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Table 1 Demographics and baseline characteristics of patients included in this analysis

From: Clinical characteristics and surgical history of Taiwanese patients with mucopolysaccharidosis type II: data from the Hunter Outcome Survey (HOS)

Characteristic

Overall population

(N = 61)

Prospective patients

(n = 44)

Retrospective patients

(n = 17)

Age at HOS entry

 Number of patients with data available

61

44

17

 Mean (SD), years

13.5 (7.0)

13.6 (7.8)

13.4 (4.5)

 Median (P10, P90), years

13.2 (5.1, 22.4)

13.4 (4.6, 23.2)

13.0 (9.5, 19.4)

Age at onset of signs and symptoms

 Number of patients with data available

55

38

17

 Mean (SD), years

2.9 (3.2)

3.1 (3.3)

2.6 (2.8)

 Median (P10, P90), years

2.5 (0.2, 5.5)

2.5 (0.3, 5.8)

1.7 (0.1, 5.5)

Age at diagnosis

 Number of patients with data available

56

39

17

 Mean (SD), years

5.3 (5.4)

5.6 (6.0)

4.6 (3.6)

 Median (P10, P90), years

3.5 (1.2, 11.9)

3.4 (1.2, 13.3)

3.6 (0.1, 10.8)

Ethnicity

 Asian, n/N (%)

59/60 (98.3)

43/44 (97.7)

16/16 (100)

 Other, n/N (%)

1/60 (1.7)

1/44 (2.3)

0/16 (0)

Family history of MPS II, yes, n/N (%)

28/50 (56.0)

18/35 (51.4)

10/15 (66.7)

Sex, male, n/N (%)

61/61 (100)

44/44 (100)

17/17 (100)

Treated with idursulfase at any time, yes, n/N (%)

24/49 (49.0)

24/38 (63.2)

0/11 (0)

Age at idursulfase treatment start

 Number of patients with data available

24

24

N/A

 Mean (SD), years

13.4 (9.2)

13.4 (9.2)

 

 Median (P10, P90), years

12.5 (4.5, 24.9)

12.5 (4.5, 24.9)

 

Length of time on idursulfase treatment

 Number of patients with data available

24

24

N/A

 Mean (SD), months

43.8 (27.0)

43.8 (27.0)

 

 Median (P10, P90), months

41.6 (9.3, 79.3)

41.6 (9.3, 79.3)

 

Cognitive impairment at any time, yes, n/N (%)

40/56 (71.4)

24/39 (61.5)

16/17 (94.1)

Age at last visit in HOS

 Number of patients with data available

44

44

N/A

 Mean (SD), years

16.9 (8.2)

16.9 (8.2)

 

 Median (P10, P90), years

17.0 (7.3, 25.0)

17.0 (7.3, 25.0)

 

Deceased, yes, n/N (%)

25/61 (41.0)

8/44 (18.2)

17/17 (100)

Age at death

 Number of patients with data available

25

8

17

 Mean (SD), years

14.4 (4.6)

16.4 (4.3)

13.4 (4.5)

 Median (P10, P90), years

13.4 (9.5, 19.4)

16.7 (9.8, 24.1)

13.0 (9.5, 19.4)

  1. The overall population contains both prospective and retrospective patients. Patient numbers are fewer than 61 for some characteristics due to data not being entered into HOS
  2. HOS Hunter Outcome Survey, MPS II mucopolysaccharidosis type II, N/A not applicable, P10, P90 10th and 90th percentiles, SD standard deviation