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

  • Sarcoma
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Abstract

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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Authors and Affiliations

  1. Department of Surgery, Institute Curie, Paris, France

    Sylvie Bonvalot MD, PhD

  2. Department of Biostatistics and Epidemiology, Gustave Roussy, Villejuif, France

    Nathalie Cozic PhD & Laurent Lam PhD

  3. Department of Medical Oncology, Gustave Roussy, Villejuif, France

    Axel Le Cesne MD

  4. Department of Medical Oncology, Centre Léon Bérard, Lyon, France

    Jean Yves Blay MD, PhD

  5. Department of Medical Oncology, Centre Oscar Lambret, Lille, France

    Nicolas Penel MD, PhD

  6. Department of Surgery, Institut Bergonié, Bordeaux, France

    Magali Fau MD

  7. Department of Medical Oncology, Oncopole, Toulouse, France

    Christine Chevreau MD

  8. Department of Orthopedics, CHU Cochin, Paris, France

    Philippe Anract MD

  9. Department of Orthopedics, CHU Nantes, Nantes, France

    Denis Waast MD

  10. Department of Medical Oncology, Institut Curie, Paris, France

    Valérie Laurence MD & Sarah Watson MD, PhD

  11. Department of Medical Oncology, CHU La Timone, Marseilles, France

    Florence Duffaud MD

  12. Department of Surgery, Centre Léon Bérard, Lyon, France

    François Gouin MD

  13. Department of Radiology, Centre Oscar Lambret, Lille, France

    Sophie Taieb MD

  14. Department of Radiology, Institut Bergonié, Bordeaux, France

    Michèle Kind MD

Authors
  1. Sylvie Bonvalot MD, PhD
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  2. Nathalie Cozic PhD
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  3. Axel Le Cesne MD
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  4. Jean Yves Blay MD, PhD
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  6. Magali Fau MD
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  7. Christine Chevreau MD
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  8. Philippe Anract MD
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  9. Denis Waast MD
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  10. Valérie Laurence MD
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  12. Florence Duffaud MD
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  13. François Gouin MD
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  14. Sophie Taieb MD
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  15. Michèle Kind MD
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  16. Laurent Lam PhD
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Corresponding author

Correspondence to Sylvie Bonvalot MD, PhD.

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DISCLOSURE

Sylvie Bonvalot: Grant from INCA (the French National Cancer Institute). Axel Le Cesne: Honoraria (fees) from Deciphera, PharmaMar, and Bayer. Jean-Yves Blay: NETSARC+, LYriCAN, and LYriCAN+ (all funded by the French National Cancer Institute). Sarah Watson: Consultancy work for Deciphera and Amgen. Philippe Anract: Consultant for Medcata International and Amplitude, and attended Scientific Meetings for Sanofi and Grunenthal. Intellectual property, patents and copyrights: Serf. Orthopedic prosthesis. Nathalie Cozic, Nicolas Penel, Magali Fau, Christine Chevreau, Denis Waast, Valérie Laurence, Florence Duffaud, François Gouin, Sophie Taieb, Michèle Kind, and Laurent Lam have no disclosures to declare.

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Bonvalot, S., Cozic, N., Le Cesne, A. et al. Initial Active Surveillance Strategy for Patients with Peripheral Sporadic Primary Desmoid-Type Fibromatosis: A Multicentric Phase II Observational Trial. Ann Surg Oncol 30, 8653–8659 (2023). https://doi.org/10.1245/s10434-023-14341-2

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  • DOI: https://doi.org/10.1245/s10434-023-14341-2

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