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.
■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。