Yi Sun, Hao Gu, Bin Zhang, Xitao Yang, Mingzhe Wen, Xiong Zhao, Xiaojie Yue, Hongyu Zhang, Xindong Fan, Zigang Xu, Lixin Su, Ren Cai
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引用次数: 0
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, skin color) and qualitative (DSA) 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.
期刊介绍:
The Journal of the American Academy of Dermatology (JAAD) is the official scientific publication of the American Academy of Dermatology (AAD). Its primary goal is to cater to the educational requirements of the dermatology community. Being the top journal in the field, JAAD publishes original articles that have undergone peer review. These articles primarily focus on clinical, investigative, and population-based studies related to dermatology. Another key area of emphasis is research on healthcare delivery and quality of care. JAAD also highlights high-quality, cost-effective, and innovative treatments within the field. In addition to this, the journal covers new diagnostic techniques and various other topics relevant to the prevention, diagnosis, and treatment of skin, hair, and nail disorders.