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The Desmoid Tumor Research Foundation

September 2023

Initial Active Surveillance Strategy for Patients with Peripheral Sporadic Primary Desmoid-Type Fibromatosis: A Multicentric Phase II Observational Trial

Author Information

Sylvie Bonvalot 1, Nathalie Cozic 2, Axel Le Cesne 3, Jean Yves Blay 4, Nicolas Penel 5, Magali Fau 6, Christine Chevreau 7, Philippe Anract 8, Denis Waast 9, Valérie Laurence 10, Sarah Watson 10, Florence Duffaud 11, François Gouin 12, Sophie Taieb 13, Michèle Kind 14, Laurent Lam 2

1Department of Surgery, Institute Curie, Paris, France. sylvie.bonvalot@curie.fr.2Department of Biostatistics and Epidemiology, Gustave Roussy, Villejuif, France.3Department of Medical Oncology, Gustave Roussy, Villejuif, France.4Department of Medical Oncology, Centre Léon Bérard, Lyon, France.5Department of Medical Oncology, Centre Oscar Lambret, Lille, France.6Department of Surgery, Institut Bergonié, Bordeaux, France.7Department of Medical Oncology, Oncopole, Toulouse, France.8Department of Orthopedics, CHU Cochin, Paris, France.9Department of Orthopedics, CHU Nantes, Nantes, France.10Department of Medical Oncology, Institut Curie, Paris, France.11Department of Medical Oncology, CHU La Timone, Marseilles, France.12Department of Surgery, Centre Léon Bérard, Lyon, France.13Department of Radiology, Centre Oscar Lambret, Lille, France.14Department of Radiology, Institut Bergonié, Bordeaux, France.

Publication

Ann Surg Oncol

Summary

Background: Stabilization or spontaneous regressions are demonstrated in more than half of patients affected by primary desmoid-type fibromatosis (DF) in retrospective studies. The objective of this phase II study was to prospectively assess the behavior of primary sporadic DT managed by active surveillance (AS). Methods: This prospective, multicenter, observational study (NCT01801176) included patients ?18 years of age with primary sporadic DF located in an extremity or the abdominal/thoracic wall. At inclusion, all patients were initially placed on AS. Follow-up was based on clinical and radiological evaluation by magnetic resonance imaging (MRI) performed at 1, 3, 6, 9, and 12 months, and then every 6 months for 3 years. The primary endpoint was progression-free survival (PFS) at 3 years according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1, as evaluated by a Central Review Board. Results: Between 2012 and 2015, 100 patients were enrolled. The female/male ratio was 8 and the median age was 34 years (interquartile range [IQR] 30.8-43.9). Median follow-up was 46.6 months (IQR 36.8-61.1) and the 3-year PFS was 53.4% (95% confidence interval 43.5-63.1%). At progression (48 patients), 23 patients received active treatment. Fifty-eight patients (58%) presented with spontaneous tumor regression (decrease > 0% compared with the initial size) during the first 3 months (n = 35, 35%) or after an initial progression (n = 23, 23%), of whom 26 (26%) had partial responses (PRs). The median time to PR was 31.7 months (25.3-not available). Conclusions: These data support the use of AS as the primary approach to select patients with peripheral DF who require aggressive treatment.

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