Desmoid tumor

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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==
*2022: Benech et al. Desmoid tumors located in the abdomen or associated with adenomatous polyposis: French intergroup clinical practice guidelines for diagnosis, treatment, and follow-up (SNFGE, FFCD, GERCOR, UNICANCER, SFCD, SFED, SFRO, ACHBT, SFR) EuroPMC
==NCCN==
*NCCN does not currently have guidelines at this granular level; please see NCCN Guidelines - Soft Tissue Sarcoma.
=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====
*Imatinib (Gleevec) 800 mg PO once per day
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===
#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 EuroPMC
#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 EuroPMC 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====
*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===
#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 EuroPMC
#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 EuroPMC 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 	Longer PFS (primary endpoint)PFS24: 76% vs 44%(HR 0.29), 95% CI 0.15-0.55)
====Targeted therapy====
*Nirogacestat (Ogsiveo) 150 mg PO twice per day
28-day cycles
===References===
#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 EuroPMC 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====
*Pazopanib (Votrient) 800 mg PO once per day
28-day cycle for up to 13 cycles (1 year)
===References===
#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 EuroPMC NCT01876082
==Pegylated liposomal doxorubicin monotherapy==
===Regimen===
Study 	Dates 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===
#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 EuroPMC
==Sorafenib monotherapy==
===Regimen===
Study 	Dates of enrollment	Evidence 	Comparator	Comparative Efficacy
Gounder et al. 2018 (Alliance A091105)	2014-2016 	Phase 3 (E-esc)	Placebo 	Longer PFSPFS24: 81% vs 36%(HR 0.13, 95% CI 0.05-0.31)
====Targeted therapy====
*Sorafenib (Nexavar) 400 mg PO once per day on days 1 to 28
28-day cycles
===References===
# 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 EuroPMC NCT02066181
Category:Desmoid tumor regimens
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Category:Soft tissue sarcomas