Early Treatment Outcomes in Pierre-Robin-Like Phenotype

Completed

• Conditions: Pierre Robin Syndrome

• Registration Number: NCT02266043
• Lead Sponsor: University Hospital Tuebingen

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 Timeline

• Study Start: 2013-01-01
• Study First Submitted: 2014-09-30
• Study First Submitted QC: 2014-10-10
• Study First Posted: 2014-10-16
• Primary Completion: 2016-08-31
• Study Completion: 2016-12-31
• Status Verified: 2018-05
• Last Update Submitted: 2018-05-09
• Last Update Posted: 2018-05-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

• Infants < 1 year at admission, Pierre-Robin-like phenotype

Exclusion Criteria

• no parental consent; need for immediate tracheotomy

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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
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

Trial Locations

Locations (3)

Dept. of Neonatology and Interdisciplinary Centre for Craniofacial Malformations, University Hospital Tuebingen; 🇩🇪 Tuebingen, Germany

Centre of Competence for Oro- and Craniofacial Malformations and Department of Neonatology, Univ. Hospital of Cologne; 🇩🇪 Cologne, Germany

Dept. of Orthodontics and University Children's Hospital, University Hospital of Wuerzburg; 🇩🇪 Würzburg, Germany