Giménez-Aleixandre C, Méndez-Aguirre N A, Martínez-Menchón T, Girón Vallejo Ó, Fernández-Ibieta M, Ferri-Ñíguez B, Villamil V, Sánchez-Sánchez Á, Montoya-Rangel C A, Hernández-Bermejo J P
Servicio de Cirugía Pediátrica. Hospital Clínico Universitario Virgen de la Arrixaca. Murcia.
Servicio de Dermatología. Hospital Clínico Universitario Virgen de la Arrixaca. Murcia.
Cir Pediatr. 2019 Jan 21;32(1):28-33.
mTOR inhibitors are being used to treat complex vascular anomalies (VA) without response to conventional treatments. We report our results in pediatric patients treated with sirolimus.
Retrospective review of patients treated with sirolimus between 2014 and 2017, analyzing vascular anomaly type, treatment response and complications. Treatment protocol included patients with complex vascular anomalies, after signing the informed consent. The initial dose was 0.8 mg/m2/12 h, verifying plasmatic levels. Favorable response was defined both in clinical and radiological terms.
Sirolimus was employed in nine patients, median age 14 months old (1 month-14 years), 66% girls. Five complex micro-cystic lymphatic malformations (LM), one multifocal lynphangioendotheliomatosis with thrombocytopenia, one kaposiform lymphangiomatosis, one lymphatic-venous malformation and one kaposiform hemangioendothelioma (KHE) were treated. Median treatment was 4 months (IQR 2-18 months). Resolution or improvement was objectified in four patients (44%). KHE patient presented complete resolution after two months of treatment. Two patients with micro-cystic LM and the one with lymphatic-venous malformation improved after a median treatment of three months. Two patients presented rebound effect after discontinuing treatment. Three patients had hypertransaminasemia and hypercholesterolemia without requiring medical treatment.
Sirolimus presented mild effects for treatment of complex VA in our study, but was highly resolutive at KHE.
雷帕霉素抑制剂正被用于治疗对传统治疗无反应的复杂血管异常（VA）。我们报告了使用西罗莫司治疗儿科患者的结果。
回顾性分析2014年至2017年间接受西罗莫司治疗的患者，分析血管异常类型、治疗反应和并发症。治疗方案包括签署知情同意书后的复杂血管异常患者。初始剂量为0.8mg/m²/12小时，并检测血浆水平。从临床和影像学角度定义良好反应。
9例患者使用了西罗莫司，中位年龄14个月（1个月至14岁），66%为女孩。治疗了5例复杂的微囊性淋巴管瘤（LM）、1例伴有血小板减少的多灶性淋巴管内皮瘤病、1例卡波西样淋巴管瘤病、1例淋巴管静脉畸形和1例卡波西样血管内皮瘤（KHE）。中位治疗时间为4个月（四分位间距2至18个月）。4例患者（44%）病情得到缓解或改善。KHE患者在治疗2个月后完全缓解。2例微囊性LM患者和1例淋巴管静脉畸形患者在中位治疗3个月后病情改善。2例患者在停药后出现反弹效应。3例患者出现高转氨酶血症和高胆固醇血症，但无需药物治疗。
在我们的研究中，西罗莫司对复杂VA的治疗效果较轻，但对KHE具有高度的缓解作用。
