Clinical trial NCT02266043: Early Treatment Outcomes in Pierre-Robin-Like Phenotype
Official title: Cohort Study on Prevalence and Early Treatment Outcomes in Pierre-Robin-Like Phenotype
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Pierre Robin Syndrome
Keywords: Upper airway obstruction
Lead sponsor: University Hospital Tuebingen
Sponsor class: OTHER
Collaborators: name: German Federal Ministry of Education and Research
First posted: 2014-10-16
Enrollment: 50
Sex: ALL
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Infants \<1 year admitted with Pierre-Robin-like phenotype to participating centres

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.

Eligibility criteria: Inclusion Criteria:

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

Exclusion Criteria:

* no parental consent; need for immediate tracheotomy

Primary outcomes:
measure: Change in mixed-obstructive apnea index (MOAI)
timeFrame: upon admission within the first year of life (before start of treatment) and 3 months after hospital discharge
Secondary outcomes:
measure: Change in standard deviation score for body weight
timeFrame: upon admission within the first year of life (before start of treatment) and 3 months after hospital discharge
measure: Duration of hospital stay
timeFrame: admission within the first year of life for initiation of study intervention
measure: Treatment failure
timeFrame: 3 months after admission for initiation of study intervention
Other outcomes:
Locations:
city: Cologne
country: Germany
facility: Centre of Competence for Oro- and Craniofacial Malformations and Department of Neonatology, Univ. Hospital of Cologne
city: Tuebingen
country: Germany
facility: Dept. of Neonatology and Interdisciplinary Centre for Craniofacial Malformations, University Hospital Tuebingen
city: Würzburg
country: Germany
facility: Dept. of Orthodontics and University Children's Hospital, University Hospital of Wuerzburg
Study officials:
affiliation: Dept. of Neonatology and Interdisciplinary Centre forCraniofacial Malformations, University Hospital Tuebingen
name: Christian F Poets, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Dept. of Craniofacial Surgery, University Hospital Tuebingen
name: Siegmar Reinert, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.