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Thalidomide for refractory hemorrhagic chronic radiation proctitis secondary to pelvic malignancy radiotherapy: A phase II clinical trial
Xiao-Yan Huang, Qing-Hua Zhong, Ying-Yi Kuang, Zhi-Jie Li, Bin-Jie Huang, Yan-Jiong He, Miao-Miao Zhu, Qi Guan, Xiao-Yan Li, Qi-Yuan Qin, Teng-Hui Ma
Xiao-Yan Huang, Xiao-Yan Li, Department of Pharmacy, The Sixth Affiliated Hospital, Sun Yat-sen University, Guangzhou 510655, Guangdong Province, China
Xiao-Yan Huang, Qing-Hua Zhong, Ying-Yi Kuang, Zhi-Jie Li, Bin-Jie Huang, Yan-Jiong He, Miao-Miao Zhu, Qi Guan, Xiao-Yan Li, Qi-Yuan Qin, Teng-Hui Ma, Guangdong Provincial Key Laboratory of Colorectal and Pelvic Floor Diseases, The Sixth Affiliated Hospital, Sun Yat-sen University, Guangzhou 510655, Guangdong Province, China
Xiao-Yan Huang, Qing-Hua Zhong, Ying-Yi Kuang, Zhi-Jie Li, Bin-Jie Huang, Yan-Jiong He, Miao-Miao Zhu, Qi Guan, Xiao-Yan Li, Qi-Yuan Qin, Teng-Hui Ma, Biomedical Innovation Center, The Sixth Affiliated Hospital, Sun Yat-sen University, Guangzhou 510655, Guangdong Province, China
Qing-Hua Zhong, Department of Endoscopic Surgery, The Sixth Affiliated Hospital, Sun Yat-sen University, Guangzhou 510655, Guangdong Province, China
Ying-Yi Kuang, Zhi-Jie Li, Bin-Jie Huang, Yan-Jiong He, Miao-Miao Zhu, Qi Guan, Qi-Yuan Qin, Teng-Hui Ma, Department of Colorectal Surgery, The Sixth Affiliated Hospital, Sun Yat-sen University, Guangzhou 510655, Guangdong Province, China
Teng-Hui Ma, Department of Clinical Nutrition, The Sixth Affiliated Hospital, Sun Yat-sen University, Guangzhou 510655, Guangdong Province, China
Co-first authors: Xiao-Yan Huang and Qing-Hua Zhong.
Co-corresponding authors: Qi-Yuan Qin and Teng-Hui Ma.
Author contributions: Huang XY and Zhong QH contributed equally as co-first authors; Ma TH, Qin QY, Li XY, and Huang XY conceived the idea and designed the study; Huang XY, Zhong QH, Kuang YY, Li ZJ, Huang BJ, He YJ, Zhu MM, and Guan Q collected the data and followed the patients; Huang XY, Zhong QH, Kuang YY, Ma TH, Qin QY, and Li XY analyzed and interpreted the data; Huang XY, Zhong QH, and Kuang YY drafted the manuscript; Ma TH, Qin QY, and Li XY supervised the study, performed validation, and revised the manuscript; Ma TH and Qin QY made equal contributions as co-corresponding authors. All authors have read and approve the final manuscript.
Supported by Clinical Research 1010 Program of the Sixth Affiliated Hospital of Sun Yat-sen University, No. 1010CG(2022)-09; Guangdong Provincial Hospital Pharmaceutical Research Foundation, No. 2021A07; Hospital Pharmaceutical Research Foundation of Guangdong Hospital Association, No. YXKY202212; China International Medical Foundation, No. Z-2021-46-2101-2023; and Program of Guangdong Provincial Clinical Research Center for Digestive Diseases, No. 2020B1111170004.
Institutional review board statement: The study was reviewed and approved by the Ethical Committee of the Sixth Affiliated Hospital of Sun Yat-sen University, No. 2020ZSLYEC-207.
Clinical trial registration statement: This was a single-arm, open-label, single-center, phase II clinical trial registered at
https://clinicaltrials.gov (NCT04680195).
Informed consent statement: All enrolled patients provided informed consent prior to receiving treatment.
Conflict-of-interest statement: All the authors report no relevant conflicts of interest for this article.
CONSORT 2010 statement: The authors have read the CONSORT 2010 Statement, and the manuscript was prepared and revised according to the CONSORT 2010 Statement.
Data sharing statement: The anonymised patient datasets created and/or analysed for the current study are available from the corresponding author on reasonable request.
Corresponding author: Teng-Hui Ma, Department of Colorectal Surgery, The Sixth Affiliated Hospital, Sun Yat-sen University, No. 26 Yuancun Erheng Road, Guangzhou 510655, Guang
dong Province, China.
matengh@mail.sysu.edu.cn
Received: November 14, 2025
Revised: December 19, 2025
Accepted: February 3, 2026
Published online: April 14, 2026
Processing time: 140 Days and 23.5 Hours
BACKGROUND
Radiotherapy, a cornerstone for the treatment of pelvic malignancies, often causes pelvic-abdominal organ damage, with chronic radiation proctitis (CRP) as the primary toxicity. Over 90% of CRP patients experience persistent, irreversible hematochezia, and its management is challenging due to the limited availability of effective drugs.
AIM
To evaluate the efficacy and safety of thalidomide in CRP patients suffering from intractable bleeding.
METHODS
A single-arm, open-label, phase II clinical trial was implemented to evaluate the therapeutic efficacy and safety of thalidomide in treating hemorrhagic CRP. Patients who had a diagnosis of hemorrhagic CRP with a Subjective Objective Management Analysis system (SOMA) score for hematochezia ≥ 2, or a transfusion history resulting from CRP, were given 50 mg thalidomide orally for 4 months. The primary endpoint was assessment of the therapeutic efficacy of thalidomide.
RESULTS
From October 2020 to February 2024, 62 individuals were enrolled, of whom the median SOMA score was 3 (interquartile range 3-4), and 8 (12.9%) patients were transfusion-dependent due to hematochezia resulting from CRP. The overall efficacy rate of thalidomide in improving the SOMA score was 83.9% (52 of 62), with rates of 58.3% (7 of 12, ≤ 2 months), 95.1% (39 of 41, 3-6 months), and 100% (6 of 6, > 6 months). Significant improvements in endoscopic manifestations were visually observed in 60% (21 of 35) of patients. Adverse events occurred in 37.1% (23 of 62) of the study patients, all categorized as the National Cancer Institute’s Common Terminology Criteria for Adverse Events grade 1-2 manifestations.
CONCLUSION
Thalidomide is effective and well-tolerated for the treatment of hemorrhagic CRP. Further prospective multicenter research is warranted to verify these findings.
Core Tip: Chronic radiation proctitis (CRP) is a common complication of pelvic radiotherapy. Its most frequent symptom is hematochezia, which is often persistent and irreversible. However, the availability of oral drug treatments for hemorrhagic CRP are still extremely limited. This single-arm, open-label, phase II clinical trial investigated the therapeutic efficacy and safety of thalidomide for refractory hemorrhagic CRP. The results demonstrated that thalidomide achieved a hematochezia remission rate of up to 83.9%, with only mild or moderate adverse events. In summary, thalidomide appears to be an effective and well-tolerated treatment option for hemorrhagic CRP.
