Cîrstoveanu Cătălin, Bizubac Ana Mihaela, Mustea Cristina, Manolache Ștefan, Istrate-Bârzan Alexandra, Sfrijan Doinița, Marcu Veronica, Iozsa Dan-Alexandru, Spătaru Radu-Iulian
Neonatal Intensive Care Unit, 'Marie S. Curie' Emergency Clinical Hospital for Children, 077120 Bucharest, Romania.
Department of Pediatrics, 'Carol Davila' University of Medicine and Pharmacy, 050474 Bucharest, Romania.
Exp Ther Med. 2021 Oct;22(4):1097. doi: 10.3892/etm.2021.10531. Epub 2021 Aug 2.
We present a series of four newborns diagnosed with complicated congenital vascular anomalies, with different localization: Congenital lymphatic malformation (CLM) on the left hemithorax extending on the left upper limb; congenital hepatic hemangioma (CHH) with important complications in the first 7 weeks of life; Kaposiform hemangioendothelioma (KHE) of the left lower limb complicated with Kasabach Merritt phenomenon (KMM) and most probable diffuse capillary malformation with overgrowth (DCMO). All patients were treated with combined antiproliferative therapy with sirolimus and propranolol. The initial dose of sirolimus was 0.45-0.5 mg/m with doses adjusted according to plasmatic levels. Therapeutic intervals of sirolimus were considered at plasmatic levels of 7-12 ng/ml. Our aim was to use the lowest therapeutic dose in order to avoid possible side effects. Propranolol was initiated in doses of 0.5-1.0 mg/kg/day and was increased up to 3.0 mg/kg/day depending on tolerability. Following two months, every patient showed a marked reduction in the size of the mass, improvement in overall appearance or even calcification in the liver vascular tumor. No patient showed life threatening side effects to the treatment. Hypertriglyceridemia was the only side effect noted in all patients. This is in accordance with several international studies, which try to demonstrate the importance of sirolimus in neonatal vascular malformations in monotherapy or combined with different drugs.
我们报告了一系列4例被诊断为复杂先天性血管异常的新生儿,病变部位各异:左侧胸腔先天性淋巴管畸形(CLM)并累及左上肢;先天性肝血管瘤(CHH)在出生后7周内出现严重并发症;左下肢卡波西样血管内皮瘤(KHE)并伴有卡萨巴赫-梅里特现象(KMM),以及最可能的弥漫性毛细血管畸形伴过度生长(DCMO)。所有患者均接受了西罗莫司和普萘洛尔联合抗增殖治疗。西罗莫司初始剂量为0.45 - 0.5 mg/m²,根据血药浓度调整剂量。当血药浓度为7 - 12 ng/ml时考虑西罗莫司的治疗间隔。我们的目标是使用最低治疗剂量以避免可能的副作用。普萘洛尔起始剂量为0.5 - 1.0 mg/kg/天,根据耐受性可增至3.0 mg/kg/天。两个月后,每位患者的肿块大小均显著减小,整体外观改善,甚至肝脏血管肿瘤出现钙化。没有患者出现危及生命的治疗副作用。高甘油三酯血症是所有患者中唯一观察到的副作用。这与几项国际研究一致,这些研究试图证明西罗莫司在新生儿血管畸形单药治疗或与不同药物联合治疗中的重要性。