Early Treatment Outcomes in Pierre-Robin-Like Phenotype

Study Overview
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Brief Summary
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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
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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.

Official Title
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Cohort Study on Prevalence and Early Treatment Outcomes in Pierre-Robin-Like Phenotype

Conditions
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Pierre Robin Syndrome

Intervention / Treatment
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* Device: Pre-epiglottic baton plate (PEBP)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Infants < 1 year at admission, Pierre-Robin-like phenotype Exclusion Criteria: no parental consent; need for immediate tracheotomy

Ages Eligible for Study
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Maximum Age: 12 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Device: Pre-epiglottic baton plate (PEBP)|modified acrylic palatal plate in which a velar extension resembling a spur or baton shifts the base of the tongue forward. This treatment is the standard care for infants with Pierre-Robin-Like Phenotype in the participating study centers and not assigned by protocol.|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in mixed-obstructive apnea index (MOAI) | 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 | upon admission within the first year of life (before start of treatment) and 3 months after hospital discharge | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in standard deviation score for body weight | Body weight will be obtained by electronic scales and the standard deviation score for weight calculated | upon admission within the first year of life (before start of treatment) and 3 months after hospital discharge | 
| Duration of hospital stay |  | admission within the first year of life for initiation of study intervention | 
| Treatment failure |  | 3 months after admission for initiation of study intervention | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Upper airway obstruction