PDF(Pubmed)
Abstract：
UNASSIGNED: 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.
UNASSIGNED: 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/m2 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.
UNASSIGNED: 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.
UNASSIGNED: 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.
UNASSIGNED: 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/m2 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.
UNASSIGNED: 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.
UNASSIGNED: 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.
摘要：
■西罗莫司已成为复杂的淋巴畸形（LM）的安全有效的治疗方法。我们的目标是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。纳入了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，每12小时服用0.8mg/m2，目标血清浓度水平为8-15ng/mL。对MRI上的临床症状和LMs体积进行评估，以评估治疗反应和毒性。对疾病反应的评价分为3个值：完全反应，部分反应(显著，中度,和谦虚)，和进行性疾病。
■西罗莫司治疗开始时的中位年龄为6.0岁（范围，1个月-26.7年）。中位治疗时间为2.0年（范围，6个月-4.4年）。最常见的病变是头颈部（58个中的25个，占43.1％）。46例患者（79.3％）在MRI上表现出LM体积减少或临床症状改善，包括2例完全缓解。年轻年龄组和之前接受过很少治疗的患者表现出更好的反应。没有患者具有可归因于西罗莫司的毒性，不良事件的通用术语标准等级≥3。
■口服西罗莫司治疗带来了成功的结果，没有严重的不良反应。这可能是一线治疗，特别是对于复杂LMs的年轻年龄组，以及对常规治疗无反应的难治性病变的额外选择。
■西罗莫司治疗开始时的中位年龄为6.0岁（范围，1个月-26.7年）。中位治疗时间为2.0年（范围，6个月-4.4年）。最常见的病变是头颈部（58个中的25个，占43.1％）。46例患者（79.3％）在MRI上表现出LM体积减少或临床症状改善，包括2例完全缓解。年轻年龄组和之前接受过很少治疗的患者表现出更好的反应。没有患者具有可归因于西罗莫司的毒性，不良事件的通用术语标准等级≥3。
■口服西罗莫司治疗带来了成功的结果，没有严重的不良反应。这可能是一线治疗，特别是对于复杂LMs的年轻年龄组，以及对常规治疗无反应的难治性病变的额外选择。
