Sun Yi, Gu Hao, Zhang Bin, Yang Xitao, Wen Mingzhe, Zhao Xiong, Yue Xiaojie, Zhang Hongyu, Fan Xindong, Xu Zigang, Su Lixin, Cai Ren
Department of Interventional Therapy, Multidisciplinary Team of Vascular Anomalies, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, PR China.
Department of Burn and Plastic Surgery, The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, PR China.
J Am Acad Dermatol. 2025 Jul 17. doi: 10.1016/j.jaad.2025.07.033.
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.
To assess the efficacy and safety of trametinib treatment for early-stage eAVMs over 12 months of treatment.
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.
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.
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.
