Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS)

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

	Patients, N = 639
Age at onset of symptoms, years (n = 517)	1.5 (0.3, 4.0)
Age at diagnosis, years (n = 582)	3.1 (1.0, 6.7)
Age at first treatment, years	6.2 (2.1, 18.2)
Age at first treatment, n (%)
0–5 years	310 (48.5)
6–11 years	189 (29.6)
12–17 years	75 (11.7)
18–29 years	45 (7.0)
> 29 years	20 (3.1)
Time on treatment, months	56.3 (18.2, 97.6)
Time in HOS, months	46.8 (9.6, 93.0)
Treatment started at or after HOS entry, n (%)	233 (36.5)
Deceased, n (%)	97 (15.2)
Age at death, years (n = 97)	15.9 (10.6, 28.0)
Cognitive impairment (at any time), n (%) (n = 626)
Yes	385 (61.5)
No	241 (38.5)
Last-reported functional classification, n (%) (n = 467)
Normal	179 (38.3)
Borderline	51 (10.9)
Educable	40 (8.6)
Trainable	71 (15.2)
Profoundly impaired	126 (27.0)

Data are presented as median (10th, 90th percentiles) unless stated otherwise. Data were available for all patients included in the analysis (N = 639) unless stated otherwise. HOS, Hunter Outcome Survey

ISSN: 1750-1172