Trametinib treatment for early-stage extracranial arteriovenous malformations: A multicenter, prospective, single-arm pilot study - 17/10/25
, Lixin Su, DDS, PhD a, ⁎ , Ren Cai, MD a, ⁎ Abstract |
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.
Le texte complet de cet article est disponible en PDF.Key words : arteriovenous malformation, chemotherapy, port-wine stains, targeted medicine, trametinib, vascular anomaly, vascular malformation
Abbreviations used : AE, AVM, CM-AVM, DSA, eAVM, PAV
Plan
| Drs Sun, Gu, and Zhang are co-first authors. |
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| Drs Xu, Su, and Cai are co-corresponding authors. |
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| Funding sources: Supported by the National Natural Science Foundation of China (Project No. 82304045; 82203948); China Postdoctoral Science Foundation Funded Project (Project No. 2022M720088, 2023T160422); Shanghai Sailing Program (Project No. 22YF1422400, 23YF1422200); Clinical Research Program of Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine (Project No. JYLJ202210); National Key Clinical Specialist; and Specialist League Development Foundation of Shanghai Ninth People's Hospital (Project No. ZKLM-介入1, ZKLM-介入2). |
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| IRB approval status: This study is approved by the committe on clinical Investigation of the Beijing Children's Hospital and Shanghai Ninth People's Hospital. |
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| Data availability statement: All supporting data of this article are included in the submitted manuscript. |
Vol 93 - N° 5
P. 1261-1267 - novembre 2025 Retour au numéro
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