Zhou Jiangyuan, Yang Kaiying, Chen Siyuan, Ji Yi
Division of Oncology, Department of Pediatric Surgery, West China Hospital of Sichuan University, #37# Guo-Xue-Xiang, Chengdu, 610041, China.
Pediatric Intensive Care Unit, Department of Critical Care Medicine, West China Hospital of Sichuan University, #37# Guo-Xue-Xiang, Chengdu, 610041, China.
Orphanet J Rare Dis. 2021 Jun 8;16(1):260. doi: 10.1186/s13023-021-01893-3.
Kaposiform lymphangiomatosis (KLA), which is a new subtype of generalized lymphatic anomaly, is a rare disease with a poor prognosis. Currently, there is no standard treatment due to the poor understanding of KLA. Sirolimus, which is an inhibitor of mammalian target of rapamycin, has been shown to have promising potential in the treatment of complicated vascular anomalies. The aim of this study was to introduce the use of sirolimus for the treatment of KLA and to highlight the challenges of managing this refractory disease.
We reported seven patients with KLA who received sirolimus therapy in our center. Combined with previously reported cases, 58.3% achieved a partial response, 25.0% had stable disease, and 16.7% experienced disease progression. No severe sirolimus-related adverse events occurred during treatment.
This study suggests that sirolimus is currently an option for the treatment of KLA, and it is hoped that more specific therapies will be developed in the future. Rapid advances in basic science and clinical practice may facilitate the development of important new treatments for KLA.
卡波西样淋巴管瘤病（KLA）是一种广义淋巴管畸形的新亚型，是一种预后较差的罕见疾病。目前，由于对KLA了解不足，尚无标准治疗方法。西罗莫司是一种哺乳动物雷帕霉素靶蛋白抑制剂，已显示出在治疗复杂血管畸形方面具有潜在的应用前景。本研究的目的是介绍西罗莫司在KLA治疗中的应用，并强调管理这种难治性疾病的挑战。
我们报告了7例在我们中心接受西罗莫司治疗的KLA患者。结合先前报道的病例，58.3%的患者获得部分缓解，25.0%的患者病情稳定，16.7%的患者病情进展。治疗期间未发生严重的西罗莫司相关不良事件。
本研究表明，西罗莫司目前是KLA治疗的一种选择，希望未来能开发出更具特异性的治疗方法。基础科学和临床实践的快速发展可能有助于开发针对KLA的重要新疗法。
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