Research progress on targeted drugs for vascular malformations
Vascular malformations, which mainly occur in the head and neck region, are a group of congenital disorders that cannot involute and dilate gradually as patients grow. Traditional therapeutic strategies for vascular malformations include laser therapy, sclerotherapy, interventional embolization, sur...
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| Format: | Article |
| Language: | zho |
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Editorial Department of Journal of Prevention and Treatment for Stomatological Diseases
2023-04-01
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| Series: | 口腔疾病防治 |
| Subjects: | |
| Online Access: | http://www.kqjbfz.com/CN/10.12016/j.issn.2096-1456.2023.04.010 |
| _version_ | 1797936770804875264 |
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| author | SHEN Yuchen WANG Deming FAN Xindong |
| author_facet | SHEN Yuchen WANG Deming FAN Xindong |
| author_sort | SHEN Yuchen |
| collection | DOAJ |
| description | Vascular malformations, which mainly occur in the head and neck region, are a group of congenital disorders that cannot involute and dilate gradually as patients grow. Traditional therapeutic strategies for vascular malformations include laser therapy, sclerotherapy, interventional embolization, surgical resection, etc. However, for some cases with a relatively larger range of lesions, traditional therapeutic strategies might fall short of the goals. With the development of molecular genetics, gene mutations are currently recognized as the root cause of the occurrence of vascular malformations. The progression of vascular malformation lesions is further promoted by the activation of related pathways. Low-flow vascular malformations mainly involve activation of the phosphatidylinositol 3-kinase (PI3K)/protein kinase B (AKT)/mammalian target of rapamycin (mTOR) pathway, whereas high-flow vascular malformations mainly involve activation of the rat sarcoma (RAS)/rapidly accelerated fibrosarcoma (RAF)/mitogen-activated protein kinase kinase (MAPKK)/extracellular-signal regulated protein kinase (ERK) pathway. Targeted drugs against relevant gene mutations and signaling pathways have also been applied in the treatment of vascular malformations, and previous studies have shown that the mTOR inhibitor rapamycin is effective and now widely used in the treatment of low-flow vascular malformations. The PI3K inhibitor alpelisib is also promising in the treatment of venous malformations, and the MAPKK inhibitor trametinib has shown good results in the treatment of arteriovenous malformations. Therefore, traditional therapies supplemented by targeted drugs may bring new breakthroughs to the treatment of vascular malformations. |
| first_indexed | 2024-04-10T18:34:28Z |
| format | Article |
| id | doaj.art-aed75c9d8a2f458d9115fb20eade17c1 |
| institution | Directory Open Access Journal |
| issn | 2096-1456 |
| language | zho |
| last_indexed | 2024-04-10T18:34:28Z |
| publishDate | 2023-04-01 |
| publisher | Editorial Department of Journal of Prevention and Treatment for Stomatological Diseases |
| record_format | Article |
| series | 口腔疾病防治 |
| spelling | doaj.art-aed75c9d8a2f458d9115fb20eade17c12023-02-02T02:17:22ZzhoEditorial Department of Journal of Prevention and Treatment for Stomatological Diseases口腔疾病防治2096-14562023-04-0131428428910.12016/j.issn.2096⁃1456.2023.04.010Research progress on targeted drugs for vascular malformationsSHEN Yuchen 0WANG Deming1 FAN Xindong2Department of Interventional Therapy, Shanghai Ninth People’s Hospital, Shanghai Jiao Tong University School of MedicineDepartment of Interventional Therapy, Shanghai Ninth People’s Hospital, Shanghai Jiao Tong University School of MedicineDepartment of Interventional Therapy, Shanghai Ninth People’s Hospital, Shanghai Jiao Tong University School of MedicineVascular malformations, which mainly occur in the head and neck region, are a group of congenital disorders that cannot involute and dilate gradually as patients grow. Traditional therapeutic strategies for vascular malformations include laser therapy, sclerotherapy, interventional embolization, surgical resection, etc. However, for some cases with a relatively larger range of lesions, traditional therapeutic strategies might fall short of the goals. With the development of molecular genetics, gene mutations are currently recognized as the root cause of the occurrence of vascular malformations. The progression of vascular malformation lesions is further promoted by the activation of related pathways. Low-flow vascular malformations mainly involve activation of the phosphatidylinositol 3-kinase (PI3K)/protein kinase B (AKT)/mammalian target of rapamycin (mTOR) pathway, whereas high-flow vascular malformations mainly involve activation of the rat sarcoma (RAS)/rapidly accelerated fibrosarcoma (RAF)/mitogen-activated protein kinase kinase (MAPKK)/extracellular-signal regulated protein kinase (ERK) pathway. Targeted drugs against relevant gene mutations and signaling pathways have also been applied in the treatment of vascular malformations, and previous studies have shown that the mTOR inhibitor rapamycin is effective and now widely used in the treatment of low-flow vascular malformations. The PI3K inhibitor alpelisib is also promising in the treatment of venous malformations, and the MAPKK inhibitor trametinib has shown good results in the treatment of arteriovenous malformations. Therefore, traditional therapies supplemented by targeted drugs may bring new breakthroughs to the treatment of vascular malformations.http://www.kqjbfz.com/CN/10.12016/j.issn.2096-1456.2023.04.010vascular malformationssignaling pathwaysmammalian target of rapamycin complex 1mitogen-activated protein kinase kinaserapamycintrametinibvascular endothelial growth factor |
| spellingShingle | SHEN Yuchen WANG Deming FAN Xindong Research progress on targeted drugs for vascular malformations 口腔疾病防治 vascular malformations signaling pathways mammalian target of rapamycin complex 1 mitogen-activated protein kinase kinase rapamycin trametinib vascular endothelial growth factor |
| title | Research progress on targeted drugs for vascular malformations |
| title_full | Research progress on targeted drugs for vascular malformations |
| title_fullStr | Research progress on targeted drugs for vascular malformations |
| title_full_unstemmed | Research progress on targeted drugs for vascular malformations |
| title_short | Research progress on targeted drugs for vascular malformations |
| title_sort | research progress on targeted drugs for vascular malformations |
| topic | vascular malformations signaling pathways mammalian target of rapamycin complex 1 mitogen-activated protein kinase kinase rapamycin trametinib vascular endothelial growth factor |
| url | http://www.kqjbfz.com/CN/10.12016/j.issn.2096-1456.2023.04.010 |
| work_keys_str_mv | AT shenyuchen researchprogressontargeteddrugsforvascularmalformations AT wangdeming researchprogressontargeteddrugsforvascularmalformations AT fanxindong researchprogressontargeteddrugsforvascularmalformations |