A Comparative Study To Assess The Effect Of On Demand Vs Prophylactic Factor VIII Infusion On Annual Total Bleed Rate, Annual Joint Bleed Rate and Quality Of Life In Children Aged 12-18 Years With Severe Hemophilia A
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Annual Total Bleed Rate, Annual joint Bleed rate and quality of life
研究概览
简要总结
INTRODUCTION- Long term prophylaxis for treatment of Severe Hemophilia is the Gold Standard for significantly reducing symptoms in comparison to on demand treatment, with significant reduction in severity of bleeding episodes, improvement in quality of life in Hemophilic arthropathy. It is ideal to start primary prophylaxis to prevent arthropathy. Very little is known about the effects of late prophylaxis and we intend to see the response of initiation of secondary/ tertiary prophylaxis in children with severe Hemophilia A, aged 12-18 years.
AIMS AND OBJECTIVES- To assess the Annual Total Bleed Rate, Annual Joint Bleed Rate and Quality of Life in children aged 12 to 18 years with severe Hemophilia A, receiving on demand or prophylaxis with Factor VIII and then compare the effect of both the therapies on the same parameters.
MATERIALS AND METHODS- 50 males, in the age group 12- 18 years , diagnosed with severe Hemophilia A meeting the inclusion and exclusion criteria will be randomized and allocated to the two groups- on demand and prophylaxis. Effect of either of the therapies on the outcomes will be compared.
研究设计
- 研究类型
- Interventional
- 分配方式
- Computer generated randomization
- 盲法
- Not Applicable
入排标准
- 年龄范围
- 12.00 Year(s) 至 18.00 Year(s)（—）
- 性别
- Male
入选标准
- •Severe Hemophilia (Factor VIII levels <1% at diagnosis)
- •Age more than equal to 12 years to less than equal to 18 years at enrolment
- •more than equal to 6 treated joint bleeds in the previous one year
- •Inhibitor screen negavtive at enrolment
- •No evidence of hepatitis
- •Viral markers negative
- •Not on any prophylactic regimen in the last one year
- •Not on any non-factor replacement therapy.
排除标准
- •Female Gender
- •Factor VIII levels >1% at diagnosis
- •Age <12 years or > 18 years at enrolment
- •<6 treated joint bleeds in the last one year.
- •Inhibitor screen positive at enrolment
- •Evidence of hepatitis.
结局指标
主要结局
Annual Total Bleed Rate, Annual joint Bleed rate and quality of life
时间窗: Baseline before the study initiation and after one year of study.
次要结局
- Hemophilia Joint Health Score, Incidence of inhibitors, Pettersson Joint score, IPSG MRI score, HEAD US scoring system, HAL/Ped-Hal, Functional Impairment Scoring for Hemophilia (FISH), Health Related Quality of life(Inhibitor screening every 3 months)