"HER2-Positive Bile Duct Cancer, Superior Efficacy of Targeted Therapy and Cytotoxic Chemotherapy Combination Treatment"

Yonsei Cancer Hospital Professors Choi Hye-jin and Lee Chung-geun's Team
Improved Treatment Outcomes in Various Indicators Including Survival Period and Tumor Reduction Effects

[Asia Economy Reporter Lee Gwan-joo] A study has found that combining cytotoxic chemotherapy with targeted therapy yields superior treatment effects for HER2-positive cholangiocarcinoma.

The research team led by Professors Choi Hye-jin and Lee Chung-geun from the Department of Oncology at Yonsei Cancer Hospital announced on the 22nd that combination therapy of cytotoxic chemotherapy and targeted therapy in HER2-positive advanced cholangiocarcinoma showed an objective response rate (ORR) of about 30%, including tumor size reduction, indicating excellent treatment efficacy.

The team conducted combination chemotherapy on 34 patients with HER2-positive cholangiocarcinoma from eight domestic institutions, including Yonsei Cancer Hospital, between June 2020 and September last year. The targeted therapy used was Herjumah (trastuzumab), and the cytotoxic chemotherapy was the FOLFOX regimen.

During an approximately 13-month follow-up period, the objective response rate, which is the proportion of patients showing objective responses such as tumor size reduction, was about 30%, demonstrating good treatment efficacy. The disease control rate was confirmed to be 79.4%.

Progression-free survival and overall survival were 5.1 months and 10.7 months, respectively. The 1-year progression-free survival rate was 49.2%, and the median duration of response, which is the time the effect lasts, was 4.9 months. Compared to the previously reported effects of FOLFOX chemotherapy alone as the standard second-line treatment?objective response rate of 5%, progression-free survival of 4 months, and overall survival of 6.1 months?the combination of targeted therapy and cytotoxic chemotherapy showed superior efficacy.

Tumor reduction rates (above) and changes in tumor size during treatment for HER2-positive cholangiocarcinoma patients treated with a combination of cytotoxic chemotherapy and targeted therapy. [Source=Yonsei Medical Center]

The side effects observed in treated patients were mainly related to cytotoxic chemotherapy and were manageable, including anemia and neutropenia. Additionally, a quality of life follow-up survey based on patient-reported outcomes revealed that patients with lower quality of life before treatment had poorer treatment prognoses.

Professor Lee stated, "This study is the first to report the excellent efficacy and improved survival of combining cytotoxic chemotherapy and targeted therapy in patients with HER2-positive cholangiocarcinoma. It is expected that this combination therapy can be used as an effective new treatment option for HER2-positive patients with cholangiocarcinoma, which is prevalent in Korea, as a second- or third-line targeted therapy combination chemotherapy."

The study results were published in the latest issue of the international journal Lancet Gastroenterology and Hepatology (IF 45.042).

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