Off-label Use of Tacrolimus in Children With Henoch-Schönlein Purpura Nephritis: Effectiveness and Safety
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 25
- 主要终点
- nonresponsive
研究概览
简要总结
Henoch-Schönlein purpura (HSP) is the most common vasculitis in children, with an incidence of approximately 10:100 000 children and a slight male predominance (male-to-female ratio of 1.5:1). Henoch-Schönlein purpura nephritis (HSPN) is the principal cause of morbidity for HSP and 1%-7% of HSPN patients may progress to renal failure or end-stage renal disease.
Immunosuppressive therapy has become the standard treatment in children with HSPN, however the use of these drugs are still mainly in an off-label manner in clinical practice. Tacrolimus, a calcineurin inhibitor, has been recently suggested in the treatment of HSPN in children. However, the evidence-based clinical data are still limited.
Given the potential benefits and unmet need in clinical practice, the purposes of this pilot study were to assess effectiveness and safety of tacrolimus in HSPN children and evaluate the potential impact of CYP3A5.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •HSPN children Aged less than 18 years; receiving tacrolimus as initial immunosuppressive therapy -
排除标准
- •Children received other immunosuppressive drug before the trial or other systemic trial drug therapy; Children had a concomitant medical condition, whose participation, in the opinion of the Investigator and/or medical advisor, may create an unacceptable additional risk.
研究组 & 干预措施
therapy group
Immunosuppressive therapy included tacrolimus and prednisone.
干预措施: tacrolimus (Drug)
therapy group
Immunosuppressive therapy included tacrolimus and prednisone.
干预措施: prednisone (Drug)
结局指标
主要结局
nonresponsive
时间窗: within 6 months
A nonresponsive patient was defined if there was no improvement in clinical symptoms or signs 6 months after the therapy of tacrolimus with or without prednisone, or urinary protein remained more than 40mg/h per m2 body surface area.
Complete remission
时间窗: within 6 months
clinical symptoms and signs disappeared and proteinuria was less than 4mg/h per m2 body surface area within 6 months.
Partial remission
时间窗: within 6 months
if proteinuria was reduced to 4.1-40mg/h per m2 body surface area within 6 months. A nonresponsive patient was defined if there was no improvement in clinical symptoms or signs 6 months after the therapy of tacrolimus with or without prednisone, or urinary protein remained more than 40mg/h per m2 body surface area.
次要结局
未报告次要终点
研究者
Wei Zhao
Head of department of clinical pharmacy and pharmacology
Shandong University