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Table 1 Prioritized symptom domains and related clinical outcome assessments

From: Caregivers' assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndrome

Domain Clinical outcome assessment and description Number of interviewees reporting on COA
Communication and relationships Vineland Adaptive Behavior Scales-3 Parent/Caregiver Form: Listening and Understanding
Caregiver-reported measure of attending, understanding, and responding appropriately to information from others [30]
9
  Vineland-3 Parent/Caregiver Form: Talking
Caregiver-reported measure of using words and sentences to express oneself verbally to others [30]
9
  Vineland-3 Parent/Caregiver Form: Relating to Others
Caregiver-reported measure of responding and relating to others, including friendships, caring, social appropriateness, and conversation [30]
Not used for interviews
Eating/swallowing Child Oral and Motor Proficiency Scale (ChOMPS)
Caregiver-reported assessment of eating, drinking, and related skills in children 6 months to 7 years of age [31]
10
Sleep Children’s Sleep Habits Questionnaire (CSHQ)
Caregiver–reported questionnaire to examine sleep behavior in young children [32]
10
  Sleep Diary
Caregiver-reported diary completed daily to gather information about sleep patterns
Not used for interviews
Getting around Timed Four Stair Climb
Clinician assessed time to climb 4 standard-sized stairs [33]
19
  Floor to Stand Test
Clinician assessed time to stand from a supine position [33]
Not used for interviews
  Vineland-3 Parent/Caregiver Form: Using Large Muscles
Caregiver-reported physical skills in using arms and legs for movement and coordination in daily life [30]
Not used for interviews
  Vineland-3 Parent/Caregiver Form: Using Small Muscles
Caregiver-reported physical skills in using hands and fingers to manipulate objects in daily life [30]
Not used for interviews
Pain Non-Communicating Child’s Pain Checklist-Revised (NCCPC-R)
Caregiver-reported pain measurement tool specifically designed for children with cognitive impairments [34]
9
Behavior Aberrant Behavior Checklist (ABC)
Caregiver-reported problematic behaviors for people with developmental disabilities [35]
10
  Vineland-3 Parent/Caregiver Form: Caring for Home
Caregiver-reported assessment of household tasks such as cleaning up after oneself, chores, and food preparation [30]
Not used for interviews
Adapting Vineland-3 Parent/Caregiver Form: Adapting
Caregiver-reported behavioral and emotional control in new/different situations involving other people [30]
9
Caring for self Vineland-3 Parent/Caregiver Form: Caring for Self
Caregiver-reported self-sufficiency in such areas as eating, dressing, washing, hygiene, and health care [30]
Not used for interviews
  1. Column 1 shows the 8 symptom domains included in this study
  2. Column 2 represents the 15 assessments assessed by all the participants in the asynchronous focus group and provides a description of the outcome assessment
  3. Column 3 shows the number of interviewees who reviewed each COA that was selected for the interviews