婴幼儿血管瘤可致严重并发症及临床治疗药物的研究进展

Infantile Hemangioma Can Cause Serious Complications and the Research Progress of Clinical Treatment Drugs

摘要: 婴幼儿血管瘤(Infantile Hemangioma, IH)是婴幼儿期最常见的良性血管肿瘤，发病率达5%~12%，可因瘤体位置或体积引发气道阻塞、肝血管瘤相关并发症及溃疡性病变等危及生命的后果。其发病机制涉及VEGF、Notch、PI3K/AKT/mTOR等多条信号通路的异常调控。近年来药物治疗进展显著，如β受体阻滞剂普萘洛尔因其高效性和安全性成为IH全身治疗的首选药物；糖皮质激素通过拮抗VEGF-A抑制血管生成；干扰素α-2a对难治性IH有效；贝伐单抗通过靶向VEGF抑制内皮细胞增殖，在小鼠模型中展现出治疗潜力；抗真菌药物伊曲康唑通过抑制Hedgehog通路及PDGF-D/PI3K/AKT/mTOR信号，已在特殊病例中成功应用并实现瘤体消退，体现了老药新用的策略；而硬化剂注射常用于局限性血管瘤治疗。但不同药物在实际治疗实践中仍具有局限性，如糖皮质激素因其免疫抑制等副作用应用受限，干扰素可能引发血液系统不良反应，硬化剂存在组织坏死风险等。综上所述，普萘洛尔仍是IH治疗的核心，而伊曲康唑等新型疗法的探索为复杂病例提供了更多选择，未来需进一步优化用药策略并开展多中心临床研究验证长期疗效与安全性。

Abstract: Infantile hemangioma (IH) is the most common benign vascular tumor in infancy, with an incidence rate as high as 5%~12%. It can lead to life-threatening complications such as airway obstruction, complications associated with hepatic hemangiomas, and ulcerative lesions due to the location or size of the tumor. The pathogenesis of IH involves abnormal regulation of multiple signaling pathways, including VEGF, Notch, and PI3K/AKT/mTOR. In recent years, significant progress has been made in the pharmacological treatment of IH. For instance, the beta-blocker propranolol has become the first-line treatment for systemic IH due to its high efficacy and safety profile. Corticosteroids, which antagonize VEGF-A to inhibit angiogenesis, have long been used in the treatment of IH. Interferon α-2a has proven effective for refractory IH cases. Bevacizumab, a monoclonal antibody targeting VEGF, has demonstrated therapeutic potential in mouse models by inhibiting endothelial cell proliferation. The antifungal agent itraconazole, which inhibits the Hedgehog pathway and the PDGF-D/PI3K/AKT/mTOR signaling, has successfully induced tumor regression in select cases, exemplifying the strategy of repurposing existing drugs. Sclerotherapy is commonly used for localized hemangiomas. However, each of these treatments has its limitations in clinical practice. Corticosteroids are restricted in use due to side effects like immunosuppression. Interferon may cause hematologic adverse reactions. Sclerotherapy carries the risk of tissue necrosis. In summary, propranolol remains the cornerstone of IH treatment. The exploration of novel therapies such as itraconazole offers additional options for complex cases. Future efforts should focus on optimizing treatment strategies and conducting multicenter clinical studies to verify long-term efficacy and safety.