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

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Abstract

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.

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Data availability

The datasets generated during and/or analyzed during the current study are available from the corresponding author on reasonable request.

References

  1. WHO Classification of Tumours Editorial Board (2020) Soft tissue and bone tumours. In: International agency for research on cancer, WHO classification of tumours series, vol. 3, 5th edn. Lyon, France

  2. Biermann JS (2000) Desmoid tumors. Curr Treat Options Oncol 1(3):262–266. https://doi.org/10.1007/s11864-000-0038-5

    Article  CAS  PubMed  Google Scholar 

  3. Penel N, Chibon F, Salas S (2017) Adult desmoid tumors: biology, management and ongoing trials. Curr Opin Oncol 29(4):268–274. https://doi.org/10.1097/cco.0000000000000374

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  4. Bonvalot S, Ternès N, Fiore M et al (2013) Spontaneous regression of primary abdominal wall desmoid tumors: more common than previously thought. Ann Surg Oncol 20(13):4096–4102. https://doi.org/10.1245/s10434-013-3197-x

    Article  PubMed  Google Scholar 

  5. Stoeckle E, Coindre JM, Longy M et al (2009) A critical analysis of treatment strategies in desmoid tumours: a review of a series of 106 cases. Eur J Surg Oncol 35(2):129–134. https://doi.org/10.1016/j.ejso.2008.06.1495

    Article  CAS  PubMed  Google Scholar 

  6. Salas S, Dufresne A, Bui B et al (2011) Prognostic factors influencing progression-free survival determined from a series of sporadic desmoid tumors: a wait-and-see policy according to tumor presentation. J Clin Oncol 29(26):3553–3558. https://doi.org/10.1200/jco.2010.33.5489

    Article  PubMed  Google Scholar 

  7. Kasper B, Baumgarten C, Garcia J et al (2017) An update on the management of sporadic desmoid-type fibromatosis: a European Consensus Initiative between Sarcoma PAtients EuroNet (SPAEN) and European Organization for Research and Treatment of Cancer (EORTC)/Soft Tissue and Bone Sarcoma Group (STBSG). Ann Oncol 28(10):2399–2408. https://doi.org/10.1093/annonc/mdx323

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  8. Gronchi A, Raut CP (2012) Optimal approach to sporadic desmoid tumors: from radical surgery to observation. time for a consensus? Ann Surg Oncol 19(13):3995–3997. https://doi.org/10.1245/s10434-012-2636-4

    Article  PubMed  Google Scholar 

  9. Lev D, Kotilingam D, Wei C et al (2007) Optimizing treatment of desmoid tumors. J Clin Oncol 25(13):1785–1791. https://doi.org/10.1200/jco.2006.10.5015

    Article  PubMed  Google Scholar 

  10. de Camargo VP, Keohan ML, D’Adamo DR et al (2010) Clinical outcomes of systemic therapy for patients with deep fibromatosis (desmoid tumor). Cancer 116(9):2258–2265. https://doi.org/10.1002/cncr.25089

    Article  PubMed  Google Scholar 

  11. Okuno SH, Edmonson JH (2003) Combination chemotherapy for desmoid tumors. Cancer 97(4):1134–1135. https://doi.org/10.1002/cncr.11189

    Article  PubMed  Google Scholar 

  12. Toulmonde M, Pulido M, Ray-Coquard I et al (2019) Pazopanib or methotrexate–vinblastine combination chemotherapy in adult patients with progressive desmoid tumours (DESMOPAZ): a non-comparative, randomised, open-label, multicentre, phase 2 study. Lancet Oncol 20(9):1263–1272. https://doi.org/10.1016/s1470-2045(19)30276-1

    Article  CAS  PubMed  Google Scholar 

  13. Gounder MM, Mahoney MR, Van Tine BA et al (2018) Sorafenib for advanced and refractory desmoid tumors. N Engl J Med 379(25):2417–2428. https://doi.org/10.1056/NEJMoa1805052

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  14. Fiore M, Colombo C, Radaelli S et al (2015) Hormonal manipulation with toremifene in sporadic desmoid-type fibromatosis. Eur J Cancer 51(18):2800–2807. https://doi.org/10.1016/j.ejca.2015.08.026

    Article  CAS  PubMed  Google Scholar 

  15. Hansmann A, Adolph C, Vogel T et al (2004) High-dose tamoxifen and sulindac as first-line treatment for desmoid tumors. Cancer 100(3):612–620. https://doi.org/10.1002/cncr.11937

    Article  CAS  PubMed  Google Scholar 

  16. Garbay D, Le Cesne A, Penel N et al (2012) Chemotherapy in patients with desmoid tumors: a study from the French Sarcoma Group (FSG). Ann Oncol 23(1):182–186. https://doi.org/10.1093/annonc/mdr051

    Article  CAS  PubMed  Google Scholar 

  17. Constantinidou A, Jones RL, Scurr M et al (2009) Pegylated liposomal doxorubicin, an effective, well-tolerated treatment for refractory aggressive fibromatosis. Eur J Cancer 45(17):2930–2934. https://doi.org/10.1016/j.ejca.2009.08.016

    Article  CAS  PubMed  Google Scholar 

  18. Gega M, Yanagi H, Yoshikawa R et al (2006) Successful chemotherapeutic modality of doxorubicin plus dacarbazine for the treatment of desmoid tumors in association with familial adenomatous polyposis. J Clin Oncol 24(1):102–105. https://doi.org/10.1200/jco.2005.02.1923

    Article  CAS  PubMed  Google Scholar 

  19. Eisenhauer EA, Therasse P, Bogaerts J et al (2009) New response evaluation criteria in solid tumours: revised RECIST guideline version 1.1. Eur J Cancer 45(2):228–247. https://doi.org/10.1016/j.ejca.2008.10.026

    Article  CAS  PubMed  Google Scholar 

  20. Colombo C, Vullo SL, Fiore M et al (2021) Active surveillance in primary desmoid tumor (DT): a prospective observational study. J Clin Oncol 39(15):11570–11570. https://doi.org/10.1200/JCO.2021.39.15_suppl.11570

    Article  Google Scholar 

  21. Gronchi A, Jones RL (2019) Treatment of desmoid tumors in 2019. JAMA Oncol 5(4):567–568. https://doi.org/10.1001/jamaoncol.2018.6449

    Article  PubMed  Google Scholar 

  22. Penel N, Le Cesne A, Bonvalot S et al (2017) Surgical versus non-surgical approach in primary desmoid-type fibromatosis patients: a nationwide prospective cohort from the French Sarcoma Group. Eur J Cancer 83:125–131. https://doi.org/10.1016/j.ejca.2017.06.017

    Article  PubMed  Google Scholar 

  23. Alman B, Attia S, Baumgarten C et al (2020) The management of desmoid tumours: a joint global consensus-based guideline approach for adult and paediatric patients. Eur J Cancer 127:96–107. https://doi.org/10.1016/j.ejca.2019.11.013

    Article  Google Scholar 

  24. Nathenson MJ, Hu J, Ratan R et al (2022) Systemic chemotherapies retain antitumor activity in desmoid tumors independent of specific mutations in CTNNB1 or APC: a multi-institutional retrospective study. Clin Cancer Res 28(18):4092–4104. https://doi.org/10.1158/1078-0432.Ccr-21-4504

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  25. Park KH, Choi YJ, Kim KW et al (2016) Combination chemotherapy with methotrexate and vinblastine for surgically unresectable, aggressive fibromatosis. Jpn J Clin Oncol 46(9):845–849. https://doi.org/10.1093/jjco/hyw081

    Article  PubMed  Google Scholar 

  26. Napolitano A, Provenzano S, Colombo C et al (2020) Familial adenomatosis polyposis-related desmoid tumours treated with low-dose chemotherapy: results from an international, multi-institutional, retrospective analysis. ESMO Open 5(1):1–6. https://doi.org/10.1136/esmoopen-2019-000604

    Article  Google Scholar 

  27. Azzarelli A, Gronchi A, Bertulli R et al (2001) Low-dose chemotherapy with methotrexate and vinblastine for patients with advanced aggressive fibromatosis. Cancer 92(5):1259–1264. https://doi.org/10.1002/1097-0142(20010901)92:5%3c1259::aid-cncr1446%3e3.0.co;2-y

    Article  CAS  PubMed  Google Scholar 

  28. Skapek SX, Ferguson WS, Granowetter L et al (2007) Vinblastine and methotrexate for desmoid fibromatosis in children: results of a Pediatric Oncology Group Phase II Trial. J Clin Oncol 25(5):501–506. https://doi.org/10.1200/jco.2006.08.2966

    Article  CAS  PubMed  Google Scholar 

  29. Skapek SX, Anderson JR, Hill DA et al (2013) Safety and efficacy of high-dose tamoxifen and sulindac for desmoid tumor in children: results of a Children’s Oncology Group (COG) phase II study. Pediatr Blood Cancer 60(7):1108–1112. https://doi.org/10.1002/pbc.24457

    Article  CAS  PubMed  Google Scholar 

  30. Szucs Z, Messiou C, Wong HH et al (2017) Pazopanib, a promising option for the treatment of aggressive fibromatosis. Anticancer Drugs 28(4):421–426. https://doi.org/10.1097/cad.0000000000000474

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  31. Kasper BRR, Alcindor T, Schoeffski P et al (2022) DeFi: a phase III, randomized controlled trial of nirogacestat versus placebo for progressing desmoid tumors. Ann Oncol 33:S681–S700

    Article  Google Scholar 

  32. Gounder MM, Jones RL, Chugh R et al (2022) Initial results of phase II/III trial of AL102 for treatment of desmoid tumors (DT). Ann Oncol 33:S681–S700

    Article  Google Scholar 

  33. Gounder M, Ratan R, Alcindor T et al (2023) Nirogacestat, a γ-secretase inhibitor for desmoid tumors. N Engl J Med 388(10):898–912. https://doi.org/10.1056/NEJMoa2210140

    Article  CAS  PubMed  Google Scholar 

  34. Martínez Trufero J, PajaresBernad I, Torres Ramón I et al (2017) Desmoid-type fibromatosis: who, when, and how to treat. Curr Treat Options Oncol 18(5):29. https://doi.org/10.1007/s11864-017-0474-0

    Article  PubMed  Google Scholar 

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Funding

This work was supported by a grant from Asan Institute for Life Sciences, Asan Medical Center, Seoul, Korea (#2018-0570) and a grant ‘Elimination of Cancer Project Fund’ from Asan Cancer Institute of Asan Medical Center, Seoul, Korea.

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Authors and Affiliations

Authors

Contributions

CRO, JEK and J-HA had full access to all the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis. Study concept: JEK and J-HA. Study design: JEK and J-HA. Data acquisition: CRO, HJ, WK, J-SL, SYS, JSS, K-JC, HWC, MHL, JYH and JL. Data analysis and interpretation: CRO and J-HA. Manuscript writing: CRO and JEK. Manuscript editing: CRO, JEK and J-HA. Final approval: CRO, HJ, WK, J-SL, SYS, JSS, K-JC, HWC, MHL, JYH, JL, JEK and J-HA.

Corresponding authors

Correspondence to Jeong Eun Kim or Jin-Hee Ahn.

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Conflict of interest

The authors have no relevant financial or non-financial interests to disclose.

Ethics approval

This study was performed in line with the principles of the Declaration of Helsinki. This study was approved by the Institutional Review Board of Asan Medical Center and Samsung Medical Center, Seoul, Korea.

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Oh, C.R., Jeong, H., Kim, W. et al. Favorable clinical efficacy of cytotoxic chemotherapy in patients with progressive desmoid tumors: a retrospective real-world study. Int J Clin Oncol (2024). https://doi.org/10.1007/s10147-024-02506-2

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