Difference between revisions of "Desmoid tumor"
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{{TOC limit|limit=3}} | {{TOC limit|limit=3}} | ||
+ | =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. [https://doi.org/10.1016/j.dld.2022.03.004 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)] [https://pubmed.ncbi.nlm.nih.gov/35508462/ PubMed] | ||
+ | |||
+ | ==NCCN== | ||
+ | *''NCCN does not currently have guidelines at this granular level; please see [https://www.nccn.org/guidelines/guidelines-detail?category=1&id=1464 NCCN Guidelines - Soft Tissue Sarcoma].'' | ||
+ | |||
=All lines of therapy= | =All lines of therapy= | ||
==Imatinib monotherapy {{#subobject:hg35dc|Regimen=1}}== | ==Imatinib monotherapy {{#subobject:hg35dc|Regimen=1}}== | ||
Line 45: | Line 53: | ||
====Targeted therapy==== | ====Targeted therapy==== | ||
*[[Imatinib (Gleevec)]] by the following BSA-based criteria: | *[[Imatinib (Gleevec)]] by the following BSA-based criteria: | ||
− | ** | + | **1.50 m<sup>2</sup> or more: 300 mg PO twice per day |
− | ** | + | **1.00 to 1.49 m<sup>2</sup>: 200 mg PO twice per day |
− | ** | + | **Less than 1.00 m<sup>2</sup>: 100 mg PO twice per day |
'''Continued indefinitely''' | '''Continued indefinitely''' | ||
</div></div> | </div></div> | ||
+ | |||
===References=== | ===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. [https://doi.org/10.1158/1078-0432.ccr-10-1177 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/20724445/ PubMed] | #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. [https://doi.org/10.1158/1078-0432.ccr-10-1177 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/20724445/ PubMed] | ||
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===References=== | ===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. [https://doi.org/10.1200/jco.2006.08.2966 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/17290057/ PubMed] | #'''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. [https://doi.org/10.1200/jco.2006.08.2966 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/17290057/ PubMed] | ||
− | #'''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. [https://doi.org/10.1016/s1470-2045(19)30276-1 link to original article] '''contains dosing details in | + | #'''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. [https://doi.org/10.1016/s1470-2045(19)30276-1 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/31331699/ PubMed] [https://clinicaltrials.gov/study/NCT01876082 NCT01876082] |
+ | ==Nirogacestat monotherapy {{#subobject:f3agdc|Regimen=1}}== | ||
+ | <div class="toccolours" style="background-color:#eeeeee"> | ||
+ | ===Regimen {{#subobject:1fc9c2|Variant=1}}=== | ||
+ | {| class="wikitable sortable" style="width: 100%; text-align:center;" | ||
+ | !style="width: 20%"|Study | ||
+ | !style="width: 20%"|Dates of enrollment | ||
+ | !style="width: 20%"|[[Levels_of_Evidence#Evidence|Evidence]] | ||
+ | !style="width: 20%"|Comparator | ||
+ | !style="width: 20%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]] | ||
+ | |- | ||
+ | |[https://doi.org/10.1056/NEJMoa2210140 Gounder et al. 2023 (DeFi)] | ||
+ | |2019-05 to 2020-08 | ||
+ | | style="background-color:#1a9851" |Phase 3 (E-RT-esc) | ||
+ | |[[Desmoid_tumor_-_null_regimens#Placebo|Placebo]] | ||
+ | | style="background-color:#1a9850" |Superior PFS (primary endpoint)<br>PFS24: 76% vs 44%<br>(HR 0.29, 95% CI 0.15-0.55) | ||
+ | |- | ||
+ | |} | ||
+ | <div class="toccolours" style="background-color:#b3e2cd"> | ||
+ | ====Targeted therapy==== | ||
+ | *[[Nirogacestat (Ogsiveo)]] 150 mg PO twice per day | ||
+ | '''28-day cycles''' | ||
+ | </div></div> | ||
+ | ===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. [https://doi.org/10.1056/NEJMoa2210140 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/36884323/ PubMed] [https://clinicaltrials.gov/study/NCT03785964 NCT03785964] | ||
+ | |||
==Pazopanib monotherapy {{#subobject:f3agdc|Regimen=1}}== | ==Pazopanib monotherapy {{#subobject:f3agdc|Regimen=1}}== | ||
<div class="toccolours" style="background-color:#eeeeee"> | <div class="toccolours" style="background-color:#eeeeee"> | ||
Line 97: | Line 131: | ||
====Targeted therapy==== | ====Targeted therapy==== | ||
*[[Pazopanib (Votrient)]] 800 mg PO once per day | *[[Pazopanib (Votrient)]] 800 mg PO once per day | ||
− | ''' | + | '''28-day cycle for up to 13 cycles (1 year)''' |
</div></div> | </div></div> | ||
===References=== | ===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. [https://doi.org/10.1016/s1470-2045(19)30276-1 link to original article] '''contains dosing details in | + | #'''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. [https://doi.org/10.1016/s1470-2045(19)30276-1 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/31331699/ PubMed] [https://clinicaltrials.gov/study/NCT01876082 NCT01876082] |
+ | |||
==Pegylated liposomal doxorubicin monotherapy {{#subobject:cd6479|Regimen=1}}== | ==Pegylated liposomal doxorubicin monotherapy {{#subobject:cd6479|Regimen=1}}== | ||
<div class="toccolours" style="background-color:#eeeeee"> | <div class="toccolours" style="background-color:#eeeeee"> | ||
Line 140: | Line 175: | ||
<div class="toccolours" style="background-color:#b3e2cd"> | <div class="toccolours" style="background-color:#b3e2cd"> | ||
====Targeted therapy==== | ====Targeted therapy==== | ||
− | *[[Sorafenib (Nexavar)]] 400 mg PO once per day | + | *[[Sorafenib (Nexavar)]] 400 mg PO once per day on days 1 to 28 |
− | ''' | + | '''28-day cycles''' |
</div></div> | </div></div> | ||
===References=== | ===References=== |
Latest revision as of 11:23, 13 May 2024
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!
5 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) PubMed
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 |
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 contains dosing details in manuscript PubMed
- 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 dosing details in manuscript 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
- 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 contains dosing details in manuscript PubMed
- 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 dosing details in manuscript 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 0.29, 95% CI 0.15-0.55) |
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 contains dosing details in manuscript 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
- 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 contains dosing details in manuscript PubMed 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 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) |
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 contains dosing details in abstract link to PMC article PubMed NCT02066181