Management of Patients with Newly Diagnosed Desmoid Tumors in a First-Line Setting

SIMPLE SUMMARY: Desmoid tumors are benign neoplasms that invade locally, causing significant disability and morbidity. Historically, patients with desmoid tumors have been treated with surgery despite the significant morbidity associated with this modality. Less invasive treatments have emerged, including active surveillance, systemic therapy, radiotherapy, and local ablation. However, it remains unclear which patients would benefit most from an initial conservative rather than interventional approach. To answer this question, we retrospectively analyzed 262 patients with desmoid tumors treated at our institution over a period of 30 years. Our results suggest that initial active surveillance is a good option for patients with small and minimally symptomatic desmoid tumors, while tyrosine kinase inhibitors, local ablation, and surgery seem to be equally effective in those with more aggressive disease. ABSTRACT: The initial management of desmoid tumors (DTs) is shifting from surgery towards active surveillance, with systemic and locally ablative treatments reserved for enlarging and/or symptomatic disease. However, it remains unclear which patients would benefit most from an initial conservative rather than interventional approach. To answer this question, we retrospectively analyzed adult and pediatric patients with DTs treated at a tertiary academic cancer center between 1992 and 2022. Outcomes measured were progression-free survival (PFS) and time to next treatment (TTNT) after first-line therapy. A total of 262 treatment-naïve patients were eligible for analysis with a median age of 36.5 years (range, 0–87 years). The 5-year PFS and the median TTNT (months) after first-line treatment were, respectively: 50.6% and 69.1 mo for surgery; 64.9% and 149.5 mo for surgery plus adjuvant radiotherapy; 57.1% and 44.7 mo for surgery plus adjuvant systemic therapy; 24.9% and 4.4 mo for chemotherapy; 26.7% and 5.3 mo for hormonal therapy; 41.3% and 29.6 mo for tyrosine kinase inhibitors (TKIs); 44.4% and 8.9 mo for cryoablation and high intensity focused ultrasound; and 43.1% and 32.7 mo for active surveillance. Age [Formula: see text] 40 years (p < 0.001), DTs involving the extremities (p < 0.001), a maximum tumor diameter [Formula: see text] 60 mm (p = 0.04), and hormonal therapy (p = 0.03) predicted a higher risk of progression. Overall, our results suggest that active surveillance should be considered initially for patients with smaller asymptomatic DTs, while upfront TKIs, local ablation, and surgery achieve similar outcomes in those with more aggressive disease.