Pediatric Adenotonsillectomy Trial for Snoring (PATS)

September 11, 2024 updated by: Susan Redline, Brigham and Women's Hospital

Impact of Treatment of Mild Sleep-Disordered Breathing on Children's Health

The purpose of this study is to evaluate the effects of early adenotonsillectomy (eAT) on the behavior, sleep-disordered breathing symptoms and quality of life for children who snore, but do not have obstructive sleep apnea, as well as identify factors that moderate responses to the surgery. Half of participants will receive eAT, while the other half will be observed with watchful waiting and supportive care.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Adenotonsillectomies are performed more than 500,000 times per year in the United States, and is the most common surgery performed under general anesthesia in children. The majority of surgeries are performed for obstructed breathing rather than for infection or other indications.

The role of adenotonsillectomy (AT) in improving the 7-month neurocognitive, behavioral and health outcomes of children with frank obstructive sleep apnea (OSA) was recently addressed in the Childhood Adenotonsillectomy Trial (CHAT). The results of this rigorous, multicenter, randomized controlled trial provided critically important data indicating that adenotonsillectomy compared to watchful waiting resulted in improved behavior, quality of life, sleep-disordered breathing (SDB) symptoms and polysomnographic parameters.

However, the Childhood Adenotonsillectomy Trial addressed the role of surgery in the minority of operative candidates who have frank obstructive sleep apnea, only one form of sleep disordered breathing on a spectrum that includes a more common phenotype, primary snoring (also termed mild sleep disordered breathing (MSDB)). Mild sleep disordered breathing is characterized by snoring without frank obstruction or gas exchange abnormalities, and has a population prevalence of about 10% in children. Since most surgeries for obstructed breathing are performed for mild sleep disordered breathing rather than obstructive sleep apnea, the next logical question is whether surgery is also effective in improving symptoms and health outcomes in this large group of children.

The Pediatric Adenotonsillectomy Trial for Snoring (PATS) intends to take advantage of a successful collaboration of leaders in sleep medicine, otolaryngology and clinical trials to efficiently leverage experiences from the CHAT trial to evaluate the role of adenotonsillectomy in children with mild sleep disordered breathing while also aiming to resolve uncertainties regarding management approaches for pediatric mild sleep disordered breathing by addressing several critical issues:

  1. Assess outcomes important to children and their families, particularly patient-reported outcomes such as behavior, quality of life, and sleep disturbances.
  2. Examine differences in treatment responses among children who are at increased risk for mild sleep disordered breathing, such as pre-school children, minorities, and children with asthma or obesity.
  3. Evaluate health care utilization of children with mild sleep disordered breathing.
  4. Assess moderating influences such as second hand smoke, insufficient sleep, socioeconomic status and family functioning
  5. Examine longer term (12 month) outcomes that were not feasible in the Childhood Adenotonsillectomy Trial (CHAT).

These aims have substantial public health significance given the high morbidity of sleep disordered breathing in children.

Study Type

Interventional

Enrollment (Actual)

459

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan Health System
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
      • Cleveland, Ohio, United States, 44106
        • University Hospitals-Case Medical Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
    • Texas
      • Dallas, Texas, United States, 75390
        • University of Texas Southwestern Medical Center
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Children's Hospital of The King's Daughters

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 12 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Diagnosis of mild sleep-disordered breathing (MSDB) defined as meeting all of the following criteria:

    • Caregiver report of habitual snoring that occurs most of the night on at least three nights per week, and has been present for at least three months (on average occurring > 3 nights per week or more half of sleep time) and
    • Centrally-scored polysomnogram (PSG) confirming an obstructive apnea index (OAI) <1/hour and apnea-hypopnea index (AHI) ≤3/hour and no oxygen saturation (SpO2) desaturation < 90% in conjunction with obstructive events, confirmed on PSG.
  • Tonsillar hypertrophy ≥2 based on a standardized scale of 0-4.
  • Deemed to be a candidate for AT by otolaryngologist (ENT) evaluation (i.e., no technical issues that would be a contraindication for surgery such as submucous cleft palate.)
  • Primary indication for AT is nocturnal obstructive symptoms (i.e., not recurrent infections or other indications).

Exclusion Criteria:

  • Previous tonsillectomy, including partial tonsillectomy
  • Recurrent tonsillitis that merits prompt adenotonsillectomy (AT) per the American Academy of Otolaryngology-Head and Neck Surgery Clinical Practice Guidelines (i.e., ≥7 episodes/yr in the past year; ≥5 episodes/year over the past 2 years or ≥3 episodes/yr over the past 3 years.)
  • Severe obesity (body mass index (BMI) z-score ≥3).
  • Failure to thrive, defined as either height or weight being below the 5th percentile for age and gender.

  • Severe chronic health conditions that might hamper participation or confound key variables under study, including but not limited to:

    • Severe cardiopulmonary disorders such as cystic fibrosis, and congenital heart disease.
    • Bleeding disorders
    • Sickle Cell Disease
    • Epilepsy requiring medication
    • Significant cardiac arrhythmia noted on PSG including: non-sustained ventricular tachycardia, atrial fibrillation, second degree atrioventricular block, sustained bradycardia, or sustained tachycardia.
    • Other severe chronic health problems such as diabetes, narcolepsy, and poorly controlled asthma.
    • Known genetic, craniofacial, neurological or psychiatric conditions likely to affect the airway, cognition or behavior;
    • Current use of psychotropic medication (other than medications for attention deficit hyperactivity disorder, hypnotics, antihypertensives, hypoglycemic agents including insulin, anticonvulsants, anticoagulants, or growth hormone.
    • Diagnosis of autism spectrum disorder.
    • Intellectual deficit or assigned to a self-contained classroom for all academic subjects.
    • History of severe developmental disability or Adaptive Behavior Assessment System (ABAS-3) score ≤60.
  • Children/caregivers planning to move out of the area within the year.
  • Children in foster care.
  • Children/caregivers who do not speak English or Spanish well enough to complete the neurobehavioral measures.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Watchful Waiting with Supportive Care
All Watchful Waiting with Supportive Care (WWSC) participants will receive information about healthy sleep habits for children and appropriate clinical referrals for management of co-morbidities.
Behavioral: Watchful Waiting with Supportive Care (WWSC)
Information about healthy sleep habits for children and appropriate clinical referrals for management of co-morbidities.
Experimental: Early Adenotonsillectomy
All Early Adenotonsillectomy (eAT) participants will receive information about healthy sleep habits for children, undergo adenotonsillectomy within 4 weeks of randomization and receive appropriate clinical referrals for management of co-morbidities
Procedure: Early Adenotonsillectomy (eAT)
Standard clinical adenotonsillectomy within 4 weeks post randomization in addition to information about healthy sleep habits for children and appropriate clinical referrals for management of co-morbidities.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline to 12 Months in Caregiver-reported Behavior Rating Inventory of Executive Function (BRIEF) Global Executive Composite (GEC) T Score
Time Frame: 12 months
The BRIEF GEC section comprises summary measures of behavioral regulation, emotion regulation, and cognitive regulation (BRIEF-2, for children aged 5 to 18 years) or inhibitory self-control, flexibility, and emergent metacognition (BRIEF-P, for preschool-aged children). These scores are linear transformations of the raw scores (mean = 50, sd = 10) where a higher T score indicates a child has a lower capacity to organize and self-regulate.
12 months
Change From Baseline to 12 Months in Go-No-Go (GNG) Signal Detection Parameter D-prime (d').
Time Frame: 12 months
Performance on combined Go-No-Go (GNG)/Continuous Performance Test (CPT) task was assessed by tracking accuracy responses to targets (fish) and false positive responses to non-targets (sharks). d' is computed for both portions of the task as an assessment of accuracy in making correct detections adjusting for the participant's tendency to respond to non-targets. The adjusted measure is computed by subtracting Z-scores for false positive responses from Z-scores for correct detections. Individual Z-scores were based on a child's performance within same age groups (3-4, 5-6 , and >=7 years old). Scores ranged across age groups from -0.53 to 3.99 for GNG and -0.61 to 4.35 for CPT. Higher scores reflect better discrimination of targets from non-targets. A value of 4.65 represents 100% accuracy, 0 represents chance performance, and minus scores represent more frequent detection of non-targets than targets, suggesting a child misunderstood instructions or preferred responding to non-targets.
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline to 12 Months in NIH-Toolbox 9-Hole Pegboard Dexterity Test Time
Time Frame: 12 months
Fine motor coordination assessed by the time (in seconds) it takes a child to complete the NIH-Toolbox 9-Hole Pegboard Dexterity Test. Shorter times indicate greater dexterity. The reported value is the average of the dominant and non-dominant hand scores.
12 months
Change From Baseline to 12 Months in Child Behavior Checklist (CBCL) Summary Scale T Scores
Time Frame: 12 months
Behavior assessed by the change from baseline to 12 months in the caregiver-reported Child Behavior Checklist (CBCL) overall summary score T scores. The T scores are standardized transformations of the raw score (mean = 50, sd = 10), where a higher scores indicate greater problems.
12 months
Change From Baseline to 12 Months in Pediatric Sleep Questionnaire: Sleep-Related Breathing Disorder Scale (PSQ-SRBD) Total Score.
Time Frame: 12 months
The PSQ-SRBD scale is a 22-item questionnaire which includes three subscales: snoring, daytime sleepiness, and hyperactive behaviors/inattention. The PSQ-SRBD is commonly used to assess sleep-disordered breathing (SDB) risk in pediatric patients, but is also increasingly used to assess symptom burden. Higher scores correspond to greater SDB symptoms and the total range is 0-1
12 months
Change From Baseline to 12 Months in Sleepiness Measured by Change in the Epworth Sleepiness Scale (ESS) Modified for Children Summary Score.
Time Frame: 12 months
The Epworth Sleepiness Scale (ESS) Modified for Children is an 8-item validated questionnaire which evaluates excessive daytime sleepiness. The wording and questions are revised from the original ESS to be more suitable for children. The total score has a range from 0-24 where higher values indicate greater sleepiness.
12 months
Change From Baseline to 12 Months in Pediatric Quality of Life Inventory (PedsQL) Caregiver Reported Total Score and Subscales.
Time Frame: 12 months
General quality of life assessed by caregiver reported PedsQL total score and subscores (Psychosocial Health Summary Score & Physical Health Summary Score). The PedsQL Total Score comprises performance on 4 subscales: emotional functioning, social functioning, school functioning (summarized by the Psychosocial Functioning Score) and physical functioning (summarized by the Physical Functioning score). Scores on all scales range from 0 to 100, with higher scores indicating an increased quality of life.
12 months
Change From Baseline to 12 Months in Quality of Life Survey Evaluation of Sleep-Disordered Breathing (OSA-18) Total Score.
Time Frame: 12 months
The OSA-18 is a disease-specific QOL survey that captures symptoms across five domains: sleep disturbance, physical suffering, emotional distress, daytime problems, and parent/caretaker concerns. With a Likert 7-point scale, caregivers rate the perceived frequency of 18 OSA-related problems ranging from 1 (none of the time) to 7 (all the time). Scores on each item are summed to produce a total score ranging from 18 to 126. Higher scores correspond to poorer sleep disordered breathing-related QOL, with a score greater than or equal to 60 signifying a clinically meaningful negative impact of sleep disordered breathing on QOL
12 months
Change From Baseline to 12 Months in Body Mass Index (BMI) Percentile
Time Frame: 12 months
Body Mass Index (BMI) percentile, calculated from the average of triplicate in-clinic height/weight measurements. Percentiles calculated from Centers for Disease Control and Prevention (CDC) BMI-for-age charts.
12 months
Change From Baseline to 12 Months in Mean Systolic and Diastolic Blood Pressures (mmHg) Percentile Scores.
Time Frame: 12 months
Mean of blood pressures measured in triplicate (to 1.0 mmHg) via automated oscillometric blood pressure cuff. Percentiles calculated respective to height, age and sex (PMID: 18230679).
12 months
Change From Baseline to 12 Months in Average Heart Rate
Time Frame: 12 months
Average heart rate (beats per minute) calculated from overnight polysomnography
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Brigham and Women's Hospital

Collaborators

Children's Hospital of Philadelphia
University of Michigan
Boston Children's Hospital
University of Texas Southwestern Medical Center
Children's Hospital Medical Center, Cincinnati
University of Rochester
University Hospitals Cleveland Medical Center
Children's Hospital of The King's Daughters

Investigators

  • Principal Investigator: Susan L. Furth, MD, PhD, Children's Hospital of Philadelphia
  • Principal Investigator: Susan S Redline, MD, MPH, Brigham and Women's Hospital
  • Principal Investigator: Rui Wang, PhD, Brigham and Women's Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 6, 2017

Primary Completion (Actual)

February 21, 2022

Study Completion (Actual)

February 28, 2022

Study Registration Dates

First Submitted

September 25, 2015

First Submitted That Met QC Criteria

September 25, 2015

First Posted (Estimated)

September 29, 2015

Study Record Updates

Last Update Posted (Actual)

November 15, 2024

Last Update Submitted That Met QC Criteria

September 11, 2024

Last Verified

September 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2014P001798
  • 1U011HL125307-O1A1 (Other Grant/Funding Number: NHLBI)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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