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Table 1 PROs used in patients with MPS

From: Health-related quality of life in mucopolysaccharidosis: looking beyond biomedical issues

Name questionnaire Acronym Age range (yrs) Assessment of Completed bya Reference
Symptom PROs
 Pain Visual Analog Scalesb VAS ≥8 Pain intensity Patient  
 Adolescent Pediatric Pain Tool APPT 8–17 Pain location, description and intensity Patient [60, 61]
 Brief Pain Inventory Short Form BPI-SF Adults Severity of pain, impact of pain on daily function, location of pain, use of pain medications, amount of pain relief Patient [30, 62]
 Six-face Faces Pain Scale-Revised FPS-R ≥8 Pain intensity Patient [15]
 Non-communicating Children’s pain Checklist-Revised NCCPC-R 3–18c Pain-associated behavior Observer [15, 63]
 Achenbach System of Empirically Based Assessment Adult Self Report ASEBA ASR 18–59 Social-adaptive and psychological symptoms Patient [30]
 Achenbach System of Empirically Based Assessment Older Adult Self Report ASEBA OASR ≥60 Social-adaptive and psychological symptoms Patient [30]
 Yatabe-Guilford Personality testd Y-G test NA personality and psychiatric aspects Patient [31]
 Tree-drawing test (Baum test) TDT NA personality and psychiatric aspects Patient [31]
 General Health Questionnaire 60 GHQ-60 Adolescents and adults Mental health Patient [31]
 State-Trait Anxiety Inventory STAI Adolescents and adults Anxiety Patient [31]
Functioning PROs
 Health Assessment Questionnaire HAQ >18 Functional capacity and independence in activities of daily living, pain, overall well-being Patient [13, 64]
 Childhood Health Assessment Questionnaire CHAQ ≤18 Patient [13, 65]
 Mucopolysaccharidosis Health Assessment Questionnaire MPS HAQ Children and adults Self-care, mobility skills, extent of caregiver assistance in performing activities Patient [32]
 Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale HS-FOCUS Children (>12) and adults Impact of MPS II on function Patient [27]
 Modified version of the Functional Independence Measuree FIM Children and adults Physical and cognitive disability Observer [31, 34]
 Pediatric Evaluation of Disability inventory PEDI 0.5–7.5 Capability and performance in self-care, mobility and social function Patient or parentf [66, 67]
 Vineland Adaptive Behavior Scales VABS Children and adults Adaptive behavior Parent/caregiver [36, 37, 39, 40]
 Behavior Assessment System for Children BASC Children Emotional adjustment and adaptive behavior Patient and/or parent [37]
 Scales of Independent Behavior-Revised SIB-R Infancy-80+ Adaptive behavior Patient [68]
Health-related quality of life
 EuroQol 5D EQ-5D Versions for children (≥8) and adults (≥16) Physical and mental health Patient [28, 69]
 Short form-36 SF-36 ≥16 Physical and mental health Patient [15, 30, 70]
 Health Utilities Index HUI ≥5 Impact of disease and therapy Patient or parent [27]
 Pediatric Quality of Life inventory PedsQL Children physical, emotional, social, and school functioning Patient or parent [15, 43, 71]
 TNO-AZL Preschool children Quality of Life TAPQOL 0.5–5 Physical, social, cognitive, and emotional functioning Parent [72]
 TNO-AZL Children Quality of Life TACQOL 6–15 Health status and children’s subjective emotional appraisal of their health Patient or parent [73]
 Childhood Health Questionnaire CHQ 5–18 functional capacity and independence in activities of daily life Patient and parent [27, 37, 39]
Impact on family/caregivers
 Pediatric Quality of Life inventory Family Impact Module PedsQL Family Impact Module Children Parent’s problems in physical, emotional, social, and cognitive functioning, communication, worry, and problems specific to the family’s daily activities and family relationships Parent [74]
 Zarit Burden Interview ZBI Adult patients Burden of caring on relationship, emotional well-being, social and family life, finances, control over one’s life Parent/caregiver [8]
  1. aIn MPS studies
  2. bPain VAS scores that have been used in MPS patients are included in the HAQ, CHAQ, EQ-5D, APPT
  3. cPatients who are unable to speak because of intellectual impairments or disabilities
  4. dJapanese version of the Guilford test
  5. eAdapted for patients with MPS
  6. fNormally completed by parent or observer
  7. NA not available