Li Lin
Department of Oncology, Zibo Municipal Hospital, Zibo, ShanDong, China.
Front Pharmacol. 2025 Jan 7;15:1454418. doi: 10.3389/fphar.2024.1454418. eCollection 2024.
Neurofibromatosis type 1 (NF1) is a rare neurogenetic disorder with limited treatment options. Selumetinib, a MEK1/2 inhibitor, has emerged as a promising therapy for inoperable NF1-related plexiform neurofibromas.
Our retrospective pharmacovigilance study utilized the FDA Adverse Event Reporting System (FAERS) to comprehensively evaluate Selumetinib's safety profile in real-world settings. Data from the third quarter of 2020 to the fourth quarter of 2023 were analyzed, identifying 498 adverse event reports with Selumetinib as the primary suspect drug.
Statistical analysis revealed disproportionate signals for skin and subcutaneous tissue disorders, eye disorders, and various congenital, familial, and genetic disorders. The most common adverse events were elevated blood creatine phosphokinase, rash, and acneiform dermatitis. Notably, several adverse events, including rhabdomyolysis, were identified but not listed on the Selumetinib product label, based on a comparison with the FDA drug labeling.
The study underscores the importance of early detection and management of adverse reactions associated with Selumetinib, particularly within the initial month of treatment. These findings provide valuable insights for clinicians and regulators to ensure the safe and effective use of Selumetinib in NF1 patients.
1型神经纤维瘤病（NF1）是一种罕见的神经遗传性疾病，治疗选择有限。司美替尼，一种MEK1/2抑制剂，已成为治疗无法手术的NF1相关丛状神经纤维瘤的一种有前景的疗法。
我们的回顾性药物警戒研究利用美国食品药品监督管理局不良事件报告系统（FAERS）全面评估司美替尼在现实环境中的安全性。分析了2020年第三季度至2023年第四季度的数据，确定了498份以司美替尼为主要可疑药物的不良事件报告。
统计分析显示，皮肤和皮下组织疾病、眼部疾病以及各种先天性、家族性和遗传性疾病的信号不成比例。最常见的不良事件是血肌酸磷酸激酶升高、皮疹和痤疮样皮炎。值得注意的是，与美国食品药品监督管理局药品标签相比，确定了包括横纹肌溶解在内的几种不良事件，但未列在司美替尼产品标签上。
该研究强调了早期发现和处理与司美替尼相关不良反应的重要性，尤其是在治疗的最初一个月内。这些发现为临床医生和监管机构确保司美替尼在NF1患者中的安全有效使用提供了有价值的见解。
