Healthy Sleep for Children With Down Syndrome
Condition: Down Syndrome; Sleep Disordered Breathing
MeSH terms: Sleep Apnea Syndromes; Down Syndrome; Syndrome
Study type: Observational

Summary: 
      The purpose of this multi-center observational study (utilizing the sites enrolling patients
      for the Pediatric Adenotonsillectomy Trial for Snoring (PATS) [1U011HL125307-O1A1]) is to
      gather data regarding children with Down syndrome (DS) and Sleep Disordered Breathing (SDB)
      referred for treatment with adenotonsillectomy to inform a future randomized controlled trial
      in this population.
    

Detailed description: 
      Down syndrome (DS) is associated with a wide range of neurobehavioral and physical
      morbidities. Obstructive sleep apnea (OSA) is prevalent in children with DS and causes a
      myriad of physiological stresses that may exacerbate neurobehavioral and cardiac morbidities.
      Improving sleep health thus provides a modifiable intervention target for improving behavior,
      cognition, quality of life, and physical health of children with DS. However, critical
      knowledge gaps limit the adoption and implementation of effective OSA interventions,
      resulting in practice variability and suboptimal treatment of OSA across the spectrum of DS.
      In particular, it is in unclear how to select candidates likely to benefit from
      adenotonsillectomy (AT) and how to utilize information from polysomnographic, clinical, and
      demographic parameters to predict children most likely to benefit from AT compared to
      alternative treatments or to watchful waiting. The goal of this study is to collect the
      necessary data to plan and execute a future pivotal RCT of the role of OSA treatment in the
      care of children with DS. This study will provide critical information on the methodological
      approaches for conducting such a randomized controlled clinical trial (RCT), informing the
      appropriate recruitment strategies, patient population and outcome measures for a future
      clinical trial. The investigators will leverage expertise in pediatric sleep medicine,
      clinical trials, neuropsychology, and DS as well as the PATS clinical trials infrastructure
      to catalyze a new DS clinical research initiative. Our proposed next steps are to enroll in
      an observational study at active PATS centers 50 children with DS who are referred for
      clinically indicated evaluation and treatment of OSA. At baseline and 6 months following AT,
      these children will undergo systematic assessments of sleep, behavior, cognition, daytime
      functioning, and quality of life, generating key data to inform the design of a later pivotal
      RCT.

      Healthy Sleep for Children with Down Syndrome (HELP-DS) intends to take advantage of a
      successful collaboration of leaders in sleep medicine, otolaryngology and clinical trials to
      efficiently leverage experiences from the The Childhood Adenotonsillectomy Trial (CHAT) as
      well as the current resources of the PATS trial to collect data for the evaluation of key
      questions needed to design a future RCT, including approaches related to the selection of
      participating sites, the patient population to receive the intervention, and outcome
      measurements.

      The primary objective of HELP-DS to determine the feasibility of recruiting and retaining
      individuals in an observational study of AT across multiple clinical sites.

      Secondary objectives are:

        1. Evaluate the feasibility of collecting a range of baseline and outcome data, and
           specifically the ability of children to perform neurobehavioral assessments (GoNoGo,
           pegboard task).

        2. Understand issues related to patient preference, equipoise, and barriers to treatment.
           The investigators will track how many children referred for AT actually undergo surgery,
           the time interval between referral and surgery, other interventions pursued, and factors
           such as insurance, socio-economic factors, and cultural norms, that associate with
           family-decision making.

        3. Examine the consistency and completeness of Healthcare Utilization (HCU) data extracted
           across our sites, describing differences by recruitment site, health, system
           characteristics, insurance-related factors, etc. The investigators will estimate
           incidence rates of HCU events for hospital admissions, emergency department/unscheduled
           office visits, specialty consultations and medication use during the 6 months following
           surgery and compare rates to those for the year prior to surgery.

      These aims have substantial public health significance given the high morbidity of Down
      syndrome and sleep-disordered breathing in children.
    

Intervention name(s): Adenotonsillectomy
Intervention type(s): Procedure
Intervention description(s): Routine AT per clinical care.
Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Dyads of caregivers and children with DS aged 3-13 years diagnosed with SDB and referred
        for treatment with adenotonsillectomy (AT) will be recruited.
      
Keywords: Pediatrics; Adenotonsillectomy