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Updated: Sep 14, 2025
Comprehensive Endovascular and Open Surgical Management of Cerebral Arteriovenous Malformations
Published on: October 20, 2017
Trametinib treatment for early-stage extracranial arteriovenous malformations: A multicenter, prospective, single-arm
1Department of Interventional Therapy, Multidisciplinary Team of Vascular Anomalies, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, PR China.
Background:
Extracranial arteriovenous malformations (eAVMs) are progressive vascular anomalies for which conventional therapies are rarely curative. Few standard therapies have been developed for early-stage eAVMs. In particular, the efficacy and safety of trametinib for treating early-stage childhood eAVM are unclear.
Objective:
To assess the efficacy and safety of trametinib treatment for early-stage eAVMs over 12 months of treatment.
Method:
Patients with early-stage eAVM received trametinib for at least 12 months. All participants underwent clinical and radiological follow-up, including quantitative (skin temperature, peak arterial velocity, and skin color) and qualitative (digital subtraction angiography) assessments.
Results:
Therapeutic outcomes of the 26 patients included the following clinical responses: an average skin lesion blanching rate of 53.5%, a decrease in skin temperature of nearly 1 °C, a decrease in peak arterial blood flow velocity of 74%, and 50% of the patients achieving more than 50% devascularization. Patients with somatic KRAS/MAP2K1 mutations had more significant clinical improvement than patients with RASA1/EPHB4 germline mutations did. Trametinib was well tolerated, with most adverse events assessed as grade 1/2.
Conclusion:
This pilot study demonstrates short-term efficacy and safety in the treatment of early-stage eAVMs utilizing trametinib. Further study is needed to evaluate long-term outcomes.
Insights
Trametinib shows short-term efficacy and safety for treating early-stage childhood Extracranial arteriovenous malformations (eAVMs). This pilot study indicates positive clinical responses and good tolerability, warranting further investigation into long-term outcomes.
Area of Science:
- Vascular Medicine
- Pediatric Oncology
- Pharmacology
Background:
- Extracranial arteriovenous malformations (eAVMs) are progressive vascular anomalies with limited curative conventional therapies.
- Effective treatments for early-stage eAVMs, particularly in children, remain scarce.
- The safety and efficacy of trametinib for early-stage childhood eAVMs are not well-established.
Purpose of the Study:
- To evaluate the efficacy and safety of trametinib in treating early-stage eAVMs.
- To assess treatment outcomes over a 12-month period.
Main Methods:
- A pilot study involving patients with early-stage eAVM treated with trametinib for at least 12 months.
- Clinical and radiological follow-up including quantitative (skin temperature, peak arterial velocity, skin color) and qualitative (digital subtraction angiography) assessments.
Main Results:
- Significant clinical improvements observed: 53.5% average skin lesion blanching, ~1 °C decrease in skin temperature, 74% reduction in peak arterial blood flow velocity, and 50% achieving >50% devascularization.
- Patients with KRAS/MAP2K1 mutations showed greater improvement than those with RASA1/EPHB4 mutations.
- Trametinib was well-tolerated, with most adverse events being grade 1/2.
Conclusions:
- Trametinib demonstrates short-term efficacy and safety for treating early-stage eAVMs.
- Further research is necessary to determine the long-term effectiveness and safety profile of trametinib for eAVMs.
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