Efficacy and Safety of Different Concentrations of Bleomycin in the Sclerotherapy of Lymphatic Malformations for Pediatric Patients
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 1
- 主要终点
- Changes of Volume
研究概览
简要总结
Bleomycin has nowadays been more and more widely used in the sclerotherapy of LMs, which has been proven to be primarily dose dependent. The investigators aim to compare the efficacy and safety of different concentrations of Bleomycin in the sclerotherapy of LMs for pediatric patients.
详细描述
Lymphatic malformations (LMs) are vascular anomalies that arise from abnormal embryonic development of the lymphatic system and might present as dilated lymphatic channels or cysts lined by lymphatic endothelial cells. With an estimated incidence of approximately 1/4000-1/2000, LMs can occur at any site in the lymphatic system, in which head, neck and axilla were mostly detected and have been reported to account for over 75%. Based on the location and size of the lesion and the extent of involvement, LMs may be asymptomatic with incidental detection, or chronic abdominal pain and distension due to their compression of surrounding structures, or critical and even fatal secondary to their volvulus, hemorrhage, infection and rupture. Surgical excision is a definitive treatment for LMs, while it may be difficult at times because of the infiltrative nature of the lesions, leading to a high incidence of complications like vital organ injuries, nerve injuries, bleeding, infection scar formation, and recurrences. Sclerotherapy is a simpler alternative to tedious surgical excision treatment for LMs and avoids the complications related to surgery. As an anticancer drug extracted from Streptomyces verticillus, Bleomycin has been more and more widely used in the sclerotherapy of LMs for pediatric patients, which has been proven to be primarily dose dependent. However, the optimum concentration of Bleomycin in the sclerotherapy of LMs for pediatric patients has not been strictly validated, due to the lack of high-quality RCT studies. The investigators aim to compare the efficacy and safety of different concentrations of Bleomycin in the sclerotherapy of LMs for pediatric patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Outcomes Assessor)
入排标准
- 年龄范围
- — 至 14 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female participants less than 14 years of age at the time of informed consent/assent form was signed.
- •Participants whose parents have voluntarily given written consent and participants who provided assent (if applicable) after the study has been explained to them.
- •Participants with LMs of all sites measured and confirmed via imaging at screening, with rapid progression, resluting in obvious symptoms or dysfunction, which could not be radically resected and could be treated by sclerotherapy.
排除标准
- •Penicillin allergy.
- •Vascular tumors or combined vascular malformations.
- •Participants who may have had surgical or sclerotherapy treatment by other hardeners.
- •LMs growing slowly, without obvious symptoms or dysfunction, which does not need to be treated prematurely.
研究组 & 干预措施
Low-dose Concentrations (1mg/ml) of Bleomycin
In this arm, patients with lymphatic malformations were treated by intracapsular injection with low-dose concentrations (1mg/ml) of Bleomycin.
干预措施: Bleomycin (Drug)
High-dose Concentrations (2mg/ml) of Bleomycin
In this arm, patients with lymphatic malformations were treated by intracapsular injection with high-dose concentrations (2mg/ml) of Bleomycin.
干预措施: Bleomycin (Drug)
结局指标
主要结局
Changes of Volume
时间窗: 3 to 6 months post-therapy
Changes of Volume is defined as follows: a complete (90%-100% reduction in LMs volume), substantial (60%-89% reduction in LMs volume), intermediate (20%-59% reduction in LMs volume), or no (\< 20% reduction in LMs volume) response 3 to 6 months post-therapy as assessed by imaging.
次要结局
- Score of Pain(3 to 6 months post-therapy)
- Global Efficacy(3 to 6 months post-therapy)
- Score of Quality of Life(3 to 6 months post-therapy)
- Number of Participants with Efficacy(3 to 6 months post-therapy)
- Number of Participants with Safety(3 to 6 months post-therapy)
研究者
Yi Ji
Clinical Professor
West China Hospital