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Table 3 Ab status at end of study by genotype class (n = 27) a

From: Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome)

 

Mutation group, n (%)

Statistical comparison

Ab status

CD/LR (n = 8)

FS/SSM (n = 6)

MS (n = 13)

CD/LR vs FS/SSM b

CD/LR vs MS b

FS/SSM vs MS b

Ab+

8 (100)

6 (100)

4 (31)

p = 1.000

p = 0.005

p = 0.011

PAb+

8 (100)

4 (67)

3 (23)

p = 0.165

p = 0.001

p = 0.129

NAb+

7 (88)

5 (83)

2 (15)

p = 1.000

p = 0.002

p = 0.010

PNAb+

7 (87.5)

4 (66.7)

2 (15.4)

p = 0.538

p = 0.002

p = 0.046

  1. Ab+, antibody positive; CD/LR, complete deletion/large rearrangement; FS/SSM, frameshift/splice site mutation; MS, missense mutation; NAb+, neutralizing antibody positive, PAb+; persistently antibody positive; PNAb+, persistently neutralizing antibody positive.
  2. aGenotype data not available for one patient enrolled in HGT-ELA-038.
  3. b p-value from Fisher’s exact test.
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172