Clinical Trial: Early Treatment Outcomes in Pierre-Robin-Like Phenotype

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Observational

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

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 Summary:

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.


Sponsor: University Hospital Tuebingen

Current Primary Outcome: 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


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in standard deviation score for body weight [ Time Frame: 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 [ Time Frame: admission within the first year of life for initiation of study intervention ]
  • Treatment failure [ Time Frame: 3 months after admission for initiation of study intervention ]


Original Secondary Outcome: Same as current

Information By: University Hospital Tuebingen

Dates:
Date Received: September 30, 2014
Date Started: January 2013
Date Completion: December 2016
Last Updated: May 11, 2016
Last Verified: May 2016