Treatment of Obstructive Sleep Apnea With Personalized Surgery in Children With Small Tonsils (TOPS-ST)

Oregon Health & Science University (OHSU)

Status: Enrolling

Conditions: Otolaryngological Disease; Obstructive Sleep Apnea

Treatments: Procedure: Adenotonsillectomy; Procedure: DISE-Directed Surgery

Study type: Interventional

Funder types: Other

Identifiers

NCT06258837

STUDY00027270

Details and patient eligibility

About

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.

Full 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.

Enrollment: 240 estimated patients

Sex: All

Ages: 2 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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240 participants in 2 patient groups

Drug-Induced Sleep Endoscopy

Experimental group

Description:

DISE will be performed at the time of surgery under the same sedation. The decision on specific surgical approach will be made at that time based on DISE findings. Prior to intubation, patients will be sedated with either a propofol infusion or a combination of ketamine and dexmedetomidine. Once adequate sedation is achieved, endoscopy will be performed using a flexible endoscope advanced through the nose. The nasal airway will be evaluated on both sides, then the endoscope will be advanced into the pharynx. The degree of obstruction is scored on a 3-point rating scale. Participants randomized to DISE-directed surgery will undergo one or more potential procedures in a single surgery. Caregivers will be consented for all possible procedures with the understanding that only those needed based on DISE will be performed. Importantly, these procedures are all established treatments with published outcomes data.

Treatment:

Procedure: DISE-Directed Surgery

Adenotonsillectomy

Active Comparator group

Description:

Adenotonsillar hypertrophy is the most common risk factor for OSA in children, and adenotonsillectomy (AT) is the first line treatment. An adenotonsillectomy is an operation to remove both the adenoids and tonsils.

Treatment:

Procedure: Adenotonsillectomy