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Validation of Sleep Questionnaires in the Down Syndrome Population

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ClinicalTrials.gov Identifier: NCT03771469
Recruitment Status : Recruiting
First Posted : December 11, 2018
Last Update Posted : September 4, 2019
Sponsor:
Information provided by (Responsible Party):
Derek Lam, MD, MPH, Oregon Health and Science University

Tracking Information
First Submitted Date  ICMJE August 10, 2018
First Posted Date  ICMJE December 11, 2018
Last Update Posted Date September 4, 2019
Actual Study Start Date  ICMJE June 25, 2018
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 7, 2018)
Apnea-hypopnea index (AHI) from polysomnography [ Time Frame: AHI collected at initial sleep study and 3 months later (if a second sleep study is needed as determined by doctor) ]
Overnight attended polysomnography is the gold standard for diagnosis of OSA. Assessment of sleep staging is done through electroencephalogram, electro-oculogram, and submental electromyogram. The primary output parameter that is commonly used to diagnose and characterize the severity of OSA is the apnea-hypopnea index (AHI).
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03771469 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: December 10, 2018)
  • Sleep Related Breathing Disorder subscale total of the Pediatric Sleep Questionnaire (SRBD-PSQ) [ Time Frame: SRBD score tallied at initial clinic visit. ]
    The SRBD-PSQ is a subjective instrument with 22-items that was designed to screen for sleep-related disordered breathing in the general pediatric population. Each question is answered with a yes (1 point), no (0 points), or don't know (0 points). The total number of points is added up, and that sum is divided by the number of questions answered to produce a ratio. When assessing the ratio, a figure greater than 0.3 is considered a positive screening for OSA. There are no subscales on this questionnaire.
  • Sleep-Related Quality of Life (OSA-18 total score) [ Time Frame: OSA-18 total collected at initial clinic visit. ]
    The OSA-18 is a subjective measure of disease-specific quality of life survey for sleep disordered breathing. It contains 18 questions. These questions are scored on a 7 point Likert scale, with "1" being the worst outcome and "7" being the best outcome. These numeric scores are totaled to produce one overall sum, with a range from 7 to 126.
  • Generic Quality of Life [ Time Frame: PedsQL summary scores collected at initial clinic visit. ]
    The Pediatric Quality of Life Inventory (PedsQL) is a validated generic quality of life measure encompassing 4 multidimensional scales (Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning) with three Summary Scores (Total Score, Physical Health Summary Score, Psychosocial Health Summary Score). The number of questions varies by age group but falls within 20-25 questions. The questions use 5-point Likert scale from 0 (Never) to 4 (Almost always). Items are reversed scored and linearly transformed to a 0-100 scale (0=100, 1=75, 2=50, 3=25, 4=0.). Dimensions are scored by transforming into mean score = Sum of the items over the number of items answered. For each of the 4 scales, higher scores indicate better HRQOL (better outcome).
Original Secondary Outcome Measures  ICMJE
 (submitted: December 7, 2018)
  • Sleep Related Breathing Disorder subscale total of the Pediatric Sleep Questionnaire (SRBD-PSQ) [ Time Frame: SRBD score tallied at initial clinic visit. ]
    The SRBD-PSQ is a subjective instrument with 22-items that was designed to screen for sleep-related disordered breathing in the general pediatric population. Each question is answered with a yes (1 point), no (0 points), or don't know (0 points). The total number of points is added up, and that sum is divided by the number of questions answered to produce a ratio. When assessing the ratio, a figure greater than 0.3 is considered a positive screening for OSA. There are no subscales on this questionnaire.
  • Sleep-Related Quality of Life (OSA-18 total score) [ Time Frame: OSA-18 total collected at initial clinic visit. ]
    The OSA-18 is a subjective measure of disease-specific quality of life survey for sleep disordered breathing. It contains 18 questions. These questions are scored on a 7 point Likert scale, with "1" being the worst outcome and "7" being the best outcome. These numeric scores are totaled to produce one overall sum, with a range from 7 to 126.
  • Generic Quality of Life [ Time Frame: PedsQL summary scores collected at initial clinic visit. ]
    The Pediatric Quality of Life Inventory (PedsQL) is a well-validated generic quality of life measure encompassing 4 multidimensional scales (Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning) with three Summary Scores (Total Score, Physical Health Summary Score, Psychosocial Health Summary Score).
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Validation of Sleep Questionnaires in the Down Syndrome Population
Official Title  ICMJE Validation of Sleep Questionnaires in the Down Syndrome Population
Brief Summary This will be a prospective validation study of a sample of consecutive pediatric Down syndrome patients who are seen through the weekly Down syndrome clinic at OHSU/Doernbecher's. Questionnaires will be administered to approximately 5 new patients per month. Since this population has a higher prevalence of OSA than the general pediatric population, and OSA is a potentially modifiable determinant of quality of life, validated instruments are critical in assessing disease burden and response to treatment.
Detailed Description

Specific Aims:

  1. Demonstrate the criterion validity of the Sleep-Related Breathing Disorder subscale of the PSQ as a screening tool for the diagnosis of OSA in children with Down Syndrome, using polysomnography as the gold standard.

    Hypothesis: Compared to the published threshold for a positive screen in the general pediatric population (≥ 7 of 22 positive responses), the threshold for a positive screen that corresponds to an optimal sensitivity and specificity in the Down syndrome population will be significantly different.

  2. Demonstrate the construct validity of the OSA-18 as a scale to assess sleep-related quality of life in children with Down Syndrome by comparing OSA-18 scores to an objective measure of disease burden (polysomnography) and a generic quality of life instrument (the Pediatric Quality of Life inventory, PedsQL).

Hypothesis: OSA-18 scores will be significantly associated with the Apnea-Hypopnea Index assessed by polysomnography and the PedsQL Total Score, Physical Health, and Psychosocial Health summary scores.

Background:

  1. Obstructive Sleep Apnea and Down Syndrome: Obstructive sleep apnea (OSA) affects 1-5% of children in the US and has been associated with a myriad of health consequences including cardiovascular complications, behavioral disturbances, and neurocognitive dysfunction. In contrast, there is a reported OSA prevalence of 31-79% in children with Down Syndrome due to traits that predispose to OSA including hypotonia, obesity, and craniofacial anatomy such as midfacial and mandibular hypoplasia which can lead to pharyngeal crowding. With increased risk of congenital cardiovascular defects in the Down Syndrome population, it is possible that these children are also at risk of the most serious complications of OSA including pulmonary hypertension.

    OSA has also been shown to have a significant impact on quality of life. Behavioral problems associated with OSA include reduced attention, hyperactivity, irritability and problems with peers. Previous studies in the general pediatric population have shown similar quality of life scores in children with symptoms of OSA as children with asthma and rheumatoid arthritis. In children with Down syndrome, reduced sleep has been associated with reduced cognitive function, memory, poor communication skills, and poor self-help skills. Furthermore, parents of children with sleep disordered breathing often suffer from sleep deprivation themselves which can result in negative impacts on family life, decreased ability to care for their children and higher levels of maternal stress.

  2. Subjective Measures of Sleep Disordered Breathing and Obstructive Sleep Apnea: Overnight polysomnography (PSG) is the gold standard for diagnosing OSA in children. However, due to cost and inconvenience, only a minority of patients being evaluated for OSA undergo PSG prior to adenotonsillectomy. One survey study conducted among pediatric otolaryngologists showed that 31% of respondents said they referred children suspected of OSA for PSG "rarely" or "never." In a separate study, 75% of pediatric otolaryngologists surveyed referred for PSG in less than 10% of children with suspected OSA. Commonly cited factors for this include cost of obtaining PSG and delay in obtaining PSG due to availability. In addition, a substantial proportion of patients referred for PSG are either lost to follow-up or experience significant delays in treatment due to testing. As a result, alternative methods of screening for or diagnosing OSA have been explored that are cheaper and less burdensome. This includes a variety of questionnaires that were designed to screen the pediatric population for symptoms of sleep disordered-breathing (SDB) and assess its impact on quality of life within a clinic setting. The Sleep-Related Breathing Disorders subscale of the Pediatric Sleep Questionnaire (SRBD-PSQ) was developed to screen for SDB using 3 categories: daytime sleepiness, snoring, and behavioral disturbances.3 This has previously been validated in children aged 2-18 within the general pediatric population. The OSA-18 is a survey that measures the impact of SDB or OSA on disease-specific quality of life in children by assessing common manifestations of the disease including sleep disturbance, emotional distress, daytime function, and caregiver concerns. This questionnaire has been validated in children ages 6 months to 12 years. Validated subjective measures like these capture different aspects of the disease experience than objective measures like PSG. They can also be used to assess large numbers of patients with far less burden and expense than PSG which frequently has long wait times due to limited capacity.
  3. No Validated Screening instruments or OSA-related QOL measures in Down Syndrome: Despite the high prevalence of OSA in the Down syndrome population and the availability of widely used questionnaires for SDB, screening for SDB is generally inconsistent in this population. Even when parental report of symptoms of SDB is solicited, multiple studies have demonstrated poor diagnostic accuracy of parental history compared to PSG. A recent study investigating parental assessment of the symptoms of SDB found that 66% of Down syndrome patients had frequent symptoms consistent with SDB including snoring, witnessed apnea, and restless sleep. However, there was no association between the frequency of these symptoms and diagnosis with OSA. Other studies have similarly demonstrated poor diagnostic accuracy of parental history with respect to PSG findings. For this reason, the most recent American Academy of Pediatrics (AAP) guideline regarding management of Down syndrome patients has recommended routine screening for OSA using PSG in all patients by the age of 4, regardless of symptomatology. There are currently no validated instruments for screening for OSA or assessing OSA-related quality of life in the Down syndrome population.
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Intervention Model: Single Group Assignment
Intervention Model Description:
This will be a prospective validation study of a sample of consecutive pediatric Down syndrome patients who are seen through the weekly Down syndrome clinic at OHSU/Doernbecher's.
Masking: None (Open Label)
Primary Purpose: Screening
Condition  ICMJE
  • Obstructive Sleep Apnea
  • Down Syndrome
Intervention  ICMJE Diagnostic Test: Sleep study
Sleep study
Other Name: polysomnography
Study Arms  ICMJE Experimental: Questionnaires and sleep studies
Caregivers of patients meeting eligibility criteria will be invited to participate. If they agree to participate, baseline SRBD-PSQ, OSA-18, and PedsQL questionnaires along with written informed consent forms will be mailed to them along with their standard scheduling paperwork. Caregivers will be asked to review the consent form and complete the questionnaires and bring the paperwork to clinic on the day of their visit. Sleep study testing will also be ordered prior to their visit so that it can be scheduled within a month of the initial clinic visit and again three months later.
Intervention: Diagnostic Test: Sleep study
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 7, 2018)
60
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 2021
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

Children with Down syndrome aged 2-17 years who are seen through the Down syndrome clinic at Oregon Health and Science University who either have a recently completed sleep study (within the past 6 months and no surgical treatment for OSA since then) or who will be having a sleep study.

Exclusion Criteria:

  • Presence of tracheostomy
  • Presence of subglottic or tracheal stenosis
  • Severe cardiopulmonary disease requiring supplemental oxygen
  • Parents or caregivers who are unable to read written English or Spanish
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE
Contact: Eleni O'Neill 503-494-3569 oneilele@ohsu.edu
Contact: Derek Lam, MD 503-494-9419 lamde@ohsu.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03771469
Other Study ID Numbers  ICMJE STUDY00018522
GOTOL0341A ( Other Grant/Funding Number: Amer Acad Otolaryngology Head/Neck Surgery Fdtn )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Plan Description: Only the PI and members of the study team will have direct access to identifiable private information. Subjects will be assigned a unique randomly generated study identification number and extracted datasets will be coded for use in subsequent analysis.
Responsible Party Derek Lam, MD, MPH, Oregon Health and Science University
Study Sponsor  ICMJE Oregon Health and Science University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Derek Lam, MD Oregon Health and Science University
PRS Account Oregon Health and Science University
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP