Obstructive Sleep Apnea, Otolaryngological Disease
Conditions
Brief summary
The purpose of this study is to compare the effectiveness of a novel personalized surgical approach to the standard AT in children with small tonsils (ST). This will be accomplished by randomizing children with ST and OSA to one of these two treatments and comparing outcomes after 6 months. It is the investigators' central hypothesis that a personalized drug-induced sleep endoscopy (DISE)-directed surgical approach that uses existing procedures to address the specific fixed and dynamic anatomic features causing obstruction (ie, anatomic endotypes) in each child with ST will perform better than the currently recommended standard first line approach of AT. This novel approach may improve OSA outcomes and reduce the burden of unnecessary AT or secondary surgery for persistent OSA after an ineffective AT. To test this hypothesis, the investigators propose to study children aged 2-17 years with small tonsils and OSA.
Detailed description
Obstructive sleep apnea (OSA) is common in children with an estimated prevalence of 1-6%. Untreated pediatric OSA is associated with hypertension, autonomic dysfunction, attention-deficit / hyperactivity disorder, neurocognitive deficits, poor school performance, poor quality of life, and a \>200% increase in health care utilization compared to controls. Adenotonsillar hypertrophy is the primary risk factor for pediatric OSA, and adenotonsillectomy (AT) is the recommended first line treatment. However, the prevalence of persistent OSA after AT has been reported to be at least 20% among children with large tonsils, indicating that AT may not be the optimal intervention in all cases. Although the prevalence of small tonsils in children with OSA symptoms is up to 70%, there is currently no clear evidence on the outcome of AT in children with small tonsils. It has been shown that clinic assessments of tonsil size do not correlate with OSA severity or response to AT, leaving confusion about how best to treat OSA in children with small tonsils. This knowledge gap represents an opportunity to apply a personalized treatment approach and improve outcomes. DISE entails passage of a flexible endoscope through the nose into the pharynx and enables direct observation of the sites and patterns of upper airway obstruction during sedated sleep. DISE was developed to guide surgical decisions in adult OSA, and in recent years has also been used to design personalized surgical interventions in children. To help standardize DISE assessments, the investigators previously developed and validated the DISE Rating Scale in children based on ordinal ratings of maximal airway obstruction (none, partial, complete) at six anatomic sites from the nose to the larynx. The investigators also demonstrated that DISE ratings of adenotonsillar obstruction during sedated sleep are strongly associated with both OSA severity and response to AT, unlike clinic assessments of tonsil size. The investigators' preliminary data and other published studies have identified a high prevalence of non-adenotonsillar obstruction that can occur at the nose, palate, base of tongue, and larynx. Surgery that is tailored to the specific anatomic sites of obstruction observed during DISE may improve pediatric OSA outcomes, but existing studies are small, uncontrolled case series in heterogeneous study populations. There are no trials directly comparing AT and DISE-directed surgery in the common scenario of surgically naïve children with small tonsils. The purpose of this study is to compare the effectiveness of a novel personalized surgical approach to the standard AT in children with small tonsils (ST). This will be accomplished by randomizing children with ST and OSA to one of these two treatments and comparing outcomes after 6 months. It is the investigators' central hypothesis that a personalized drug-induced sleep endoscopy (DISE)-directed surgical approach that uses existing procedures to address the specific fixed and dynamic anatomic features causing obstruction (i.e., anatomic endotypes) in each child with ST will perform better than the currently recommended standard first line approach of AT. This novel approach may improve OSA outcomes and reduce the burden of unnecessary AT or secondary surgery for persistent OSA after an ineffective AT. To test this hypothesis, the investigators propose to study children aged 2-17 years with small tonsils and OSA.
Interventions
PROCEDUREDISE-Directed Surgery
Participants randomized to DISE-directed surgery will undergo one or more potential procedures in a single surgery (i.e. DISE and subsequent sleep surgery performed) concurrently under the same general anesthetic), depending on anatomic assessment.
PROCEDUREAdenotonsillectomy
Tonsil and/or adenoid removal
Sponsors
Oregon Health and Science University
University of Michigan
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Masking description
It is not practical or feasible to maintain blinding among the surgeons performing the procedures or the clinical staff caring for patients in the perioperative setting. Given this, it is unlikely that blinding could be maintained among parents or caregivers of patients, therefore there will be no attempt to blind caregivers to the treatment assignment. However, the sleep medicine collaborators who will be reviewing and scoring postoperative PSGs will be instructed not to review the medical record so as to ensure blinding to treatment assignment.
Intervention model description
Participants will be recruited from pediatric otolaryngology clinics. The study will be introduced to caregivers of children with small tonsils and a history of sleep disordered breathing/OSA who are referred for surgery and meet the inclusion and exclusion criteria noted. If current or recent PSG testing demonstrates OSA, they will be invited to participate. Randomization will be done. Patients will be randomized to either the personalized DISE-directed surgery or the standard AT after collection of baseline measures and pre-op PSG. Allocation will be made by using a computer-generated randomization scheme. Caregivers will be advised of the outcomes and risks of different surgical procedures included in DISE-directed surgery and informed consent will be obtained. All patients will be planned for overnight observation to monitor for safety and perioperative complications.
Eligibility
Sex/Gender
ALL
Age
2 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
To be in the study: * Child has a diagnosis of moderate to severe OSA diagnosed by PSG (oAHI ≥ 5). * Child age is 2.00 to 17.99 years of age. * Child has small tonsils (Brodsky 1+ or 2+) noted during routine physical exam. * Caregiver can provide signed and dated consent and is 18 years of age or older at the time of consent. * Caregiver can speak, read, and write in English or Spanish. * Caregiver is primary caretaker of the child. * Child is not expecting their own child. * Child is eligible for surgical treatment Cannot be in the study if: * Child has history of previous tonsillectomy, tonsillotomy, or partial tonsillectomy. * Child has any contraindication to surgery (e.g. bleeding disorders). * Child has significant cardiopulmonary comorbidity besides OSA requiring supplemental oxygen, subglottic or tracheal stenosis, tracheostomy dependence. * Child has a genetic abnormality, Down syndrome, neuromuscular disorder, craniofacial anomaly. * Caregiver is unwilling or unable to comply with study procedures. * Child is or plans to become a parent themselves.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline Polysomnography Measures: Obstructive Apnea-Hypopnea Index (oAHI) at 6 months | 6 month follow up sleep study (after surgery) | Objective results from sleep studies (polysomnography): Obstructive Apnea-Hypopnea Index (oAHI): 6 months follow up sleep study difference from baseline sleep study. Higher scores indicate higher disease burden. Scores can range from zero to two hundred. |
| Change from Baseline Polysomnography Measures: Total Apnea-Hypopnea Index (AHI) at 6 months | 6 month follow up sleep study (after surgery) | Objective results from sleep studies (polysomnography): Total Apnea-Hypopnea Index: 6 months follow up sleep study difference from baseline sleep study. Higher scores indicate higher disease burden. Scores can range from zero to two hundred. |
| Change from Baseline Polysomnography Measures: REM Apnea-Hypopnea Index (REM AHI) at 6 months | 6 month follow up sleep study (after surgery) | Objective results from sleep studies (polysomnography): REM Apnea-Hypopnea Index REM AHI: 6 months follow up sleep study difference from baseline sleep study. Higher scores indicate higher disease burden. Scores can range from zero to two hundred. |
| Change from Baseline Polysomnography Measures: minimum Oxygen saturation (SpO2) at 6 months | 6 month follow up sleep study (after surgery) | Objective results from sleep studies (polysomnography): Minimum Oxygen Saturation (Min SpO2): 6 months follow up sleep study difference from baseline sleep study. Lower scores indicate higher disease burden. Scores range from 0-100, although neither extreme is actually seen in patients. |
| Change from Baseline Polysomnography Measures: Oxyhemoglobin desaturation ≥ 3% Index (desat index) at 6 months | 6 month follow up sleep study (after surgery) | Oxyhemoglobin desaturation ≥ 3% Index: 6 months follow up sleep study difference from baseline sleep study. Higher scores mean higher disease burden. Mild desaturation (\< 5.0 events/h), moderate desaturation (≥ 5.0 events/h and \< 10.0 events/h), and severe desaturation (≥ 10.0 events/h). |
| Change from Baseline Polysomnography Measures: Percent Total Sleep Time with ETCO2 > 50 mmHg at 6 months | 6 month follow up sleep study (after surgery) | % Total Sleep Time with ETCO2 \> 50 mmHg: 6 months follow up sleep study difference from baseline sleep study. Higher scores mean higher disease burden. Scores range from 0-100. |
| Change from Baseline Polysomnography Measures: Max End Tidal CO2 (ETCO2) at 6 months | 6 month follow up sleep study (after surgery) | Max End Tidal CO2 (ETCO2): 6 months follow up sleep study difference from baseline sleep study. Higher scores mean higher disease burden. Normal is 35-40 mmHg. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Obstructive Sleep Apnea (OSA)-18 Questionnaire score | 6 month follow up | Disease specific quality of life measure: 18 questions, scores range from 18-126; higher scores means higher disease burden |
| Change in Generic PedsQL (Pediatric Quality of Life) Questionnaire score | 6 month follow up | Generic quality of life measure: an age specific questionnaire with 23 questions (scores range from 0-100); higher scores indicate better quality of life. |
| Change in Generic PedsQL (Pediatric Quality of Life) Questionnaire answers | 6 month follow up | Quality of life: questionnaire results by individual question. Adjusted scores range from 0-100; higher scores indicate better quality of life. |
| Total Drug induced sleep endoscopy (DISE) score | At time of surgery | Subjective ratings of degree of obstruction at 6 levels of the upper airway, done by surgeon. Scores range from 0 to 12. Higher scores means more breathing obstruction, or more disease burden. |
| Adverse Events | 24 hour period after surgery | Did any adverse events occur in the post-operative time frame? This is a yes/no question, looking at the following outcomes: dehydration and poor oral intake due to post-operative pain, post-tonsillectomy hemorrhage, and respiratory compromise. |
| Change in Child Behavior Checklist (CBCL) Questionnaire answers | Baseline versus 6 month follow up | Assesses behavioral and emotional problems: questionnaire results by total score and by "syndrome scale scores". Adjusted norms scores range from 0.0-1.0; higher scores indicate greater problems. |
Countries
United States
Contacts
CONTACTEleni O'Neill
CONTACTDerek Lam, MD
PRINCIPAL_INVESTIGATORDerek Lam, MD
Oregon Health and Science University
Outcome results
None listed