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Table 1 Patient baseline characteristics

From: Clinical investigator perspectives on patient outcomes in children with neuronopathic mucopolysaccharidosis II during intrathecal idursulfase-IT treatment

 

Patients (n = 56)

Median (range) age at study baseline, years

 Phase 2/3 study

4.6 (3.1, 14.3)

 Substudy

2.6 (1.4, 3.0)

Age category at study baseline, n (%)

  ≥ 6 years

8 (14%)

 3– < 6 years

39 (70%)

  < 3 years

9 (16%)

Age at time of interview with investigator, years

 Median (range)

9 (5–18)

IDS genotype category, n (%)

 Missense class variant

26 (46%)

 Not missense class variant

30 (54%)

Treatment group,a n (%)

 Early IT

41 (73%)

 Delayed IT

15 (27%)

  1. IDS iduronate-2-sulfatase gene, IT intrathecal
  2. aThe early-IT group comprised patients who received idursulfase-IT in the phase 2/3 trial and extension, and patients who received idursulfase-IT during the substudy and extension. The delayed IT group comprised patients who initiated idursulfase-IT in the extension and did not receive idursulfase-IT during the phase 2/3 trial
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172