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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 toolAbbreviationOriginal target populationTime to completeWhat does it measure?Validation literatureNormative data literatureUsed in MPS?Language(s)Target age
Hearing & speech
Hearing
  Attitudes Toward Loss of Hearing QuestionnaireALHQHearing impairment, with or without hearing aids±10 minAttitudes toward hearing loss and hearing aidsSaunders G 2005 [58]Saunders G 2005 [58]NoEnglish, KoreanAdults
  Spatial Hearing QuestionnaireSHQNot disease-linked±10 minPerception of spatial hearing abilities/disabilitiesTyler RS 2009 [59]Perreau AE 2014 [60]NoEnglish + 10 translationsNA
Speech
  Speech Handicap IndexSHISpeech problems5 minSpeech-related problems in daily life (psychosocial and speech function)Rinkel RN 2008 [61]Rinkel RN 2008 [61]NoEnglish, French, Dutch, Portuguese, ChineseAdults
  Voice Handicap IndexVHIVoice disorders5 minImpact of voice disorders on QoL (functional, physical and emotional)Francis DO 2017 [62]Arffa RE 2012 [63]NoEnglish + 6 translationsAdults
  Voice Outcome SurveyVOSUncompensated unilateral vocal fold paralysis2–5 minVocal status and impact on daily activitiesGliklich RE 1999 [64]Gliklich RE 1999 [64] English, ChineseAdults
Vision
 Visual Function Short FormVF-8RCataract5 minFunctional impairment caused by vision lossGothwal VK 2010 [65]Gothwal VK 2010 (pre- vs post-op) [65]NoEnglish, ChineseAdults
Touch
Upper limb function
  Health Assessment QuestionnaireHAQArthritis5 minPhysical disabilityBruce B 2003 [66]Bruce B 2003 [66]Yes [8]aEnglish + 62 translationsAdultsd
  Quick Disabilities of the Arm, Shoulder and Hand QuestionnaireQuick-DASHUpper-extremity disorders2 minSymptoms and ability to perform certain activitiesBeaton DE 2005 [67]Aasheim T 2014 [68]No50 languagesAdults
Pain
  Brief Pain Inventory Short FormBPI-SFChronic or acute pain5 minPain severity and impact of pain on daily functioningCleeland CS 2009 [69]NAcYes [8]English + 52 translationsAdults
  West Haven - Yale Multidimensional Pain InventoryWHYMPIChronic pain5–10 minDescription of pain and how it affects the individualKerns RD 1985 [70]https://www.va.gov/PAINMANAGEMENT/docs/NoEnglish + 9 translationsAdults
Smell & taste
 Chronic Sinusitis SurveybCSSChronic sinusitis5 minHealth status and treatment effectiveness in chronic rhinosinusitisGliklich RE 1995 [71]; Stavem K 2006 [72]Gliklich RE 1997 [73]NoEnglish, Norwegian, Chinese, TurkishAdults
Health status
 Five-level EuroQol five-dimensional questionnaireEQ-5D-5 LGeneral population< 5 minGeneric measure of health status for clinical and economic appraisalHerdman M 2011 [54]Szende A 2014 [74]Yes [8]> 120 languagesAdults
  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