New guidance on managing desmoid fibromatosis recommends a more conservative approach, encompassing the patient perspective, active surveillance, tumor location, and risk-benefit assessment.
Surgery plus anlotinib as a treatment regimen for desmoid fibromatosis (DF), an aggressive connective tissue malignancy also known as aggressive fibromatosis1 and a type of soft tissue sarcoma (STS),2 was proven effective and safe in the setting of local recurrence control, according to a new study published in Frontiers in Pharmacology.3
Outcomes were investigated among patients who have resectable DF of the extremities, who were divided into 2 cohorts: surgery alone (n = 23) or surgery plus anlotinib (n = 25). Anlotinib is a multitarget tyrosine kinase inhibitor approved for use in non–small cell lung cancer and under investigation for use in STS in China,4 which is where the present retrospective study took place. In the US, oral Ogsiveo (nirogacestat) is the only FDA-approved treatment for desmoid tumors.5
All of the included patients had pathologically confirmed DF treated with surgery between January 2010 and June 2022; crossover from the surgery-alone to the surgery-plus-anlotinib group was allowed if a patient had postop disease recurrence. Data were gathered on tumor location, anlotinib toxicity, time to recurrence, surgical complications, follow-up, visual analogue scale (VAS) score, and Musculoskeletal Tumor Society (MSTS) score at last follow-up. The primary outcome was recurrence-free survival (RFS), defined as “interval from the date of surgical intervention to the date of tumor recurrence or patient death due to the tumor or the last follow-up.”
Overall, the 3-year RFS rate was close to twice as high in the surgery-plus-anlotinib compared with the surgery-alone group: 72.6% vs 37.7% (P = .022), a finding the investigators deemed statistically significant.
The median (IQT) patient age was 25 (19-38.8) years, 60.4% were female patients, the most common tumor locations were the glutes (25%) and the thigh and scapular region (20.8% each), median tumor size was 8.2 (5.8-11.3) cm, median previous surgeries were 2 (1-2), and 83.3% of patients had recurrent disease vs 16.7% who had primary disease. Ten patients crossed from the surgery-alone to the surgery-plus-anlotinib cohort.
Seventy-three percent of the study population had their surgery before 2018. No patients died during the study.
The surgery-only patient group had a longer median follow-up, at 45 (28.5-66.5) months vs 40 (27-50) months, but a shorter time from surgery to disease recurrence of 17.5 (12.5-31) months vs 24 (19.5-35) months. The VAS score of the surgery-alone group at final follow-up was higher than the surgery-with-anlotinib group (5 [3-6] vs 2 [1-3]), indicating less symptom improvement, and median MSTS scores were 19 (16.5-24) and 27 (25-28), respectively, meaning those who underwent surgery and received anlotinib had better physical functioning and less pain.
Thirteen patients overall experienced surgical complications. There were 3 cases of wound infection, 3 cases of temporary iatrogenic nerve injury, and 7 cases of wound healing problems. The top 5 major adverse events (AEs) were hand-foot-skin syndrome (52.2%), hypertension (43.5%), fatigue (43.5%), paramenia (34.8%), and vomiting (30.4%). No patients experienced grade 4 AEs or discontinued anlotinib.
Limitations on these findings include its retrospective nature, which could have introduced selection and recall biases; the small patient population; patients with incomplete data; and the short follow-up.
The authors recommend that future studies include patients with abdominal or trunk lesions, and that new analyses should be prospective randomized clinical trials.
References
1. Ganeshan DM, Amini B, Nikolaidis P, Assing M, Vikran R. Current update on desmoid fibromatosis. J Comput Assist Tomogr. 2019;4391):29-38. doi:10.1097/RCT.0000000000000790
2. Desmoid sarcoma. Sarcoma Foundation of America. Accessed April 23, 2024. https://www.curesarcoma.org/sarcoma-resources/patient-resources/sarcoma-subtypes/desmoid-sarcoma/
3. Yuan D, Liu Y, Fang X, et al. Surgery combined with anlotinib for local control of patients with resectable extremity desmoid fibromatosis: a retrospective study. Front Pharmacol. 2024:15:1357071. doi:10.3389/fphar.2024.1357071
4. Shen G, Zhen F, Ren D, et al. Anlotinib: a novel multi-targeting tyrosine kinase inhibitor in clinical development. J Hematol Oncol. 2018;11(1):120. doi:10.1186/s13045-018-0664-7
5. Caffrey M, Steinzor P. FDA approves nirogacestat for treatment of desmoid tumors. AJMC. November 27, 2023. Accessed April 9, 2024. https://www.ajmc.com/view/fda-approves-nirogacestat-for-treatment-of-desmoid-tumors
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