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Targeted therapy of desmoid-type fibromatosis: mechanism, current situation, and future prospects
Zhen Wang1, Jianhui Wu1, Xiuyun Tian1
1Key Laboratory of Carcinogenesis and Translational Research, Ministry of Education, Department of Hepato-Pancreato-Biliary Surgery, Peking University Cancer Hospital and Institute, Beijing, 100142, China.
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.
Insights
Targeted therapy for desmoid-type fibromatosis (DF) shows limited advantages over other treatments. Further research is needed to clarify its role, dosage, and mechanisms in managing this rare fibroblastic tumor.
Area of Science:
- Oncology
- Dermatology
- Medical Research
Background:
- Desmoid-type fibromatosis (DF) is a rare, locally infiltrative fibroblastic tumor.
- Current targeted therapies, including tyrosine kinase and γ-secretase inhibitors, lack robust evidence due to small retrospective studies.
Purpose of the Study:
- To review existing literature on the mechanism, efficacy, and safety of targeted therapies for DF.
- To compare targeted therapy with other systemic treatments for DF.
Main Methods:
- Systematic review of previous studies on DF targeted therapy.
- Comparative analysis of efficacy and safety data between targeted and other systemic therapies.
Main Results:
- Targeted therapy for DF does not demonstrate significant advantages in efficacy or safety compared to other medical treatments.
- Targeted therapy is typically reserved for cases refractory to antihormonal therapies, NSAIDs, and chemotherapy.
Conclusions:
- Current evidence does not support substantial benefits of targeted therapy over existing treatments for DF.
- Further investigation into the mechanisms, indications, and optimal dosing of targeted DF therapies is essential.
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