Ünlüsoy Aksu Aysel, Sari Sinan, Eğritaş Gürkan Ödül, Dalgiç Buket
Department of Pediatrics, Division of Pediatric Gastroenterology, Hepatology and Nutrition, Gazi University Faculty of Medicine, Ankara, Turkey.
J Pediatr Hematol Oncol. 2017 Mar;39(2):147-149. doi: 10.1097/MPH.0000000000000681.
Recently, sirolimus was demonstrated to be effective in treating vascular lesions and lessening the frequency of bleeding and secondary iron deficiency anemia. We present a child with blue rubber bleb nevus syndrome who had prolonged history of iron deficiency anemia secondary to unrecognized gastrointestinal bleeding. Treatment with propranolol, omeprazole and iron had failed. After 2.5 months of sirolimus therapy (trough levels 1 to 5 ng/mL), his hemoglobin concentration improved into the normal range and remained stable. Vascular malformations on both the patient's tongue and in the fundus of his stomach shrank within 5 months of the initiation of sirolimus. In gastrointestinal involvement of blue rubber bleb nevus syndrome sirolimus was found to be effective even in the tongue's vascular lesions.
最近，西罗莫司被证明在治疗血管病变以及减少出血频率和继发性缺铁性贫血方面有效。我们报告一名患有蓝色橡皮疱痣综合征的儿童，该患儿因未被识别的胃肠道出血而长期患有缺铁性贫血。使用普萘洛尔、奥美拉唑和铁剂治疗均失败。在接受2.5个月的西罗莫司治疗（谷浓度为1至5 ng/mL）后，他的血红蛋白浓度升至正常范围并保持稳定。该患者舌头和胃底的血管畸形在开始使用西罗莫司后的5个月内缩小。在蓝色橡皮疱痣综合征的胃肠道受累中，发现西罗莫司即使对舌头的血管病变也有效。
