Final Research Plan
Autism Spectrum Disorder in Young Children: Screening
June 20, 2013
Recommendations made by the USPSTF are independent of the U.S. government. They should not be construed as an official position of the Agency for Healthcare Research and Quality or the U.S. Department of Health and Human Services.
The final Research Plan is used to guide a systematic review of the evidence by researchers at an Evidence-based Practice Center. The resulting Evidence Report forms the basis of the USPSTF Recommendation Statement on this topic.
The draft Research Plan was available for comment from March 28 until April 24, 2013 at 5:00 p.m., ET.
Abbreviations: ASD = autism spectrum disorder.
The analytic framework illustrates the process through which children ages 12 to 36 months may receive screening for autism spectrum disorder (ASD). Screening tests are associated with varied performance characteristics (sensitivity, specificity, negative and positive predictive value) (KQ 2), and their performance may be modified by a child's risk factors (KQ 2a), age (KQ 2b), and other characteristics of the child or family. Children at risk for ASD may progress to a diagnostic evaluation, and those identified as having ASD may receive interventions. Screening for ASD and interventions may lead to outcomes such as changes in core ASD symptoms, cognitive/intellectual functioning, language and communication skill development, challenging behavior, adaptive behavior, educational placement/achievement, and quality of life for the child and family (KQs 1 and 4) or harms (KQs 3 and 5). Outcomes of interventions may be modified by timing or ASD severity (KQs 4a and 4b). Numbers in circles within the diagram indicate the placement of key questions in relation to the process.
- Is screening for autism spectrum disorder (ASD) in children ages 12 to 36 months associated with improved short- and long-term outcomes?
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What are the performance characteristics (e.g., sensitivity, specificity, positive and negative predictive values) of ASD screening tests in children ages 12 to 36 months?
- Do certain risk factors (e.g., prematurity or having a sibling diagnosed with ASD) modify the performance characteristics of ASD screening tests?
- Does the age at which ASD screening is performed modify the performance characteristics of ASD screening tests?
- Do other characteristics of the child or family (e.g., intellectual disability, socioeconomic status, literacy level, insurance status, race/ethnicity, primary language spoken in home, limited English proficiency) modify the performance characteristics of ASD screening tests?
- What are the harms (e.g., distress, potential misclassification) of screening for ASD to the child and family?
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What is the effect of interventions targeting young children (in preschool and elementary school) on the following outcomes: core ASD symptoms, cognitive and intellectual functioning, language and communication skill development, challenging behavior, adaptive behavior, educational placement/achievement, and quality of life for the child and family?
- What is the effect of intervention timing (by age and in relation to the establishment of a definitive diagnosis) on treatment outcomes?
- What is the effect of severity of ASD on treatment outcomes?
- What are the harms of treatment for ASD in young children?
Contextual questions will not be systematically reviewed and are not shown in the Analytic Framework.
- Are there adequate screening resources (e.g., training for health care providers, reimbursement for tools and time, referral systems for positive results) to support routine screening in primary care or affiliated care systems?
- Are there adequate diagnostic and treatment resources to provide services to children who screen positive for ASD?
- To what degree does evidence indicate that children can be accurately diagnosed with ASD using the Autism Diagnostic Observation Schedule and/or expert clinical opinion? What does the current scientific literature demonstrate about the persistence of an ASD diagnosis?
- To what extent does overdiagnosis (false-positive results) exist in children who are referred to diagnostic evaluation after screening?
- To what extent does underdiagnosis exist in children who are referred to diagnostic evaluation after screening?
- Do the outcomes of screening for ASD and the efficacy and harms of interventions for ASD in young children differ by subgroups, such as racial/ethnic minority, low-income, and uninsured children?
The Research Approach identifies the study characteristics and criteria that the Evidence-based Practice Center will use to search for publications and to determine whether identified studies should be included or excluded from the Evidence Report. Criteria are overarching as well as specific to each of the key questions (KQs).
| Category | Include | Exclude |
|---|---|---|
| Definition of Disease | Clinical diagnosis of ASD | |
| Populations |
KQs 1–3: Young children ages 12 to 36 months undergoing screening for ASD
KQs 4, 5: Young children ages 0 to 12 years undergoing interventions for ASD |
KQs 1–3: Studies exclusively focusing on infants, older children, or adults or assessing general developmental screening
KQs 4, 5: Studies of treatments for adolescents or adults; studies not including at least one child age 4 years or younger |
| Interventions |
KQs 1–3: Tools and approaches used specifically to screen for ASD
KQs 4, 5: Interventions for ASD targeted to children ages 0 to 12 years |
KQs 1–3: Studies of screening for other conditions, general developmental screening, or genetic or biomarker screening
KQs 4, 5: Studies of interventions targeted to older children, adolescents, or adults (as these would not follow from screening); studies not including at least one child age 4 years or younger |
| Comparators |
KQs 1–3: No screening or alternate screening approaches when comparing two or more approaches
KQs 4, 5: Placebo, other intervention, or no intervention |
KQs 4, 5: Noncomparative studies |
| Outcomes |
KQ 1: Timing of referral/evaluation, diagnosis, and access to intervention; core ASD symptoms; cognitive and intellectual functioning; language and communication skill development; challenging behavior; adaptive behavior; educational placement/achievement; quality of life for child and family assessed in preschool and elementary school age groups
KQ 2: Performance characteristics of ASD screening approaches KQ 3: Harms from ASD screening (e.g., labeling, family distress) KQ 4: Core ASD symptoms, cognitive and intellectual functioning, language and communication skill development, challenging behavior, adaptive behavior, educational placement/achievement, and quality of life for child and family KQ 5: Harms of ASD treatment (e.g., worsening of behavior, adverse drug events, other unintended consequences) |
KQ 1, 4: Short-term outcomes, such as changes in joint attention
KQ 2: Studies not allowing calculation of performance characteristics |
| Setting |
KQs 1–3: Primary care settings and primary care–referable settings, early intervention and education settings
KQs 4, 5: Clinical, educational, early intervention, or home settings |
KQs 1–3: Studies not conducted in a country rated as “very high” on the Human Development Index: Andorra, Argentina, Australia, Austria, Bahrain, Barbados, Belgium, Brunei Darussalam, Canada, Chile, Croatia, Cyprus, Czech Republic, Denmark, Estonia, Finland, France, Germany, Greece, Hong Kong (China), Hungary, Iceland, Ireland, Israel, Italy, Japan, Republic of Korea, Latvia, Liechtenstein, Lithuania, Luxembourg, Malta, Netherlands, New Zealand, Norway, Poland, Portugal, Qatar, Singapore, Slovakia, Slovenia, Spain, Sweden, Switzerland, United Arab Emirates, United Kingdom, United States |
| Study Designs |
KQs 1–3 (benefits or harms of screening, screening performance): Any study design except single case reports
KQs 4, 5 (benefits or harms of interventions): Any comparative study design targeting young children with ASD, including at least 10 children with ASD and at least one child ages 12 to 36 months, and published since prior systematic review of therapies for ASD in children; good-quality systematic reviews |
KQs 1–3: Single case reports
KQs 4, 5: Single case reports; studies not including at least 10 children with ASD and at least one child age 4 years or younger; studies included in prior comprehensive systematic review |
The draft Research Plan was posted for public comment on the U.S. Preventive Services Task Force (USPSTF) Web site from March 28 to April 24, 2013. Several comments noted that child characteristics (such as race, socioeconomic status) can be important modifiers of screening test performance; the USPSTF added a key question addressing these factors. Other comments suggested time to diagnosis or intervention as outcomes and broadening the outcome of “language development” to explicitly include communication; the USPSTF changed the Research Plan to reflect these outcomes. Additional comments on data extraction and analysis, contextual issues, and discussion points did not necessitate changes to the Research Plan but will be considered in the preparation of the systematic review.