Department of Obstetrics and Gynecology, National Clinical Research Center for Obstetric & Gynecologic Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Fengzhi Feng, No. 1, Shuaifuyuan, Beijing, 100730, Wangfujing, China.
BMC Med. 2024 Mar 13;22(1):119. doi: 10.1186/s12916-024-03344-9.
Intravenous leiomyomatosis (IVL), pulmonary benign metastatic leiomyomatosis (PBML), and leiomyomatosis peritonealis disseminata (LPD) are leiomyomas with special growth patterns and high postoperative recurrence rates. We report the safety and efficacy of a pilot study of sirolimus in the treatment of recurrent IVL, PBML, and recurrent LPD.
This was a pilot study to evaluate the safety and efficacy of sirolimus in the treatment of leiomyomatosis (ClinicalTrials.gov identifier NCT03500367) conducted in China. Patients received oral sirolimus 2 mg once a day for a maximum of 60 months or until disease progression, intolerable toxicity, withdrawal of consent, or investigator decision to stop. The primary end point of this study was the objective response rate. Secondary end points included safety and tolerability, disease control rate, and progression-free survival.
A total of 15 patients with leiomyomatosis were included in the study, including five with recurrent IVL, eight with PBML and two with recurrent LPD. The median follow-up time was 15 months (range 6-54 months), nine patients (60%) had treatment-related adverse events (including all levels), and two patients had treatment-related grade 3 or 4 adverse events. The objective response rate was 20.0% (95% CI, 7.1-45.2%), and the disease control rate was 86.7% (95% CI, 62.1-96.3%). Partial response was achieved in three patients. The median response time in the three partial response patients was 33 months (range 29-36 months), and the sustained remission time of these three patients reached 0, 18, and 25 months, respectively.
Sirolimus was safe and effective in the treatment of recurrent IVL, PBML, and recurrent LPD.
ClinicalTrials.gov identifier NCT03500367. Registered on 18 April 2018.
静脉内平滑肌瘤病（IVL）、肺良性转移性平滑肌瘤病（PBML）和播散性腹膜平滑肌瘤病（LPD）是具有特殊生长模式且术后复发率较高的平滑肌瘤。我们报告了西罗莫司治疗复发性 IVL、PBML 和复发性 LPD 的初步研究的安全性和疗效。
这是一项在中国进行的评估西罗莫司治疗平滑肌瘤安全性和疗效的初步研究（ClinicalTrials.gov 标识符 NCT03500367）。患者接受口服西罗莫司 2mg，每天一次，最长 60 个月，或直至疾病进展、无法耐受毒性、患者撤回同意或研究者决定停止治疗。该研究的主要终点为客观缓解率。次要终点包括安全性和耐受性、疾病控制率和无进展生存期。
共有 15 例平滑肌瘤患者入组该研究，其中 5 例为复发性 IVL，8 例为 PBML，2 例为复发性 LPD。中位随访时间为 15 个月（范围 6-54 个月），9 例患者（60%）发生与治疗相关的不良事件（包括所有级别），2 例患者发生与治疗相关的 3 级或 4 级不良事件。客观缓解率为 20.0%（95%CI，7.1-45.2%），疾病控制率为 86.7%（95%CI，62.1-96.3%）。3 例患者获得部分缓解。3 例部分缓解患者的中位缓解时间为 33 个月（范围 29-36 个月），这 3 例患者的持续缓解时间分别为 0、18 和 25 个月。
西罗莫司治疗复发性 IVL、PBML 和复发性 LPD 安全有效。
ClinicalTrials.gov 标识符 NCT03500367。于 2018 年 4 月 18 日注册。
