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Table 1 Caregiver and trial participant demographics

From: Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II

 

Interviewed caregivers (N = 46)

 

Trial participants (N = 50)

Sex, n (%)

Male

8 (17.4)

 

50 (100.0)

Female

38 (82.6)

 

0 (0.0)

Age, mean (range)

Age at time of interview, years

38.6 (27–50)a

 

7.9 (3–17)

Age at diagnosis for all trial participants, months

–

 

24.3 (in utero–5 years)

Age at diagnosis for substudy participants, months

–

 

12.8 (in utero–20 months)

Age at trial entry for all trial participants,b years

–

 

4.0 (2–12)

Age at trial entry for substudy participants, years

–

 

2 (2)

Caregiver/participant country, n (%)

USA

 

29 (63.0)

 

Spain

 

5 (10.9)

 

Mexico

 

4 (8.7)

 

Canada

 

3 (6.5)

 

France

 

3 (6.5)

 

UK

 

2 (4.3)

 

Race/ethnicity,c n (%)

White

32 (69.6)

 

29 (58.0)

Hispanic or Latino

10 (21.7)

 

10 (20.0)

Two or more races

4 (8.7)

 

7 (14.0)

Asian

3 (6.5)

 

3 (6.0)

Black

2 (4.3)

 

–

Other (not specified)

–

 

1 (2.4)

Caregiver education,d n (%)

High school or equivalent

6 (13.0)

 

–

Some college

8 (17.4)

 

–

Associate or technical degree

4 (8.7)

 

–

College degree

12 (26.1)

 

–

Postgraduate degree

9 (19.6)

 

–

  1. aNot reported for 5 caregivers
  2. bData not available for participants outside the USA and Canada
  3. cNot reported for patients in the UK and France (collecting these data in France is against regulations). Totals were greater than 50 because Hispanic or Latino was a separate question from race
  4. dNot reported for 7 caregivers