Department of general surgery, Shanghai Children's Medical Center (National Children's Medical Center-Shanghai), Shanghai Jiaotong University School of Medicine, Shanghai, China.
Clinical Research Institute, Shanghai Jiao Tong University School of Medicine, Shanghai, 200025, China.
J Pediatr Surg. 2020 Nov;55(11):2454-2458. doi: 10.1016/j.jpedsurg.2019.12.021. Epub 2020 Jan 29.
Lymphatic malformations are common congenital vascular lesions. Neither surgical resection nor other surgical treatments have been found to be effective for invasive cases. Recent research has suggested that sirolimus is effective in treating complex lymphatic malformations (LMs). We aimed to evaluate the effectiveness and safety of oral sirolimus for children living with LMs in our hospital.
Fifty-six cases of complex LMs treated with sirolimus were collected from Shanghai Children's Medical Centre between June 2016 and March 2019. All cases were confirmed either by pathology (44) or enhanced MRI (12). Following informed consent, sirolimus 0.8 mg/m bid was administered orally to participants and maintained at a trough concentration of 10-15 ng/ml. Children's ages at diagnosis were neonate to 16 years (mean 44.3 months). All children were followed up for 5 to 30 months, with a mean of 16.8 months.
During the follow-up period, blood, liver and kidney function as well as disseminated intravascular coagulation was regularly reviewed in all 56 children. Enhanced MRI was regularly performed to evaluate therapeutic effects. Total effective rate (complete response or partial response) of LMs was 89.3% (50/56). No serious adverse reactions were found.
This study suggests that sirolimus is effective and tolerable for decreasing lesions in children with complex LMs, leading to fewer and more tolerable side effects. There is no need to pursue an excision rate to reduce unnecessary operative complications since adjuvant sirolimus therapy modifies the complex LMs clinical appearance and alleviates their symptoms.
Clinical research.
Level IV.
淋巴管畸形是常见的先天性血管病变。手术切除或其他手术治疗均不适用于侵袭性病例。最近的研究表明，西罗莫司在治疗复杂淋巴管畸形（LM）方面有效。我们旨在评估西罗莫司口服治疗我院 LM 患儿的有效性和安全性。
2016 年 6 月至 2019 年 3 月，上海儿童医学中心共收集了 56 例接受西罗莫司治疗的复杂 LM 病例。所有病例均经病理（44 例）或增强 MRI（12 例）证实。在获得知情同意后，患儿口服西罗莫司 0.8mg/m2，bid，维持谷浓度 10-15ng/ml。患儿确诊时的年龄为新生儿至 16 岁（平均 44.3 个月）。所有患儿的随访时间为 5 至 30 个月，平均 16.8 个月。
在随访期间，对所有 56 例患儿定期检查了血液、肝肾功能及弥漫性血管内凝血情况。定期行增强 MRI 以评估治疗效果。LM 的总有效率（完全缓解或部分缓解）为 89.3%（50/56）。未发现严重不良反应。
本研究表明，西罗莫司可有效且耐受地减少复杂 LM 患儿的病变，从而减少且可耐受更多的副作用。由于辅助西罗莫司治疗改变了复杂 LM 的临床外观并缓解了其症状，因此无需追求切除率以减少不必要的手术并发症。
临床研究。
IV 级。
