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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