Desmoid tumor

Section editor
	Elizabeth J. Davis, MD Vanderbilt University Nashville, TN ejdavis25

Note: these are regimens tested in subtype-specific populations, please see the main soft tissue sarcoma page for other regimens.

5 regimens on this page 7 variants on this page

All lines of therapy

Doxorubicin pegylated liposomal monotherapy

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Regimen

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

Chemotherapy

• Pegylated liposomal doxorubicin (Doxil) 50 mg/m² IV over 60 minutes once on day 1

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

Methotrexate & Vinblastine

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Regimen

Study	Years 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

• Methotrexate (MTX) 30 mg/m² IV once on day 1
• Vinblastine (Velban) 5 mg/m² IV once on day 1

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 contains verified protocol 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 contains protocol PubMed NCT01876082

Imatinib monotherapy

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Regimen variant #1, fixed dose

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

Targeted therapy

• Imatinib (Gleevec) 800 mg PO once per day

2 years of therapy

Regimen variant #2, BSA-based dosing

Study	Years of enrollment	Evidence
Chugh et al. 2010	2002-2005	Phase 2

Targeted therapy

• Imatinib (Gleevec) by the following criteria:
  • BSA of 1.5 m² or more: 300 mg PO twice per day
  • BSA between 1 and 1.49 m²: 200 mg PO twice per day
  • BSA less than 1 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 contains verified protocol 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 contains verified protocol PubMed NCT01137916

Pazopanib monotherapy

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Regimen

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

Note: this study was randomized but non-comparative.

Targeted therapy

• Pazopanib (Votrient) 800 mg PO once per day

Up to 12 months of therapy

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 contains protocol PubMed NCT01876082

Sorafenib monotherapy

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Regimen

Study	Years of enrollment	Evidence	Comparator	Comparative Efficacy
Gounder et al. 2018 (Alliance A091105)	2014-2016	Phase 3 (E-esc)	Placebo	Superior PFS

Targeted therapy

• Sorafenib (Nexavar) 400 mg PO once per day

Continued indefinitely

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 contains protocol link to PMC article PubMed NCT02066181