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

[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/TACQOL^b