Efficacy and Safety of Rituximab in Reducing Relapses in Children With Steroid-Sensitive Nephrotic Syndrome: An Open-label, Multi-center Clinical Study.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 91
- 试验地点
- 1
- 主要终点
- Total Effective Rate
研究概览
简要总结
This study investigated the combined impact of rituximab and targeted nursing care versus tacrolimus and targeted nursing care on efficacy, quality of life, adverse reactions, and recurrence rate in children with challenging (steroid-dependent or frequently relapsing) nephrotic syndrome. Ninety-one pediatric patients were randomized to either receive rituximab plus targeted nursing or tacrolimus plus targeted nursing, and outcomes were assessed over a 6-month period.
详细描述
Nephrotic syndrome in children, particularly steroid-dependent (SDNS) or frequently relapsing (FRNS) types, poses significant treatment challenges. While glucocorticoids are initial mainstays, second-line immunosuppressants like tacrolimus or rituximab are often required. Rituximab, a monoclonal antibody targeting CD20 on B-lymphocytes, has shown promise. Targeted nursing care, a patient-centered approach involving joint goal-setting and personalized interventions, aims to improve adherence and overall well-being. This study aimed to evaluate the synergistic effect of rituximab therapy combined with a structured targeted nursing care program compared to tacrolimus with the same targeted nursing care.
A total of 91 pediatric patients (aged 6-15 years) with SDNS or FRNS were enrolled from January 2021 to June 2023. They were randomized into a study group (n=46, rituximab + targeted nursing) and a control group (n=45, oral tacrolimus + targeted nursing). Both groups received baseline glucocorticoid therapy. The study group received intravenous rituximab (375 mg/m² weekly for 12 weeks). The control group received oral tacrolimus (0.05-0.1 mg/kg/day, adjusted to trough levels). Both groups received identical targeted nursing care protocols.
Outcomes compared included clinical efficacy (remission status), quality of life (IS-LQ questionnaire), incidence of adverse reactions, and recurrence rate over a 6-month follow-up. Peripheral blood CD19+ B-cell counts were monitored in the rituximab group. The study was approved by the institutional ethics committee, and informed consent was obtained.
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
- None
入排标准
- 年龄范围
- 6 Years 至 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of primary pediatric nephrotic syndrome, specifically steroid-dependent nephrotic syndrome (SDNS) or frequently relapsing nephrotic syndrome (FRNS), as defined by the Kidney Disease: Improving Global Outcomes (KDIGO) guidelines. (SDNS is defined as relapse occurring during tapering of glucocorticoids or within 14 days of discontinuation; FRNS is defined as ≥2 relapses within 6 months of initial response or ≥4 relapses in any 12-month period).
- •Age between 6 and 15 years.
- •Normal intellectual development and the ability to understand and communicate.
- •Informed consent obtained from the guardians and assent from children capable of understanding.
排除标准
- •Steroid-resistant nephrotic syndrome (SRNS).
- •Severe renal insufficiency (according to KDIGO criteria or other relevant standards).
- •Cognitive or mental disorders.
- •Worsening condition upon admission necessitating intensive care.
- •Parents unable to provide long-term care or unwilling to participate.
- •Interruption of hospitalization for any reason.
- •Prior treatment with rituximab or tacrolimus.
- •Co-existing significant kidney, urinary system diseases (other than NS), or severe active urinary tract infection.
- •Significant liver function impairment.
- •Known hypersensitivity to rituximab or tacrolimus.
研究组 & 干预措施
Rituximab with Targeted Nursing Care
Patients received intravenous rituximab in conjunction with baseline glucocorticoid therapy and a standardized targeted nursing care program
干预措施: Rituximab (Drug)
Rituximab with Targeted Nursing Care
Patients received intravenous rituximab in conjunction with baseline glucocorticoid therapy and a standardized targeted nursing care program
干预措施: Targeted Nursing Care (Behavioral)
Rituximab with Targeted Nursing Care
Patients received intravenous rituximab in conjunction with baseline glucocorticoid therapy and a standardized targeted nursing care program
干预措施: Baseline Glucocorticoid Therapy (Drug)
Tacrolimus with Targeted Nursing Care
Patients received oral tacrolimus capsules in conjunction with baseline glucocorticoid therapy and a standardized targeted nursing care program identical to the study group.
干预措施: Targeted Nursing Care (Behavioral)
Tacrolimus with Targeted Nursing Care
Patients received oral tacrolimus capsules in conjunction with baseline glucocorticoid therapy and a standardized targeted nursing care program identical to the study group.
干预措施: Baseline Glucocorticoid Therapy (Drug)
Tacrolimus with Targeted Nursing Care
Patients received oral tacrolimus capsules in conjunction with baseline glucocorticoid therapy and a standardized targeted nursing care program identical to the study group.
干预措施: Tacrolimus (Drug)
结局指标
主要结局
Total Effective Rate
时间窗: At 6 months post-treatment initiation.
The percentage of participants who achieved a "Significantly effective" or "Effective" response. Efficacy is defined based on remission status, renal function, and proteinuria levels. Total effective rate is calculated as the number of (Significantly effective + Effective) cases divided by the total number of cases in the group.
Recurrence Rate
时间窗: Within the 6-month follow-up period after treatment initiation.
Relapse defined as urinary protein ≥2+ or urinary protein quantity \>50 mg/kg (or urine protein/creatinine ratio \>2.0 mg/mg), occurring on three consecutive days within a 7-day period, after having achieved remission.
次要结局
- Change in Quality of Life as Assessed by the Children's Subjective Quality of Life Questionnaire (IS-LQ)(Baseline and at 6 months post-treatment initiation.)
- Adverse Reactions(During the 6-month study period.)
研究者
Ting Xu
Principal investigator
Wuhan Children's Hospital