Cho Yu Jeong, Kwon Hyunhee, Ha Suhyeon, Kim Seong Chul, Kim Dae Yeon, Namgoong Jung-Man, Cho Min Jeng, Lee Ju Yeon, Jung Eunyoung, Nam So Hyun
Department of Surgery, Hanyang University Guri Hospital, Hanyang University School Medicine, Guri, Korea.
Department of Pediatric Surgery, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea.
Ann Surg Treat Res. 2024 Mar;106(3):125-132. doi: 10.4174/astr.2024.106.3.125. Epub 2024 Feb 22.
Sirolimus has emerged as a safe and effective treatment for complicated lymphatic malformations (LMs). We aim to prove the effectiveness and safety of sirolimus as a therapeutic option for patients with complicated LMs.
Fifty-eight patients with complicated LMs treated with sirolimus for at least 6 months at multicenter between July 2018 and January 2023 were enrolled. All patients were administered oral sirolimus starting at 0.8 mg/m every 12 hours, with target serum concentration levels of 8-15 ng/mL. Evaluation for clinical symptoms and LMs volume on MRI were reviewed to assess treatment response and toxicities. Evaluation of disease response was divided into 3 values: complete response, partial response (significant, moderate, and modest), and progressive disease.
The median age at the initiation of sirolimus treatment was 6.0 years (range, 1 month-26.7 years). The median duration of treatment was 2.0 years (range, 6 months-4.4 years). The most common lesions were head and neck (25 of 58, 43.1%). Forty-six patients (79.3%) demonstrated a reduction in LMs volume on MRI or improvement of clinical symptoms including 2 complete responses. The young age group and the patients who underwent few prior therapies showed better responses. None of the patients had toxicities attributable to sirolimus with a Common Terminology Criteria for Adverse Events grade of ≥3.
Oral sirolimus treatment brought a successful outcome without severe adverse effects. It could be the first-line therapy, especially for the young age group of complicated LMs, and an additional option for refractory lesions that did not respond to conventional treatment.
西罗莫司已成为治疗复杂淋巴管畸形（LM）的一种安全有效的方法。我们旨在证明西罗莫司作为复杂LM患者治疗选择的有效性和安全性。
纳入2018年7月至2023年1月期间在多中心接受西罗莫司治疗至少6个月的58例复杂LM患者。所有患者均口服西罗莫司，起始剂量为0.8mg/m²，每12小时一次，目标血清浓度水平为8 - 15ng/mL。回顾对临床症状和MRI上LM体积的评估，以评估治疗反应和毒性。疾病反应评估分为3个值：完全缓解、部分缓解（显著、中度和轻度）和疾病进展。
开始西罗莫司治疗的中位年龄为6.0岁（范围1个月 - 26.7岁）。中位治疗持续时间为2.0年（范围6个月 - 4.4年）。最常见的病变部位是头颈部（58例中的25例，43.1%）。46例患者（79.3%）在MRI上显示LM体积减小或临床症状改善，包括2例完全缓解。年龄较小的组和既往接受治疗较少的患者显示出更好的反应。没有患者出现不良事件通用术语标准≥3级的西罗莫司相关毒性。
口服西罗莫司治疗取得了成功的结果，且无严重不良反应。它可能是一线治疗方法，特别是对于复杂LM的年轻患者群体，也是对传统治疗无反应的难治性病变的另一种选择。
