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Table 1 Overview of PRO tools suitable for assessing impairment in the senses and overall health status in patients with MPS, with focus on adults and self-completion and based on the following criteria: applicability to patients with MPS, applicability to different countries and cultures, ease of use (≤10 min to complete), validated, and availability of normative data

From: Assessing the impact of the five senses on quality of life in mucopolysaccharidoses

Name tool Abbreviation Original target population Time to complete What does it measure? Validation literature Normative data literature Used in MPS? Language(s) Target age
Hearing & speech
  Attitudes Toward Loss of Hearing Questionnaire ALHQ Hearing impairment, with or without hearing aids ±10 min Attitudes toward hearing loss and hearing aids Saunders G 2005 [58] Saunders G 2005 [58] No English, Korean Adults
  Spatial Hearing Questionnaire SHQ Not disease-linked ±10 min Perception of spatial hearing abilities/disabilities Tyler RS 2009 [59] Perreau AE 2014 [60] No English + 10 translations NA
  Speech Handicap Index SHI Speech problems 5 min Speech-related problems in daily life (psychosocial and speech function) Rinkel RN 2008 [61] Rinkel RN 2008 [61] No English, French, Dutch, Portuguese, Chinese Adults
  Voice Handicap Index VHI Voice disorders 5 min Impact of voice disorders on QoL (functional, physical and emotional) Francis DO 2017 [62] Arffa RE 2012 [63] No English + 6 translations Adults
  Voice Outcome Survey VOS Uncompensated unilateral vocal fold paralysis 2–5 min Vocal status and impact on daily activities Gliklich RE 1999 [64] Gliklich RE 1999 [64]   English, Chinese Adults
 Visual Function Short Form VF-8R Cataract 5 min Functional impairment caused by vision loss Gothwal VK 2010 [65] Gothwal VK 2010 (pre- vs post-op) [65] No English, Chinese Adults
Upper limb function
  Health Assessment Questionnaire HAQ Arthritis 5 min Physical disability Bruce B 2003 [66] Bruce B 2003 [66] Yes [8]a English + 62 translations Adultsd
  Quick Disabilities of the Arm, Shoulder and Hand Questionnaire Quick-DASH Upper-extremity disorders 2 min Symptoms and ability to perform certain activities Beaton DE 2005 [67] Aasheim T 2014 [68] No 50 languages Adults
  Brief Pain Inventory Short Form BPI-SF Chronic or acute pain 5 min Pain severity and impact of pain on daily functioning Cleeland CS 2009 [69] NAc Yes [8] English + 52 translations Adults
  West Haven - Yale Multidimensional Pain Inventory WHYMPI Chronic pain 5–10 min Description of pain and how it affects the individual Kerns RD 1985 [70] No English + 9 translations Adults
Smell & taste
 Chronic Sinusitis Surveyb CSS Chronic sinusitis 5 min Health status and treatment effectiveness in chronic rhinosinusitis Gliklich RE 1995 [71]; Stavem K 2006 [72] Gliklich RE 1997 [73] No English, Norwegian, Chinese, Turkish Adults
Health status
 Five-level EuroQol five-dimensional questionnaire EQ-5D-5 L General population < 5 min Generic measure of health status for clinical and economic appraisal Herdman M 2011 [54] Szende A 2014 [74] Yes [8] > 120 languages Adults
  1. aAn adapted version, the MPS-HAQ has been developed for patients with MPS [75]; bOne question of the CSS is not applicable to MPS, but specific for allergic rhinosinusitis
  2. cThe BPI-SF was included although no normative data are available, based on its ease of use and previous use in MPS
  3. dA version of this questionnaire, i.e. the Child Health Assessment Questionnaire (CHAQ) is also available for children
  4. NA Not available