Table 4

Multivariate logistic regression analysis for binary endpoints

Endpoint, predictor, and cutoffNo (%)%OR95% CIP
Neurological endpoints      
 Hydrocephalus (presence) 10 (6)     
 Cerebral atrophy (presence) 56 (34)     
  Age at HCT      
   <16 months*  23 1   
   ≥16 months*  46 3.22 1.60-6.50 .001 
Orthopedic endpoints      
 Thoracolumbar kyphosis (progression) 133 (75)     
  IDUA level      
   <Reference†  93 1   
   ≥Reference†  69 0.19 0.05-0.64 .008 
 Cervical instability (progression) 18 (9)     
  IDUA level      
   <Reference†  21 1   
   ≥Reference†  5 0.24 0.09-0.69 .008 
  Follow-up age      
   <9.2 years‡  5 1   
   ≥9.2 years‡  14 3.23 1.04-10.01 .04 
 Cord compression (progression) 29 (15)     
  IDUA level      
   <Reference†  34 1   
   ≥Reference†  9 0.19 0.08-0.45 <.001 
  Age at HCT      
   <16 months*  8 1   
   ≥16 months*  21 3.02 1.17-7.76 .02 
  Follow-up age      
   <9.2 years‡  6 1   
   ≥9.2 years‡  25 5.36 1.97-14.58 .001 
 Hip dysplasia (progression) 140 (79)     
  IDUA level  95 1   
   <Reference†  75 0.17 0.04-0.74 .02 
   ≥Reference†      
  Follow-up age      
   <9.2 years‡  71 1   
   ≥9.2 years‡  87 2.50 1.13-5.53 .02 
 Genu valgum (progression) 144 (84)     
  Follow-up center   1.20 1.05-1.38 .008 
Orthopedic endpoints      
 Carpal tunnel syndrome 65 (71)     
  IDUA level      
   <Reference†  85 1   
   ≥Reference†  64 0.29 0.09-0.94 .04 
  Follow-up age      
   <9.2 years‡  50 1   
   ≥9.2 years‡  86 6.17 2.20-17.29 .001 
 Trigger finger (progression) 33 (18)     
Cardiac endpoints      
 Cardiomyopathy (progression) 8 (5)     
 Mitral valve insufficiency (progression) 71 (37)     
  IDUA level      
   <Reference†  56 1   
   ≥Reference†  30 0.36 0.18-0.74 .005 
  Age at HCT      
   <16 months*  26 1   
   ≥16 months*  47 2.46 1.30-4.65 .006 
  Follow-up age      
   <9.2 years‡  26 1   
   ≥9.2 years‡  50 2.71 1.44-5.11 .002 
 Aortic valve insufficiency (progression) 53 (29)     
  Age at HCT      
   <16 months*  19 1   
   ≥16 months*  37 2.40 1.19-4.82 .01 
  Follow-up age      
   <9.2 years‡  18 1   
   ≥9.2 years‡  41 3.32 1.66-6.64 .001 
Respiratory endpoint      
 Overnight hypoxia (progression) 13 (8)     
  IDUA level      
   <Reference†  27 1   
   ≥Reference†  3 0.10 0.03-0.35 <.001 
Respiratory endpoint      
 Overnight hypoxia (progression)      
  Follow-up age      
   <9.2 years‡  3 1   
   ≥9.2 years‡  15 4.42 1.09-17.83 .04 
Ophthalmologic endpoint      
 Corneal clouding (progression) 48 (26)     
  IDUA level      
   <Reference†  46 1   
   ≥Reference†  20 0.28 0.11-0.67 .005 
  Follow-up age      
   <9.2 years‡  6 1   
   ≥9.2 years‡  49 15.43 5.93-40.15 <.001 
 Glaucoma 12 (7)     
  IDUA level      
   <Reference†  15 1   
   ≥Reference†  5 0.28 0.09-0.95 .04 
 Cataract 16 (8)     
  TBI      
   No  4 1   
   Yes  39 12.53 3.73-42.08 <.001 
  Follow-up age      
   <9.2 years‡  1 1   
   ≥9.2 years‡  16 16.51 2.05-133.26 .008 
Audiologic endpoint      
 Hearing loss (progression) 14 (9)     
  Follow-up age      
   <9.2 years‡  4 1   
   ≥9.2 years‡  16 4.80 1.27-18.21 .02 
Endpoint, predictor, and cutoffNo (%)%OR95% CIP
Neurological endpoints      
 Hydrocephalus (presence) 10 (6)     
 Cerebral atrophy (presence) 56 (34)     
  Age at HCT      
   <16 months*  23 1   
   ≥16 months*  46 3.22 1.60-6.50 .001 
Orthopedic endpoints      
 Thoracolumbar kyphosis (progression) 133 (75)     
  IDUA level      
   <Reference†  93 1   
   ≥Reference†  69 0.19 0.05-0.64 .008 
 Cervical instability (progression) 18 (9)     
  IDUA level      
   <Reference†  21 1   
   ≥Reference†  5 0.24 0.09-0.69 .008 
  Follow-up age      
   <9.2 years‡  5 1   
   ≥9.2 years‡  14 3.23 1.04-10.01 .04 
 Cord compression (progression) 29 (15)     
  IDUA level      
   <Reference†  34 1   
   ≥Reference†  9 0.19 0.08-0.45 <.001 
  Age at HCT      
   <16 months*  8 1   
   ≥16 months*  21 3.02 1.17-7.76 .02 
  Follow-up age      
   <9.2 years‡  6 1   
   ≥9.2 years‡  25 5.36 1.97-14.58 .001 
 Hip dysplasia (progression) 140 (79)     
  IDUA level  95 1   
   <Reference†  75 0.17 0.04-0.74 .02 
   ≥Reference†      
  Follow-up age      
   <9.2 years‡  71 1   
   ≥9.2 years‡  87 2.50 1.13-5.53 .02 
 Genu valgum (progression) 144 (84)     
  Follow-up center   1.20 1.05-1.38 .008 
Orthopedic endpoints      
 Carpal tunnel syndrome 65 (71)     
  IDUA level      
   <Reference†  85 1   
   ≥Reference†  64 0.29 0.09-0.94 .04 
  Follow-up age      
   <9.2 years‡  50 1   
   ≥9.2 years‡  86 6.17 2.20-17.29 .001 
 Trigger finger (progression) 33 (18)     
Cardiac endpoints      
 Cardiomyopathy (progression) 8 (5)     
 Mitral valve insufficiency (progression) 71 (37)     
  IDUA level      
   <Reference†  56 1   
   ≥Reference†  30 0.36 0.18-0.74 .005 
  Age at HCT      
   <16 months*  26 1   
   ≥16 months*  47 2.46 1.30-4.65 .006 
  Follow-up age      
   <9.2 years‡  26 1   
   ≥9.2 years‡  50 2.71 1.44-5.11 .002 
 Aortic valve insufficiency (progression) 53 (29)     
  Age at HCT      
   <16 months*  19 1   
   ≥16 months*  37 2.40 1.19-4.82 .01 
  Follow-up age      
   <9.2 years‡  18 1   
   ≥9.2 years‡  41 3.32 1.66-6.64 .001 
Respiratory endpoint      
 Overnight hypoxia (progression) 13 (8)     
  IDUA level      
   <Reference†  27 1   
   ≥Reference†  3 0.10 0.03-0.35 <.001 
Respiratory endpoint      
 Overnight hypoxia (progression)      
  Follow-up age      
   <9.2 years‡  3 1   
   ≥9.2 years‡  15 4.42 1.09-17.83 .04 
Ophthalmologic endpoint      
 Corneal clouding (progression) 48 (26)     
  IDUA level      
   <Reference†  46 1   
   ≥Reference†  20 0.28 0.11-0.67 .005 
  Follow-up age      
   <9.2 years‡  6 1   
   ≥9.2 years‡  49 15.43 5.93-40.15 <.001 
 Glaucoma 12 (7)     
  IDUA level      
   <Reference†  15 1   
   ≥Reference†  5 0.28 0.09-0.95 .04 
 Cataract 16 (8)     
  TBI      
   No  4 1   
   Yes  39 12.53 3.73-42.08 <.001 
  Follow-up age      
   <9.2 years‡  1 1   
   ≥9.2 years‡  16 16.51 2.05-133.26 .008 
Audiologic endpoint      
 Hearing loss (progression) 14 (9)     
  Follow-up age      
   <9.2 years‡  4 1   
   ≥9.2 years‡  16 4.80 1.27-18.21 .02 

Only statistically significant results are shown.

*

Median age at HCT.

†

Lower limit of local reference.

‡

Median age at follow-up.

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