From: Health-related quality of life in mucopolysaccharidosis: looking beyond biomedical issues
Reference | MPS type | N | Age (yrs) | PRO instrument |
---|---|---|---|---|
[40] | MPS IH | 41 | NAa | VABS |
[39] | MPS IH | 47 | Mean 10.5 | VABS II CHQ |
[34] | MPS II | 27 | 5–41 | FIM |
[31] | MPS II | 10 | Mean 23.2 | FIM Personality tests: Y-G test, Tree-drawing test Psychological tests: GHQ-60, STAI |
[38] | MPS II | 50 | Mean 6.0 | Different standardized tests for cognitive, adaptive, language, and motor functions |
[35] | MPS II | 29 | Mean 11.5 | MPS HAQ |
[27] | MPS II | 96 patients & caregivers | Mean 14.2 | CHAQ HS-FOCUSb CHQ HUI3 |
MPS II | 73 patients & parents | Mean 12.5 | PedsQL Peds QL Family Impact Module VABS II | |
[37] | MPS II | 15 | 10.8 | VABS II CHQ BASC-2 |
[14] | MPS IVA | 326 | 1–73 | (C)HAQ |
[11] | MPS IVA | 325 | Mean 14.5 | MPS HAQ |
MPS IVA | 63 patients 56 caregivers | 5–17 years (N = 36) ≥18 years (N = 27) | Patients: EQ-5D, APPT (<18 years)/BPI-SF (≥18 years), fatigue question Caregivers : caregiver questionnaire, ZBI | |
[30] | MPS IVA | 20 | NA | ASEBA ASR/ OASR SF-36 BPI |
[41] | MPS VIA | 24 | 10–17 (N = 10) 18–54 (N = 14) | EQ-5D |
[13] | MPS VI | 121c | 4–56 | (C)HAQ |
[15] | MPS I, II, III, IV, VI | 55 | Median 11.3 | MPS-specific questionnaire NCCPC-Rd FPS-Rd Pain VASd SF-36d PedsQL |
[42] | MPS I, II, IVA, IVB, VI | 81 | ≥18 | EQ-5D |