Multivariate Cox regression analysis for event-time endpoints
| Endpoint, predictor, and cutoff . | N (%) . | % . | HR . | 95% CI . | P . |
|---|---|---|---|---|---|
| Orthopedic endpoints | |||||
| Thoracolumbar kyphosis (surgical intervention) | 67 (34) | ||||
| Cervical instability (surgical intervention) | 9 (5) | ||||
| Cord compression (surgical intervention) | 21 (11) | ||||
| IDUA level | |||||
| <Reference* | 22 | 1 | |||
| ≥Reference* | 7 | 0.34 | 0.14-0.82 | .02 | |
| Age at HCT | |||||
| <16 months† | 5 | 1 | |||
| ≥16 months† | 16 | 2.84 | 1.02-1.41 | .04 | |
| Hip dysplasia (surgical intervention) | 71 (36) | ||||
| IDUA level | |||||
| <Reference* | 52 | 1 | |||
| ≥Reference* | 31 | 0.53 | 0.32-0.86 | .01 | |
| Genu valgum (surgical intervention) | 75 (38) | ||||
| IDUA level | |||||
| <Reference* | 54 | 1 | |||
| ≥Reference* | 33 | 0.50 | 0.31-0.81 | .005 | |
| Carpal tunnel syndrome (surgical intervention) | 89 (45) | ||||
| Age at HCT | |||||
| <16 months† | 33 | 1 | |||
| ≥16 months† | 56 | 1.72 | 1.11-2.68 | .02 | |
| IDUA level | |||||
| <Reference* | 61 | 1 | |||
| ≥Reference* | 39 | 0.58 | 0.37-0.92 | .02 | |
| Trigger fingers (surgical intervention) | 33 (18) | ||||
| Cardiac endpoints | |||||
| Cardiomyopathy (treatment) | 33 (18) | ||||
| Respiratory endpoint | |||||
| Overnight hypoxia (respiratory support) | 9 (4) | ||||
| IDUA level | |||||
| <Reference* | 12 | 1 | |||
| ≥Reference* | 2 | 0.15 | 0.03-0.78 | .02 | |
| Ophthalmologic endpoints | |||||
| Corneal clouding (surgical intervention) | 20 (10) | ||||
| Glaucoma (surgical intervention) | 11 (6) | ||||
| Cataract (surgical intervention) | 6 (3) | ||||
| TBI | |||||
| No | 0 | 1 | |||
| Yes | 22 | 40.13 | 4.66-345.72 | .001 | |
| Audiologic endpoint | |||||
| Hearing loss (hearing aids) | 59 (32) | ||||
| IDUA level | |||||
| <Reference* | 51 | 1 | |||
| ≥Reference* | 26 | 0.42 | 0.24-0.73 | .002 | |
| Endocrinologic endpoints | |||||
| Growth retardation (GH treatment) | 26 (13) | ||||
| TBI | |||||
| No | 11 | 1 | |||
| Yes | 32 | 4.82 | 1.84-12.65 | .001 | |
| Follow-up center | 1.20 | 1.02-1.41 | .03 | ||
| Hypothyroidism (treatment) | 12 (7) | ||||
| TBI | |||||
| No | 5 | 1 | |||
| Yes | 18 | 6.12 | 1.42-26.33 | .02 | |
| Follow-up center | 1.37 | 1.03-1.81 | .03 |
| Endpoint, predictor, and cutoff . | N (%) . | % . | HR . | 95% CI . | P . |
|---|---|---|---|---|---|
| Orthopedic endpoints | |||||
| Thoracolumbar kyphosis (surgical intervention) | 67 (34) | ||||
| Cervical instability (surgical intervention) | 9 (5) | ||||
| Cord compression (surgical intervention) | 21 (11) | ||||
| IDUA level | |||||
| <Reference* | 22 | 1 | |||
| ≥Reference* | 7 | 0.34 | 0.14-0.82 | .02 | |
| Age at HCT | |||||
| <16 months† | 5 | 1 | |||
| ≥16 months† | 16 | 2.84 | 1.02-1.41 | .04 | |
| Hip dysplasia (surgical intervention) | 71 (36) | ||||
| IDUA level | |||||
| <Reference* | 52 | 1 | |||
| ≥Reference* | 31 | 0.53 | 0.32-0.86 | .01 | |
| Genu valgum (surgical intervention) | 75 (38) | ||||
| IDUA level | |||||
| <Reference* | 54 | 1 | |||
| ≥Reference* | 33 | 0.50 | 0.31-0.81 | .005 | |
| Carpal tunnel syndrome (surgical intervention) | 89 (45) | ||||
| Age at HCT | |||||
| <16 months† | 33 | 1 | |||
| ≥16 months† | 56 | 1.72 | 1.11-2.68 | .02 | |
| IDUA level | |||||
| <Reference* | 61 | 1 | |||
| ≥Reference* | 39 | 0.58 | 0.37-0.92 | .02 | |
| Trigger fingers (surgical intervention) | 33 (18) | ||||
| Cardiac endpoints | |||||
| Cardiomyopathy (treatment) | 33 (18) | ||||
| Respiratory endpoint | |||||
| Overnight hypoxia (respiratory support) | 9 (4) | ||||
| IDUA level | |||||
| <Reference* | 12 | 1 | |||
| ≥Reference* | 2 | 0.15 | 0.03-0.78 | .02 | |
| Ophthalmologic endpoints | |||||
| Corneal clouding (surgical intervention) | 20 (10) | ||||
| Glaucoma (surgical intervention) | 11 (6) | ||||
| Cataract (surgical intervention) | 6 (3) | ||||
| TBI | |||||
| No | 0 | 1 | |||
| Yes | 22 | 40.13 | 4.66-345.72 | .001 | |
| Audiologic endpoint | |||||
| Hearing loss (hearing aids) | 59 (32) | ||||
| IDUA level | |||||
| <Reference* | 51 | 1 | |||
| ≥Reference* | 26 | 0.42 | 0.24-0.73 | .002 | |
| Endocrinologic endpoints | |||||
| Growth retardation (GH treatment) | 26 (13) | ||||
| TBI | |||||
| No | 11 | 1 | |||
| Yes | 32 | 4.82 | 1.84-12.65 | .001 | |
| Follow-up center | 1.20 | 1.02-1.41 | .03 | ||
| Hypothyroidism (treatment) | 12 (7) | ||||
| TBI | |||||
| No | 5 | 1 | |||
| Yes | 18 | 6.12 | 1.42-26.33 | .02 | |
| Follow-up center | 1.37 | 1.03-1.81 | .03 |
Only statistically significant results are shown.
*Lower limit of local reference.
Median age at HCT.