"The Safety and Efficacy of Mini-Pool IVIG Initiation and Maintenance Therapy for Management of Children With Persistent ITP, A Novel Approach for LMICs"
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 3
- 主要终点
- Bleeding Frequency and Severity
研究概览
简要总结
The goal of this clinical trial is to learn if mini-pool intravenous immunoglobulin (IVIG) is a safe and effective treatment for children with persistent immune thrombocytopenia (ITP). ITP is a condition that causes low platelet levels and increases the risk of bleeding. The main questions this study aims to answer are:
Can mini-pool IVIG raise platelet levels in children with persistent ITP?
Can it reduce bleeding episodes and hospital visits?
What side effects, if any, are seen with this treatment?
There is no comparison group in this study. All participants will receive mini-pool IVIG, which is made from small pools of donated plasma using a cost-effective process.
Participants will:
Receive one dose of mini-pool IVIG through a vein over 6 to 8 hours
Receive follow-up doses every 2 to 4 weeks for up to 5 doses, based on their platelet count
Have regular blood tests and checkups during the study and for 6 months after treatment
Report on bleeding episodes, physical activity, school attendance, and side effects
详细描述
Immune thrombocytopenia (ITP) is an autoimmune condition where the immune system destroys platelets, leading to low platelet counts and increased risk of bleeding. Persistent ITP is defined as ongoing thrombocytopenia lasting 3 to 12 months after initial diagnosis. Children with persistent ITP who lose their response to first-line treatments, such as steroids or standard intravenous immunoglobulin (IVIG), have limited therapeutic options, especially in low- and middle-income countries, due to the high cost of commercial IVIG preparations.
Mini-pool IVIG is produced from small pools of plasma collected locally, using a validated process with virus inactivation and IgG purification steps. This method enables safe, cost-effective preparation of IVIG in resource-limited settings. Prior research has shown that mini-pool IVIG is effective and well-tolerated in acute pediatric ITP, but its role as a second-line therapy for persistent ITP has not been evaluated.
This multicenter, prospective clinical trial will enroll 20 children aged 1 to 10 years with persistent ITP at three tertiary care pediatric hematology centers in Egypt. Participants will receive a loading dose of mini-pool IVIG at 1 g/kg, followed by maintenance doses of 0.5 g/kg every 2 to 4 weeks for up to five additional doses, with dose intervals adjusted based on platelet counts.
Throughout the study, participants will undergo regular blood counts, bleeding assessments using the Bleeding Assessment Tool (BAT), and monitoring for infusion-related or delayed adverse events. Data on school attendance, physical activity, and patient or family satisfaction will also be collected.
Responses to therapy will be classified as complete response (CR), response (R), or no response (NR) based on platelet count thresholds and bleeding status, with response duration measured from achievement of CR or R to loss of response. Participants achieving sustained response off therapy (SRoT) or response off therapy (RoT) during the 6-month post-treatment follow-up will be identified to evaluate durability of treatment effects.
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
- 1 Year 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 1 - 10 years
- •Gender: Males and Females
- •Persistent ITP according to ASH definition
- •No history of treatment with thrombopoietin agonists
排除标准
- •History of severe drug adverse events to IVIG
- •Previous history of ICH
- •Difficult venous access
- •Congenital thrombocytopenia, secondary ITP and non-immune thrombocytopenia
研究组 & 干预措施
Mini-Pool IVIG Treatment Arm
Participants in this arm will receive mini-pool intravenous immunoglobulin (IVIG) as second-line therapy for persistent immune thrombocytopenia (ITP). Treatment includes a loading dose of 1 g/kg infused over 6-8 hours, followed by maintenance doses of 0.5 g/kg given every 2 to 4 weeks for up to five additional doses. The dosing interval is adjusted based on each participant's platelet count. Participants will have regular blood tests and clinical assessments to monitor platelet levels, bleeding episodes, and treatment safety.
干预措施: Mini-Pool IVIG (Biological)
结局指标
主要结局
Bleeding Frequency and Severity
时间窗: From enrollment through 6 months post-treatment.
Number, location, and severity of bleeding episodes assessed using the Bleeding assessed using the ISTH Bleeding Assessment Tool (BAT) score, which evaluates bleeding symptoms across multiple anatomical sites. Each site is scored from 0 to 4, with higher scores indicating more severe bleeding. The total score varies depending on the number and severity of bleeding events.Assessment Tool (BAT) score.
Platelet Count Response
时间窗: Assessed monthly during treatment and for 6 months after last dose.
Number of participants achieving complete response (platelet count ≥100×10⁹/L) or response (platelet count ≥30×10⁹/L and at least 2-fold increase from baseline) without bleeding.
Frequency of Hospital Admissions Due to Critical Bleeding
时间窗: From enrollment through 6 months post-treatment.
Number of hospital admissions for life-threatening bleeding episodes, excluding admissions solely for IVIG infusion.
次要结局
- Adverse Events(From first infusion through 6 months after the last dose.)
- School Attendance(From enrollment through 6 months post-treatment.)
- Patient and Family Satisfaction(Assessed at the end of treatment and at 6-month follow-up.)
- Sustained Response Off-Therapy (SRoT/RoT)(6 months after last mini-pool IVIG dose.)
研究者
Mervat A. M. Youssef
Principal investigator
Assiut University