Early Treatment Outcomes in Pierre-Robin-Like Phenotype

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

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.

Study Overview

• Status: Completed
• Conditions: Pierre Robin Syndrome
• Intervention / Treatment: Device: Pre-epiglottic baton plate (PEBP)

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 Type: Observational
• Enrollment (Anticipated): 50

Contacts and Locations

Study Locations

• Germany
  • Cologne, Germany, 50924
    • Centre of Competence for Oro- and Craniofacial Malformations and Department of Neonatology, Univ. Hospital of Cologne
  • Tuebingen, Germany, 72076
    • Dept. of Neonatology and Interdisciplinary Centre for Craniofacial Malformations, University Hospital Tuebingen
  • Würzburg, Germany, 97080
    • Dept. of Orthodontics and University Children's Hospital, University Hospital of Wuerzburg

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 1 year (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Infants <1 year admitted with Pierre-Robin-like phenotype to participating centres

Description

Inclusion Criteria:

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

Exclusion Criteria:

• no parental consent; need for immediate tracheotomy

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: University Hospital Tuebingen
• Collaborators: German Federal Ministry of Education and Research
• Investigators: Principal Investigator: Christian F Poets, MD, Dept. of Neonatology and Interdisciplinary Centre forCraniofacial Malformations, University Hospital Tuebingen; Principal Investigator: Siegmar Reinert, MD, PhD, Dept. of Craniofacial Surgery, University Hospital Tuebingen

Publications and helpful links

General Publications

• Poets CF, Maas C, Buchenau W, Arand J, Vierzig A, Braumann B, Muller-Hagedorn S. Multicenter study on the effectiveness of the pre-epiglottic baton plate for airway obstruction and feeding problems in Robin sequence. Orphanet J Rare Dis. 2017 Mar 9;12(1):46. doi: 10.1186/s13023-017-0602-8.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2013
• Primary Completion (Actual): August 31, 2016
• Study Completion (Actual): December 31, 2016

Study Registration Dates

• First Submitted: September 30, 2014
• First Submitted That Met QC Criteria: October 10, 2014
• First Posted (Estimate): October 16, 2014

Study Record Updates

• Last Update Posted (Actual): May 11, 2018
• Last Update Submitted That Met QC Criteria: May 9, 2018
• Last Verified: May 1, 2018

More Information

Terms related to this study

• Keywords: Upper airway obstruction
• Additional Relevant MeSH Terms: Congenital Abnormalities; Musculoskeletal Diseases; Stomatognathic Diseases; Stomatognathic System Abnormalities; Jaw Abnormalities; Jaw Diseases; Maxillofacial Abnormalities; Craniofacial Abnormalities; Musculoskeletal Abnormalities; Pierre Robin Syndrome
• Other Study ID Numbers: FACE-Subproject SP5-Tuebingen