Pierre Robin Syndrome
Conditions
Keywords
Upper airway obstruction
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.
Interventions
DEVICEPre-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.
Sponsors
German Federal Ministry of Education and Research
University Hospital Tuebingen
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
No minimum to 12 Months
Healthy volunteers
No
Inclusion criteria
* Infants \< 1 year at admission, Pierre-Robin-like phenotype
Exclusion criteria
* no parental consent; need for immediate tracheotomy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in mixed-obstructive apnea index (MOAI) | 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
| Measure | Time frame | Description |
|---|---|---|
| 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 | Body weight will be obtained by electronic scales and the standard deviation score for weight calculated |
| 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 | — |
Countries
Germany
Outcome results
None listed