Comparison of Different Prophylaxis Regimens for Moderate to Severe Hemophilia A Pediatric Patients
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 1
- 主要终点
- Annualized bleeding rate per year
研究概览
简要总结
Title: Comparison of different prophylaxis regimens for hemophilia A pediatric patients Principal Investigator: Assistant Professor Darintr Sosothikul, MD Research Question: Does different factor VIII dosage effect outcome of hemophilia treatment in term of breakthrough bleeding, number of hospital stay and day-off from school? Type Research: Clinical research
Study design: Single center clinical trials
Concise methodology:
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Study Population: Children with hemophilia A who received treatment at KCMH from May 2015 to March 2016 will be enrolled in this study. The consent will be obtained before the study.
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Observation and measurement:
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History, interesting clinical data and laboratory data will be recorded in Clinical record Form (CRF)
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Measurement:
i. Complete blood count (CBC), Factor VIII level, Factor VIII inhibitor level ii. Number of breakthrough bleedings, number of hospital stay and day-off from school iii. Joint score from Hemophilia Joint Health Score 2.1 iv. EQ-5D-5L quality of life assessment score 3. Data analysis: The p-value of less than 0.05 will be considered statistically significant. Mann-Whitney test will be used to test correlation of these variables (CBC, Factor VIII level, Factor VIII inhibitor level, Number of breakthrough bleedings, number of hospital stay, day-off from school, Hemophilia Joint Health Score 2.1, EQ-5D-5L quality of life assessment score) Sample size: 16 patients
Potential impacts:
The outcomes of different factor VIII concentrate dose between 15-20 U/kg/dose 2 times/week and 35-40 U/kg/dose 1 time/week will be revealed. These outcomes include number of breakthrough bleeding, number of hospital stay, day-off from school, joint health and quality of life. The result of this study will guide further study on optimal dose and duration of factor VIII treatment of hemophilia A patients in the future.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Months 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Hemophilia patients with moderate (factor level 1-3%) or severe (factor level <1%) severity
排除标准
- •Platelet count less than 100,000 /mm3 or other bleeding tendency
- •Hemophilia patients who have FVIII inhibitor >0.6 BU (modified Nijmogen method)
- •Hemophilia patients who have no bleeding symptoms
研究组 & 干预措施
Arm 1
FVIII concentration at 35-40 U/kg/dose 1 time/week for 5 months
干预措施: FVIII (Drug)
Arm 2
FVIII concentration at 15-20 U/kg/dose 2 time/week for 5 months
干预措施: FVIII (Drug)
结局指标
主要结局
Annualized bleeding rate per year
时间窗: 10 months
次要结局
- Hemophilia joint health score(10 months)
- Amount of FVIII use(10 months)
- Quality of life score(10 months)
- Number of hospital stays(10 months)
- Number of school days loss(10 months)
研究者
Darintr Sosothikul
Associate professor
Chulalongkorn University