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Frontiers of Medicine

Front. Med.    2019, Vol. 13 Issue (4) : 427-437     https://doi.org/10.1007/s11684-018-0672-6
REVIEW
Targeted therapy of desmoid-type fibromatosis: mechanism, current situation, and future prospects
Zhen Wang, Jianhui Wu, Xiuyun Tian, Chunyi Hao()
Key Laboratory of Carcinogenesis and Translational Research, Ministry of Education, Department of Hepato–Pancreato–Biliary Surgery, Peking University Cancer Hospital and Institute, Beijing 100142, China
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Abstract

Desmoid-type fibromatosis (DF) is a rare monoclonal fibroblastic proliferation that is characterized by locally infiltrative but rarely metastatic lesions. Tyrosine kinase and γ-secretase inhibitors are primarily used in the targeted therapy of DF. The use of these drugs, however, is mainly based on the recommendations of retrospective studies with small sample sizes. Previous studies that focused on the mechanism, efficacy, and safety of targeted therapy for DF were reviewed to provide references for clinical applications and research. The efficacy and safety of targeted therapy were compared with those of other systemic therapy options. Targeted therapy does not provide considerable advantages in efficacy and safety over other medical treatments and is usually applied after the failure of antihormonal therapies, nonsteroidal anti-inflammatory drugs, and chemotherapy. Further studies are required to explore the mechanism, indications, and appropriate drug dosage of the targeted therapy of DF.

Keywords targeted therapy      desmoid-type fibromatosis      tyrosine kinase inhibitor      γ-secretase inhibitor     
Corresponding Authors: Chunyi Hao   
Just Accepted Date: 28 December 2018   Online First Date: 22 February 2019    Issue Date: 02 August 2019
 Cite this article:   
Zhen Wang,Jianhui Wu,Xiuyun Tian, et al. Targeted therapy of desmoid-type fibromatosis: mechanism, current situation, and future prospects[J]. Front. Med., 2019, 13(4): 427-437.
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http://journal.hep.com.cn/fmd/EN/10.1007/s11684-018-0672-6
http://journal.hep.com.cn/fmd/EN/Y2019/V13/I4/427
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Zhen Wang
Jianhui Wu
Xiuyun Tian
Chunyi Hao
Treatment protocols Study Patient number Overall response rate PFS Grade 3–4 side effects/dose reduction due to side effects Characteristics
Imatinib Mace et al. (2002) [26] 2 100% 100% (FUT: 9 and 11 months) 0%
Heinrich et al. (2006) [28] 19 16% 6-M 53%; 12-M 37%; 36-M 12% Majority
Wcislo et al. (2007) [29] 1 100% 100% (FUT 24-M) 0%
Chugh et al. (2010) [30] 51 6% 6-M 84%; 12-M 66%; 36-M 58%; 60-M 52% 39%
Penel et al. (2011) [20] 40 10% 6-M 80%; 12-M 67%; 24-M 55%; 36-M 40% 45%
Kasper et al. (2017) [19] 38 19% 12-M 59%; 24-M 45% 13%
Total# 151 13% 33%
Nilotinib Kasper et al. (2017) [19] 8 3-M 88% 13% Had the potential to stabilize DF after the failure of imatinib treatment
Pazopanib Martin-Liberal et al. (2013) [39] 2 50% 100% * 50%
Szucs et al. (2017) [22] 8 38% 6-M 75%; 12-M 75%; 36-M 12.5% 13%
Total# 10 40% 20%
Sorafenib Gounder et al. (2011) [40] 26 25% 6-M 95% 15% Clinical improvement was noted within 2 weeks
Sunitinib Skubitz et al. (2009) [24] 1 100% 36-M 100% 0%
PF-903084014 Messersmith et al. (2015) [53] 7 71% Response time ranged from 1.74+ months to 24+ months 43%
Kummar et al. (2017) [21] 17 29% 94% (median FUT>25 months) 47%
Total# 24 42% 46%
Antihormone and/or NSAIDS Hansmann et al. (2003) [70] 27 37% 50%* 0% 30% of the cases developed ovarian cysts; 22% CR
Tanaka et al. (2008) [80] 1 100% 100%* 0%
Bocale et al. (2011) [81] 168 50% 80%* 0%
Quast et al. (2016) [68] 134 33% 85%* 0%
Total# 330 42% 0%
Anthracycline-based regimens Gega et al. [74] 7 100% Average PFS was 74 months (32.5–107.5) 43% 43% CR
Bertagnolli et al. [79] 10 90% 90%*
Camargo et al. [73] 35 37% 88%* 6%
Garbay et al. [76] 13 54% 100% (median PFS was 40.8 months)
Total# 65 55% 12%
Methotrexate and/or vinblastine Weisset al. [77] 15 60% 87%* 20%
Azzarelli et al. [78] 30 40% 6-M 96%; 12-M 92%; 36-M 80%; 60-M 67% 13%
Bertagnolli et al. [79] 4 25% 75%*
Camargo et al. [73] 22 27% 82%*
Garbay et al. [76] 27 15% 67% (median PFS 40.8 months)
Li et al. [82] 71 35% 24-M 80%; 36-M 68%; 60-M 36%
Total# 169 34% 16%
Tab.1  Summary of previous studies on systemic therapy for desmoid-type fibromatosis
Treatment protocols Overall response Grade 3–4 side effects
N Yes No P N Yes No P
Antihormone and/or NSAIDS 330 139 (42%) 191 (58%) 330 0 (0%) 330 (100%)
Imatinib 151 20 (13%) 131 (87%) <0.001 132 43 (33%) 89 (67%) <0.001
Nilotinib 8 8 1 (13%) 7 (87%) 0.024
Pazopanib 10 4 (40%) 6 (60%) 0.893 10 2 (20%) 8 (80%) 0.001
Sorafenib 26 7 (25%) 19 (75%) 0.129 26 4 (15%) 22 (85%) <0.001
PF-903084014 24 10 (42%) 14 (58%) 0.965 24 11 (46%) 13 (54%) <0.001
Tab.2  Comparison of the overall response rates and grade 3–4 side effect rates of antihormone and/or NSAIDS therapy and various targeted therapies
Fig.1  Overall response rates of systemic therapies for DF (case number>5). The median overall response rates and ranges are shown in the figure. Overall response rate, the rate of the cases that showed DF shrinkage. NSAIDS, nonsteroidal anti-inflammatory drugs. DF, desmoid-type fibromatosis.
Fig.2  Rates of grade 3–4 side effects of systemic therapies for DF (case number>5). The median overall response rates and ranges are shown in the figure. NSAIDS, nonsteroidal anti-inflammatory drugs. DF, desmoid-type fibromatosis.
Fig.3  Overall response and grade 3–4 side effects of various systemic therapies. NSAIDS, nonsteroidal anti-inflammatory drugs. Overall response rate, the rate of the cases that showed DF shrinkage. *Grade 3–4 side effects occurred in none of the patients that underwent antihormone and/or NSAIDS therapy. **The overall response rate of nilotinib is unavailable. NSAIDS, nonsteroidal anti-inflammatory drugs. DF, desmoid-type fibromatosis.
Treatment protocols Overall response Grade 3–4 side effects
N Yes No P N Yes No P
Anthracycline-based regimens 65 36 (55%) 29 (45%) 42 5 (12%) 37 (88%)
Imatinib 151 20 (13%) 131 (87%) 0.001 132 43 (33%) 89 (67%) 0.009
Nilotinib 8 8 1 (13%) 7 (87%) 0.962
Pazopanib 10 4 (40%) 6 (60%) 0.570 10 2 (20%) 8 (80%) 0.874
Sorafenib 26 7 (25%) 19 (75%) 0.014 26 4 (15%) 22 (85%) 0.965
PF-903084014 24 10 (42%) 14 (58%) 0.250 24 11 (46%) 13 (54%) 0.002
Tab.3  Comparison of the overall response rates and grade 3–4 side effect rates of anthracycline-based regimens and various targeted therapies
Treatment protocols Overall response Grade 3–4 side effects
N Yes No P N Yes No P
Methotrexate and/or vinblastine 169 57 (34%) 112 (66%) 45 7 (16%) 38 (84%)
Imatinib 151 20 (13%) 131 (87%) <0.001 132 43 (33%) 89 (67%) 0.029
Nilotinib 8 8 1 (13%) 7 (87%) 1.000
Pazopanib 10 4 (40%) 6 (60%) 0.950 10 2 (20%) 8 (80%) 1.000
Sorafenib 26 7 (25%) 19 (75%) 0.492 26 4 (15%) 22 (85%) 1.000
PF-903084014 24 10 (42%) 14 (58%) 0.445 24 11 (46%) 13 (54%) 0.006
Tab.4  Comparison of the overall response rates and grade 3–4 side effect rates of methotrexate and/or vinblastine regimens and various targeted therapies
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