4, Tak Yun5, Keun-Wook Lee6, Joo Hang Kim7, Bhumsuk Keam8, Byoung Chul Cho1, So Yeon Oh9, Sang Hee Cho10, Sangwoo Kim11,12, Sung-Bae Kim13orcid , Min Hee Hong1orcid , Hye Ryun Kim1,12orcid

DOI: https://doi.org/10.4143/crt.2024.1195 [Accepted]
Published online: January 31, 2025
1Divison of Medical Oncology, Department of Internal Medicine, Yonsei Cancer Center, Yonsei University College of Medicine, Seoul, Korea
2Department of Dermatology, Yonsei University College of Medicine, Seoul, Korea
3Division of Hematology-Oncology, Department of Internal Medicine, Yeungnam University Hospital, Yeungnam University College of Medicine, Daegu, Korea
4Department of Internal Medicine, Keimyung University Dongsan Hospital, Daegu, Korea
5Rare Cancers Clinic, Center for Specific Organs Center, National Cancer Center, Goyang, Korea
6Department of Internal Medicine, Seoul National University Bundang Hospital, Seoul National University College of Medicine, Seongnam, Korea
7Department of Internal Medicine, CHA Bundang Medical Center, CHA University, Seongnam, Korea
8Department of Internal Medicine, Seoul National University Hospital, Seoul, Korea
9Department of Internal Medicine, Pusan National University Yangsan Hospital, Pusan National University School of Medicine, Yangsan, Korea
10Department of Hemato-Oncology, Chonnam National University Hwasun Hospital, Chonnam National University Medical School, Hwasun, Korea
11Department of Biomedical Systems Informatics, Yonsei University College of Medicine, Seoul, Korea
12Brain Korea 21 PLUS Project for Medical Science, Yonsei University College of Medicine, Seoul, Korea
13Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea
Corresponding author:  Sung-Bae Kim
Tel: 82-2-3010-3217 Email: sbkim3@amc.seoul.kr
Min Hee Hong
Tel: : 82-2-2228-8129  Email: minhee_hong@yuhs.ac
Hye Ryun Kim
Tel: 82-2-2228-8125 Email: nobelg@yuhs.ac
Received: 11 December 2024   • Accepted: 27 January 2025
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Purpose
The TRIUMPH trial was a biomarker-driven umbrella trial for patients with recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC). This analysis focuses on the PIK3CAɑ inhibitor alpelisib (arm 1) in patients with phosphoinositide 3-kinase (PI3K) pathway alterations.
Materials and Methods
Patients with PI3K pathway altered tumors were enrolled in the alpelisib arm of the TRIUMPH study. We conducted a detailed analysis of the correlation between PI3K pathway mutations and treatment outcomes including disease control rate (DCR), overall survival (OS), and progression-free survival (PFS).
Results
From Oct 2017 and Aug 2020, 203 were enrolled, with 42 treated with alpelisib. Response evaluation was possible for 33 patients. Genomic profiles revealed PIK3CA amplifications in 26.2%, and point mutations in E542K (26.2%), E545K (23.8%), and H1047R (9.5%). Neither PIK3CA amplification nor co-occurring TP53 mutations had a notable influence on alpelisib response or survival outcomes. Although the overall response rates were similar between helical domain mutations (E542, E545) and kinase domain mutations (H1047), patients with H1047 mutations exhibited significantly poorer PFS compared to those with non-H1047 PIK3CA alterations (1.6 vs. 7.3 months, p=0.017). OS in patients with H1047 kinase domain mutations showed a trend toward being shorter compared to others, though this difference did not reach statistical significance.
Conclusion
Alpelisib showed differential efficacy based on PI3K pathway alterations in patients with R/M HNSCC and was well-tolerated. These findings suggest the usefulness of NGS testing-based decision-making when using the targeted agents in R/M HNSCC. We need to confirm results in larger cohorts.

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    Differential Efficacy of Alpelisib by PIK3CA Mutation Site in Head and Neck Squamous Cell Carcinoma: An Analysis from the KCSG HN 15-16 TRIUMPH Trial
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