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Table 3 Clinical studies assessing the impact of ERT on PROs in MPS patients

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

Reference MPS type Treatment Comparator N Mean age (years)a Study duration PRO instrument
[33] I
83 % Hurler-Scheie, 13 % Scheie
iv laronidase
(0.58 mg/kg/week)
Placebo 45 15.6 26 weeks (C)HAQ
[23] I iv laronidase
(0.58 mg/kg/week)
/ 45 15.7 3.5 year
(extension of [66]
(C)HAQ
[52] I iv laronidase
(0.58 mg/kg/week)
/ 5 12.0 6 year Modified MPS HAQ
[53] I
Scheie, Hurler- Scheie
iv laronidase
(0.58 mg/kg/week)
/ 7 16.3 52–208 weeks MPS HAQ
[55] II iv idursulfase
(0.5 mg/kg/week)
/ 94 14.5 2 years extension (C)HAQ
[32, 45] IVA iv elosulfase alfa
(2.0 mg/kg every other week or weekly)
Placebo 176 15.3 and 13.1 24 weeks MPS HAQ
[29] IVA iv elosulfase alfa
(2.0 or 4.0 mg/kg/week)
/ 25 13.7 27 weeks APPT
[24] VI iv galsulfase
(1.0 or 2.0 mg/kg/week)
/ 5 11.0 48 weeks (C)HAQ
[25] VI iv galsulfase
(1.0 mg/kg/week)
/ 10 12.7 48 weeks (C)HAQ
[26] VI iv galsulfase
(1.0 mg/kg/week)
Placebo 39 13.7 24 weeks Joint pain and stiffness, physical energy level
[56] VI iv galsulfase
(1.0 mg/kg/week)
/ 9 NA 2 years (C)HAQ
[5] VI iv galsulfase
(1.0 mg/kg/week)
/ 55 12.0 6.8 ± 2.2 years (C)HAQ
[44] VI iv galsulfase
(1.0 mg/kg/week)
/ 8 6.8 1.0–4.5 years TAPQOL/TACQOLb
  1. aMean age at baseline from all patients or from ERT group
  2. bThe TAPQOL was completed by the parents of four patients <6 years, TACQOL was completed by seven parents of patients ≥6 years
  3. NA not available