Department of Pediatrics, Samsung Medical Center, Sungkyunkwan University School of Medicine, 81 Irwon-ro, Gangnam-gu, Seoul, 06351, South Korea.
BMC Pediatr. 2021 Jan 7;21(1):21. doi: 10.1186/s12887-020-02447-5.
Intestinal lymphangiectasia is a rare disease. Thus, prospective studies are impossible, and therapy is still controversial. Several medicines are suggested for treatment but there are no existing indications for drug choice and treatment guidelines. We aimed to introduce the action mechanism of each drug and treatment overview in a single-center experience and a review of the literature on second-line therapy for primary intestinal lymphangiectasia.
Children under 18 years old diagnosed with intestinal lymphangiectasia from June 2000 to June 2020 were included and retrospectively reviewed in the study. Capsule endoscopy, MR lymphangiography, or whole-body MRI for investigating the extent of abnormal lymphatic vessels in addition to endoscopy and biopsy were conducted. The individual treatment approaches depended upon the lymphangiectasis locations involved.
Only one patient showed a response to dietary therapy. One patient was successfully cured after two therapeutic lymphatic embolization. Octreotide was tried for two patients who had extensive lymphangiectasis. Lymphangiectasis recurred when octreotide was used for 3 months in one patient, and there was no effect in the other patient. Sirolimus was tried for four patients. Two of them had abnormal lymphatic lesions only in the intestine, and the others had extensive lymphangiectasis. The former group showed clinical improvement after 3-4 months of sirolimus treatment, whereas the latter group showed clinical improvement only after 1 month of sirolimus treatment.
Surgery or embolization is a potential therapeutic option for patients with focal abnormal lymphatic lesions. Octreotide is not an optimal choice for patients with extensive lymphangiectasis. Sirolimus is an effective and safe drug and can be the first drug of choice for patients with extensive lymphangiectasis.
肠淋巴管扩张症是一种罕见疾病。因此，前瞻性研究是不可能的，治疗仍然存在争议。有几种药物被建议用于治疗，但没有现有的药物选择和治疗指南。我们旨在介绍一种单中心经验中的每种药物的作用机制和治疗概述，并对原发性肠淋巴管扩张症的二线治疗的文献进行综述。
本研究纳入了 2000 年 6 月至 2020 年 6 月期间在我院诊断为肠淋巴管扩张症的 18 岁以下儿童，并进行回顾性研究。除了内镜和活检外，还进行胶囊内镜、MR 淋巴管造影或全身 MRI 以调查异常淋巴管的范围。个体治疗方法取决于涉及的淋巴管扩张部位。
仅 1 例患者对饮食治疗有反应。1 例患者经两次治疗性淋巴栓塞后成功治愈。2 例广泛淋巴管扩张患者尝试使用奥曲肽。1 例患者使用奥曲肽 3 个月后淋巴管扩张复发，另 1 例患者无效果。4 例患者尝试使用西罗莫司。其中 2 例患者仅肠道存在异常淋巴管病变，另 2 例患者广泛淋巴管扩张。前者组在使用西罗莫司治疗 3-4 个月后显示出临床改善，而后者组仅在使用西罗莫司治疗 1 个月后显示出临床改善。
手术或栓塞是局灶性异常淋巴管病变患者的潜在治疗选择。奥曲肽不是广泛淋巴管扩张症患者的最佳选择。西罗莫司是一种有效且安全的药物，可作为广泛淋巴管扩张症患者的首选药物。
