Desmoid tumor

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Section editor
Elizabeth J. Davis, MD
Vanderbilt University
Nashville, TN, USA

Note: these are regimens tested in subtype-specific populations, please see the main soft tissue sarcoma page for other regimens. For placebo or observational studies in this condition, please visit this page. If you still can't find it, please let us know so we can add it!

Last updated on 2024-07-23:
6 regimens on this page
7 variants on this page


Guidelines

Given the rapid change in evidence in many areas of hematology/oncology, readers are encouraged to consider any guideline published 5+ years ago to be for historical purposes, only.

French Intergroup


NCCN


All lines of therapy

Imatinib monotherapy

Regimen variant #1, fixed dose

Study Dates of enrollment Evidence
Kasper et al. 2017 (GISG-01) 2010-2013 Phase 2

Targeted therapy

2 years of therapy


Regimen variant #2, BSA-based dosing

Study Dates of enrollment Evidence
Chugh et al. 2010 2002-10 to 2005-12 Phase 2

Targeted therapy

  • Imatinib (Gleevec) by the following BSA-based criteria:
    • 1.50 m2 or more: 300 mg PO twice per day
    • 1.00 to 1.49 m2: 200 mg PO twice per day
    • Less than 1.00 m2: 100 mg PO twice per day

Continued indefinitely

References

  1. Chugh R, Wathen JK, Patel SR, Maki RG, Meyers PA, Schuetze SM, Priebat DA, Thomas DG, Jacobson JA, Samuels BL, Benjamin RS, Baker LH; Sarcoma Alliance for Research through Collaboration (SARC). Efficacy of imatinib in aggressive fibromatosis: Results of a phase II multicenter Sarcoma Alliance for Research through Collaboration (SARC) trial. Clin Cancer Res. 2010 Oct 1;16(19):4884-91. Epub 2010 Aug 19. link to original article dosing details in manuscript have been reviewed by our editors PubMed
  2. GISG-01: Kasper B, Gruenwald V, Reichardt P, Bauer S, Rauch G, Limprecht R, Sommer M, Dimitrakopoulou-Strauss A, Pilz L, Haller F, Hohenberger P. Imatinib induces sustained progression arrest in RECIST progressive desmoid tumours: Final results of a phase II study of the German Interdisciplinary Sarcoma Group (GISG). Eur J Cancer. 2017 May;76:60-67. Epub 2017 Mar 8. link to original article dosing details in manuscript have been reviewed by our editors PubMed NCT01137916


Methotrexate & Vinblastine

Regimen

Study Dates of enrollment Evidence
Skapek et al. 2007 (POG 9650) 1997-2001 Phase 2
Toulmonde et al. 2019 (DESMOPAZ) 2012-2017 Randomized Phase 2

Note: DESMOPAZ was randomized but non-comparative.

Chemotherapy

7-day cycle for 26 cycles, then 14-day cycle for 13 cycles

References

  1. POG 9650: Skapek SX, Ferguson WS, Granowetter L, Devidas M, Perez-Atayde AR, Dehner LP, Hoffer FA, Speights R, Gebhardt MC, Dahl GV, Grier HE; Pediatric Oncology Group. Vinblastine and methotrexate for desmoid fibromatosis in children: results of a Pediatric Oncology Group Phase II Trial. J Clin Oncol. 2007 Feb 10;25(5):501-6. link to original article dosing details in manuscript have been reviewed by our editors PubMed
  2. DESMOPAZ: Toulmonde M, Pulido M, Ray-Coquard I, Andre T, Isambert N, Chevreau C, Penel N, Bompas E, Saada E, Bertucci F, Lebbe C, Le Cesne A, Soulie P, Piperno-Neumann S, Sweet S, Cecchi F, Hembrough T, Bellera C, Kind M, Crombe A, Lucchesi C, Le Loarer F, Blay JY, Italiano A. Pazopanib or methotrexate-vinblastine combination chemotherapy in adult patients with progressive desmoid tumours (DESMOPAZ): a non-comparative, randomised, open-label, multicentre, phase 2 study. Lancet Oncol. 2019 Sep;20(9):1263-1272. Epub 2019 Jul 19. link to original article dosing details in manuscript have been reviewed by our editors PubMed NCT01876082


Nirogacestat monotherapy

Regimen

Study Dates of enrollment Evidence Comparator Comparative Efficacy
Gounder et al. 2023 (DeFi) 2019-05 to 2020-08 Phase 3 (E-RT-esc) Placebo Superior PFS (primary endpoint)
PFS24: 76% vs 44%
(HR 0), 95% CI 0.15-0.55)

Targeted therapy

28-day cycles

References

  1. DeFi: Gounder M, Ratan R, Alcindor T, Schöffski P, van der Graaf WT, Wilky BA, Riedel RF, Lim A, Smith LM, Moody S, Attia S, Chawla S, D'Amato G, Federman N, Merriam P, Van Tine BA, Vincenzi B, Benson C, Bui NQ, Chugh R, Tinoco G, Charlson J, Dileo P, Hartner L, Lapeire L, Mazzeo F, Palmerini E, Reichardt P, Stacchiotti S, Bailey HH, Burgess MA, Cote GM, Davis LE, Deshpande H, Gelderblom H, Grignani G, Loggers E, Philip T, Pressey JG, Kummar S, Kasper B. Nirogacestat, a γ-Secretase Inhibitor for Desmoid Tumors. N Engl J Med. 2023 Mar 9;388(10):898-912. link to original article dosing details in manuscript have been reviewed by our editors PubMed NCT03785964


Pazopanib monotherapy

Regimen

Study Dates of enrollment Evidence
Toulmonde et al. 2019 (DESMOPAZ) 2012-2017 Randomized Phase 2

Note: this study was randomized but non-comparative.

Targeted therapy

28-day cycle for up to 13 cycles (1 year)

References

  1. DESMOPAZ: Toulmonde M, Pulido M, Ray-Coquard I, Andre T, Isambert N, Chevreau C, Penel N, Bompas E, Saada E, Bertucci F, Lebbe C, Le Cesne A, Soulie P, Piperno-Neumann S, Sweet S, Cecchi F, Hembrough T, Bellera C, Kind M, Crombe A, Lucchesi C, Le Loarer F, Blay JY, Italiano A. Pazopanib or methotrexate-vinblastine combination chemotherapy in adult patients with progressive desmoid tumours (DESMOPAZ): a non-comparative, randomised, open-label, multicentre, phase 2 study. Lancet Oncol. 2019 Sep;20(9):1263-1272. Epub 2019 Jul 19. link to original article dosing details in manuscript have been reviewed by our editors PubMed NCT01876082


Pegylated liposomal doxorubicin monotherapy

Regimen

Study Dates of enrollment Evidence
Constantinidou et al. 2009 2006-2009 Case series

Chemotherapy

28-day cycles

References

  1. Case series: Constantinidou A, Jones RL, Scurr M, Al-Muderis O, Judson I. Pegylated liposomal doxorubicin, an effective, well-tolerated treatment for refractory aggressive fibromatosis. Eur J Cancer. 2009 Nov;45(17):2930-4. Epub 2009 Sep 18. link to original article PubMed


Sorafenib monotherapy

Regimen

Study Dates of enrollment Evidence Comparator Comparative Efficacy
Gounder et al. 2018 (Alliance A091105) 2014-2016 Phase 3 (E-esc) Placebo Superior PFS
PFS24: 81% vs 36%
(HR 0.13, 95% CI 0.05-0.31)

Targeted therapy

28-day cycles

References

  1. Alliance A091105: Gounder MM, Mahoney MR, Van Tine BA, Ravi V, Attia S, Deshpande HA, Gupta AA, Milhem MM, Conry RM, Movva S, Pishvaian MJ, Riedel RF, Sabagh T, Tap WD, Horvat N, Basch E, Schwartz LH, Maki RG, Agaram NP, Lefkowitz RA, Mazaheri Y, Yamashita R, Wright JJ, Dueck AC, Schwartz GK. Sorafenib for Advanced and Refractory Desmoid Tumors. N Engl J Med. 2018 Dec 20;379(25):2417-2428. link to original article dosing details in abstract have been reviewed by our editors link to PMC article PubMed NCT02066181


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