Risk Models to Optimise Prophylaxis Schedules in Children With Haemophilia
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 9
- 主要终点
- Acceptability of injection frequencies
研究概览
简要总结
The MOrPH study is designed to identify optimal prophylaxis schedules for children with haemophilia. This involves development of combined pharmacokinetic and pharmacodynamic models. Interpretation of model outputs will be informed by two surveys. The first will survey families of children with haemophilia to ascertain families' values and preferences concerning prophylaxis schedules. The second will survey haemophilia physicians to ascertain the criteria physicians use to prescribe prophylaxis schedules.
详细描述
Methods: Part I (surveys)
Two cross-sectional surveys will be conducted, a family survey and a clinician survey. The surveys will be completed online. Participation will be voluntary and anonymous.
Family survey. The family survey will identify preferences of families of children with haemophilia for different prophylaxis schedules. At least 20 families will participate. This number should provide a clear indication of family's preferences for different prophylaxis schedules.
People will be eligible to participate in the family survey if they have haemophilia A or B and are aged between 14 and 17 years, or if they are the parent of a child (< 18 years) with haemophilia.
Participants will be recruited using advertisements placed in community print-based and/or electronic communications and, if necessary, by inviting families attending a youth camp for people with haemophilia. The survey will ask participants about the characteristics of the child with haemophilia including the child's age, current level and frequency of physical activity and sports participation, current prophylactic medication schedule and method of administration. They will also be asked to rate the acceptability of a number of possible prophylactic schedules as "acceptable", "marginally acceptable" or "unacceptable".
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Cross Sectional
入排标准
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •People will be eligible to participate in the family survey if they have haemophilia A or B and are aged between 14 and 17 years, or if they are the parent of a child (< 18 years) with haemophilia
- •People will be eligible to participate in the physician survey if they are physicians currently practising in paediatric haemophilia treatment centres.
排除标准
- •Unable to communicate effectively in English.
结局指标
主要结局
Acceptability of injection frequencies
时间窗: Day 1 (i.e. on the day of response to the survey)
Families will be asked to rate as "acceptable", marginally acceptable" or "unacceptable" different frequencies of prophylactic factor injections.
Acceptability of time of injections
时间窗: Day 1 (i.e. on the day of response to the survey)
Families will be asked to rate as "acceptable", marginally acceptable" or "unacceptable" different injection times.
Importance of factors influencing prescription of prophylaxis schedules
时间窗: Day 1 (i.e. on the day of response to the survey)
Physicians will be asked to rank the importance of factors (including cost, tolerability for families, venous access, physical activity and sport, pharmacokinetics, inhibitor development and age) that influence their prescription of prophylaxis schedules.
次要结局
未报告次要终点