Cohort Study on Prevalence and Early Treatment Outcomes in Pierre-Robin-Like Phenotype
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Enrollment
- 50
- Locations
- 3
- Primary Endpoint
- Change in mixed-obstructive apnea index (MOAI)
Study Overview
Brief Summary
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.
Detailed Description
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.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- — to 12 Months (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Infants < 1 year at admission, Pierre-Robin-like phenotype
Exclusion Criteria
- •no parental consent; need for immediate tracheotomy
Outcomes
Primary Outcomes
Change in mixed-obstructive apnea index (MOAI)
Time Frame: 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
Secondary Outcomes
- 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)