Successful Treatment of Netherton Syndrome With Combination of Secukinumab and Dupilumab.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- overal survival Kaplan-Mayer method
研究概览
简要总结
In this study, we describe our experience with secukinumab (IL-17A inhibitor) and dupilumab (IL-4/IL-13 inhibitor) treatment of a group of pediatric patients with severe Neterton syndrome.
详细描述
Pediatric patients qualified for participation in the study if they had a documented diagnosis of Neterton syndrome and they received treatment with sekukinumab or dupilumab for at least 3 months.
All patients received treatment in the Department of Immunology at the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology from January 2021 to June 2024.
A total of 15 patients from 13 families with NS were enrolled in the study. The median age of the patients was 8 years (1;15), and the male/female ratio 9/6.
In this study, 14 patients initially received secukinumab therapy, for 12/14 patients dupilumab was added, and 2 patients continued secukinumab monotherapy. The median time to switch from secukinumab monotherapy to combination therapy was 4 months (3;27). One patient initially received dupilumab monotherapy and was switched to combination therapy (Fig. 1). Treatment duration for each type of treatment was at least 3 months at the time of this report.
Secukinumab was administered in a weight-adapted dosing regimen equivalent to that used in clinical trials for psoriasis: 75 mg for less than 25 kg,150 mg for 25 to 50 kg, and 300 mg for greater than 50 kg at baseline and weeks1,2,3, and 4 and monthly there after.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- — 至 18 Years(Child, Adult)
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pediatric patients with Neterton syndrome
- •Treatment with secukinumab and/or dupilumab for at least 3 months
排除标准
- •Irregular administration of therapy
- •Development of a serious adverse event
结局指标
主要结局
overal survival Kaplan-Mayer method
时间窗: 3 and 6 months from the start of each type of therapy
Evaluation of the efficacy and safety of dupilumab and secukinumab therapy in patients with Netherton syndrome
次要结局
- event free survival(3 and 6 months from the start of each type of therapy)
