The Desmoid Tumor Research Foundation

July 2026

Deni Study: Real-World Data on the Use of Nirogacestat in Patients With Desmoid Tumors in the French Sarcoma Group

Author Information

Pierre Kubicek 1, Catherine Guérin-Charbonnel 1 2, Maud Toulmonde 3, Pascaline Boudou-Rouquette 4, Valentin Thibaud 5, Nicolas Penel 6, Antoine Italiano 3, Thomas Ryckewaert 6, Nelly Firmin 7, François Bertucci 8, Armelle Dufresne 9, Patrick Soulié 1, Christophe Perrin 10, Sabine Noal 11, Mehdi Brahmi 9, Jean-Yves Blay 9, Emmanuelle Bompas 2

1Institut de Cancérologie de l'Ouest, Angers, France. 2Institut de Cancérologie de l'Ouest, Nantes, France. 3Institut Bergonié, Bordeaux, France. 4Hôpital Cochin, Paris, France. 5Institut Claudius Regaud, Toulouse, France. 6Centre Oscar Lambret, Lille, France. 7Institut du Cancer de Montpellier, INSERM U1194, IRCM, Université de Montpellier, Montpellier, France. 8Institut Paoli-Calmettes, Aix-Marseille Université, Marseille, France. 9Centre Léon Bérard, Lyon, France. 10Centre Eugène Marquis, Rennes, France. 11Centre François Baclesse, Caen, France.

Publication

Int J Cancer

Summary

The DeFi trial revealed a significant improvement in progression-free survival (PFS) with nirogacestat in patients with desmoid tumors. The DeNi study reports outcomes of patients treated with nirogacestat through the French compassionate use program. DeNi is a retrospective, real-world study. Outcomes included objective response, pain improvement, 1-year PFS, and toxicity. Between February and June 2024, 55 patients with desmoid tumors were included (37 women). Data were updated until November 2025. The median age was 38.6 years (range: 18.0-66.7). The median number of previous therapies was 2, including local therapies. The median follow-up was 21.4 months [95% CI: 19.5; 25.6]. The estimated median duration of nirogacestat treatment was 16 months. Partial response and stable disease were observed in 33 (60%) and 17 patients (31%), respectively. The 1-year PFS rate was 76.2%, and 77.0% of patients remained without treatment change at 1 year, including patients with dose reductions. Of the 49 patients with baseline pain, 38 (78%) experienced pain improvement under nirogacestat; 35 (71%) reduced or discontinued analgesics. The most frequent side effects (all grades) were diarrhea (53%), fatigue (47%), and rash (29%). Thirteen patients (24%) required dose reduction: 11 for digestive adverse events (85%), 1 for mucositis, and 1 for fatigue with hypertension. The DeNi study confirms the clinical benefits of nirogacestat in patients with desmoid tumors, with improvements in pain and tumor shrinkage observed in 60% of patients and PFS consistent with that reported in the phase III DeFi trial.

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