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The Desmoid Tumor Research Foundation

March 2024

Favorable clinical efficacy of cytotoxic chemotherapy in patients with progressive desmoid tumors: a retrospective real-world study

Author Information

Chung Ryul Oh 1 2, Hyehyun Jeong 1, Wanlim Kim 3, Jong-Seok Lee 3, Si Yeol Song 4, Joon Seon Song 5, Kyung-Ja Cho 5, Hye Won Chung 6, Min Hee Lee 6, Jung Yong Hong 7, Jeeyun Lee 7, Jeong Eun Kim # 8, Jin-Hee Ahn # 9

1Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, 88, Olympic-ro 43-gil, Songpa-gu, 05505, Seoul, Republic of Korea. 2Department of Internal Medicine, Chung-Ang University Hospital, Chung-Ang University College of Medicine, Seoul, Republic of Korea. 3Department of Orthopedic Surgery, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea. 4Department of Radiation Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea. 5Department of Pathology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea. 6Department of Radiology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea. 7Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea. 8Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, 88, Olympic-ro 43-gil, Songpa-gu, 05505, Seoul, Republic of Korea. jeongeunkim@amc.seoul.kr. 9Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, 88, Olympic-ro 43-gil, Songpa-gu, 05505, Seoul, Republic of Korea. drjiny@amc.seoul.kr. #Contributed equally.

Publication

Int J Clin Oncol

Summary

Background: The real-world evidence about the efficacy of cytotoxic chemotherapy in desmoid tumors is still limited. We investigated the efficacy of chemotherapy in the treatment of recurrent or progressive desmoid tumors. Methods: The patients with desmoid tumors who had received cytotoxic chemotherapy between November 2007 and June 2020 in two tertiary hospitals in Korea were reviewed. Results: A total of 25 patients were included in the analysis. The most common primary tumor site was the intra-abdominal or pelvic cavity (56%), followed by the trunk and abdominal wall (24%), extremities (16%), and head and neck (4%). Sixty percent of the patients had familial adenomatous polyposis and 76% received doxorubicin plus dacarbazine. The objective response rate and disease control rate was 64% (95% confidence interval [CI]: 40.7-82.8) and 96% (95% CI: 77.2-99.9), respectively. With the median follow-up time of 55 months (95% CI: 41.0-68.2), the 3-year PFS rate was 65% (95% CI: 41.1-80.5), and the 3-year OS rate was 89% (95% CI: 63.8-97.3). Grade 3 or 4 hematologic adverse events were reported in 14 patients, all of which were manageable. Conclusion: Our real-world evidence suggests that doxorubicin-based cytotoxic chemotherapy can be an effective treatment option for recurrent and progressive desmoid tumors with respect to favorable clinical outcomes. Keywords: Cytotoxic chemotherapy; Desmoid tumors; Doxorubicin; Familial adenomatous polyposis; Real-world evidence.

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