Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference?
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 200
- 试验地点
- 1
- 主要终点
- Phe values
研究概览
简要总结
Treatment of PKU implies for most patients that with strict adherence to dietary treatment they can achieve excellent neurocognitive outcome. Dietary treatment, though, is hard to comply to every day and with every single meal. Unsurprisingly, health-related quality of life (HrQol) is negatively affected if patients have to follow a dietary regime of this kind. Adherence to treatment in PKU is very variable. Factors of significant impact on adherence to treatment and well-being in chronic disease such as self-efficacy or parenting stress have not yet been widely investigated in PKU patients.
The ideal treatment prescription (and guideline) recommends as much as necessary and as little as possible, based on the best evidence available. Patients should neither be deprived of treatment options nor be exposed to overtreatment.
This study investigates adherence, metabolic control, HrQol in PKU patients treated by centres which follow different guidelines
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with PKU from age 10 years requiring dietary and / or pharmacological treatment according to locally applied guidelines
- •Parents of patients with PKU aged 10 to 18 years requiring dietary and / or pharmacological treatment according to locally applied guidelines
- •Ability and willingness to answer the questionnaires and follow the study procedures
- •Informed consent as documented by signature
- •Exclusion criteria for patients and parents :
- •Inability to follow the procedures of the study, e.g. due to language problems or severely reduced health status
- •Pregnant patients or patients who have disclosed to their physician that they are planning a pregnancy in the near future
- •Hyperphenylalaninaemia or mild forms of PKU or other inborn errors associated with elevated Phe but not requiring dietary and / or pharmacological treatment according to locally applied guidelines
- •No inform
排除标准
- 未提供
结局指标
主要结局
Phe values
时间窗: report on measurements from past 24 months
biochemical marker for PKU
次要结局
- Health-related quality of life(one hour)
- Burden on family (patients from 10-18 years)(one hour)
- Self-efficacy(one hour)