Clinical Trial Details
— Status: Active, not recruiting
Administrative data
| NCT number |
NCT05154214 |
| Other study ID # |
STUDY00023287 |
| Secondary ID |
|
| Status |
Active, not recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
November 9, 2021 |
| Est. completion date |
January 1, 2025 |
Study information
| Verified date |
February 2024 |
| Source |
Oregon Health and Science University |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
The purpose of this study is to prospectively compare the effectiveness of a novel
personalized approach to the surgical treatment of obstructive sleep apnea (OSA) in children,
drug induced sleep endoscopy (DISE) directed surgery versus the standard adenotonsillectomy
(AT). This will also serve to test the feasibility of recruiting families for a future
randomized protocol comparing the same surgical techniques. It is the investigators' central
hypothesis that a personalized 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 small tonsils or Down syndrome will be superior to 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 will study children aged
2 to 18 years with clinically small tonsils (Brodsky score 1+ or 2+ on a scale 1+ to 4+) OR
Down syndrome.
Description:
Obstructive sleep apnea (OSA) is defined as repetitive obstruction of the upper airway during
sleep and has been estimated to affect 1-6% of the general pediatric population. Untreated
OSA in children has been associated with childhood hypertension, autonomic dysfunction,
attention-deficit/hyperactivity disorder, neurobehavioral impairment, and poor quality of
life. These sequelae contribute to a 226% increase in health care utilization among children
with OSA compared to controls, primarily in the form of increased hospitalizations, emergency
department visits, and medication use. Adenotonsillar hypertrophy is considered the most
common risk factor for OSA in children, and adenotonsillectomy (AT) is the first line
treatment.
Although AT is the recommended first line treatment for pediatric OSA in treatment
guidelines, multiple studies have demonstrated that AT is not always curative, with estimates
of persistent OSA after AT ranging from 25 to 75%. Many risk factors for AT failure have been
reported, but the impact of tonsil size on AT outcome remains a controversial topic. Not
every child with enlarged tonsils will have OSA, and conversely, there are many children with
proven OSA who do not have clinically large tonsils or adenoids. The prevalence of small
tonsils (1+ or 2+ on Brodsky tonsil scoring scale of 1+ to 4+) has been reported to be 67-70%
among children with symptoms of OSA presenting to either the emergency department or a
pediatric pulmonology clinic. Clinic assessment of tonsil size during an awake examination
has not consistently correlated with either baseline disease severity or response to AT. The
outcome of AT in children with small tonsils (Brodsky 1+ or 2+) remains unknown and clinical
decisions in children with small tonsils are not evidence-based.
Patients with Down Syndrome have a higher incidence of OSA than the general pediatric
population, with rates of 30-60%, resulting in increased morbidity and decreased quality of
life for affected individuals. Children with Down Syndrome often undergo T&A for obstructive
sleep apnea; however 30-50% will have persistent obstructive sleep patterns requiring
continuous positive pressure airway support (CPAP) or tracheotomy. Persistent obstruction is
attributed to anatomic and physiologic differences in this population, including reduced
muscular tone, macroglossia, maxillary hypoplasia, and lingual tonsil hypertrophy.
Drug-induced sleep endoscopy (DISE) enables direct observation of the sites and patterns of
obstruction during sedated sleep. DISE increasingly helps guide surgical decision-making in
adult OSA, and the investigators hypothesize DISE can guide surgical decisions in pediatric
OSA and improve outcomes. Towards that end, the study team has developed and validated the
DISE Rating Scale in children. Furthermore, the investigators have demonstrated 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. This preliminary data
and others' studies have identified non-adenotonsillar obstruction can also occur at the
nose, palate, base of tongue, and larynx. DISE findings in surgically naïve children and the
outcomes of procedures based on those findings have been reported in mostly small
observational case series. The surgical plan may be modified from the standard AT in up to
77% of children based on DISE findings. These alternative procedures can be less invasive
(e.g., only isolated adenoidectomy or isolated tonsillectomy) or include procedures to
address other anatomic sites in addition to or instead of the tonsils and adenoids. The most
commonly reported non-adenotonsillar sites of obstruction are the nose, velopharynx (soft
palate), tongue base, and the larynx. DISE-directed surgery at these sites 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. This study
will inform a future randomized trial that has the potential to change the paradigm of
treating OSA in children with small tonsils or Down syndrome, thereby improving outcomes, and
reducing inappropriate AT and need for secondary sleep surgery.
