Cohort Study on Prevalence and Early Treatment Outcomes in Pierre-Robin-Like Phenotype
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 50
- 试验地点
- 3
- 主要终点
- Change in mixed-obstructive apnea index (MOAI)
研究概览
简要总结
In a longitudinal cohort study, we want to assess the effect of the pre-epiglottic baton plate, the main treatment approach currently used in Germany for Pierre-Robin-Like phenotype, on upper airway obstruction and failure to thrive using objective criteria (mixed-obstructive apnea index assessed by polysomnography, standard deviation score for body weight) upon admission and 3 months after hospital discharge.
详细描述
Pierre-Robin sequence consists of a small lower jaw, a retropositioned tongue (glossoptosis), and optionally cleft palate, leading to severe upper airway obstruction and failure to thrive; occasionally even to sudden infant death. Treatment approaches are heterogeneous, some are considerably invasive; few have been evaluated by objective tests.
In a longitudinal cohort study, we want to assess the effect of the pre-epiglottic baton plate on upper airway obstruction and failure to thrive using objective criteria (mixed-obstructive apnea index assessed by polysomnography, standard deviation score for body weight) upon admission and 3 months after hospital discharge. The pre-epiglottic baton plate is the main treatment approach currently used in Germany for this condition and practised standard care in the three participating study centers.
These data will help to compare the effect of the main treatment currently used in Germany with international data on more invasive treatments used in this rare condition.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 12 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Infants < 1 year at admission, Pierre-Robin-like phenotype
排除标准
- •no parental consent; need for immediate tracheotomy
结局指标
主要结局
Change in mixed-obstructive apnea index (MOAI)
时间窗: upon admission within the first year of life (before start of treatment) and 3 months after hospital discharge
Change in mixed-obstructive apnea index (MOAI) assessed by polygraphy from pre- to post-intervention as a marker of Pierre-Robin-sequence-related breathing disorder
次要结局
- Change in standard deviation score for body weight(upon admission within the first year of life (before start of treatment) and 3 months after hospital discharge)
- Treatment failure(3 months after admission for initiation of study intervention)
- Duration of hospital stay(admission within the first year of life for initiation of study intervention)