Table 1.
Patient descriptives: values are mean (sd) or N (%) where indicated. Values represent measurements from baseline (second visit for all except first for one participant). Age split at 12 and PSS score at 9. Because medical symptoms are part of the PSS score, only means by age were calculated.
| Covariate | MPS II |
Age < 12 |
Age ≥ 12 |
PSS < 9 |
PSS ≥ 9 |
|---|---|---|---|---|---|
| (N = 15) | (N = 10) | (N = 5) | (N = 9) | (N = 6) | |
| Age (years) | 10.84 (3.09) | 9.20 (1.93) | 14.11 (2.22) | 9.75 (3.02) | 12.47 (2.61) |
| IQ | 103 (15.57) | 104 (13.84) | 100 (20.15) | 108 (10.65) | 94.67 (19.34) |
| PSS | 7.93 (1.49) | 7.40 (1.43) | 9.00 (1.00) | 7.00 (1.12) | 9.33 (0.52) |
| Number of patients with hearing loss | 14 (93%) | 9 (90%) | 5 (100%) | – | – |
| Number of patients using hearing aids | 10 (67%) | 6 (60%) | 4 (80%) | – | – |
| Number of patients with short stature | 8 (53%) | 4 (40%) | 4 (80%) | – | – |
| Number with joint contractures | 14 (93%) | 9 (90%) | 5 (100%) | – | – |
| Number with hydrocephalus | 3 (20%) | 2 (20%) | 1 (20%) | – | – |
| Age at first ERT treatment | 7.11 (3.27) | 5.65 (2.69) | 10.03 (2.07) | – | – |
| Years of ERT treatment | 3.73 (1.88) | 3.55 (1.95) | 4.10 (1.07) | – | – |
| CHQ (T score)a | |||||
| – Missing CHQ | 4 (26.67%) | 4 (40.00%) | 0 (0.00%) | 2 (22.22%) | 2 (33.33%) |
| – Psychosocial summary score | 49.52 (10.95) | 45.99 (12.93) | 53.74 (7.07) | 53.55 (8.08) | 42.45 (12.84) |
| – Physical summary score | 35.76 (17.86) | 43.45 (9.45) | 26.53 (22.15) | 43.15 (10.74) | 22.82 (21.95) |
| VABS-II (none missing) (standard score)a | |||||
| – Composite | 98.40 (14.57) | 98.60 (13.41) | 98.00 (18.38) | 105 (12.78) | 88.67 (11.98) |
| – Daily living skills | 95.27 (16.26) | 97.40 (16.50) | 91.00 (16.69) | 102 (15.09) | 85.33 (13.37) |
| – Communication | 100 (15.67) | 98.00 (13.84) | 105 (19.63) | 105 (12.39) | 92.83 (18.09) |
| – Socialization | 102 (13.85) | 104 (12.92) | 98.40 (16.53) | 108 (12.64) | 92.67 (10.60) |
| BASC-P: parent report (none missing) (T score)a | |||||
| – Depression | 53.47 (12.61) | 53.90 (13.84) | 52.60 (11.15) | 47.11 (7.79) | 63.00 (12.90) |
| – Withdrawal T-score | 56.53 (14.74) | 54.80 (16.36) | 60.00 (11.64) | 47.67 (8.17) | 69.83 (12.16) |
| – Attention problems T-score | 49.27 (9.15) | 51.00 (10.37) | 45.80 (5.36) | 49.56 (10.03) | 48.83 (8.57) |
| – Adaptive skills T-score | 48.20 (8.48) | 47.00 (8.81) | 50.60 (8.14) | 51.00 (7.92) | 44.00 (8.10) |
| BASC: self-report (T-score)a Clinical scales (higher scores are more abnormal) |
|||||
| – Missing values for scales below | 3 (20.00%) | 2 (20.00%) | 1 (20.00%) | 3 (33.33%) | 0 (0.00%) |
| – Social stress T-score | 47.08 (9.09) | 48.50 (9.70) | 44.25 (8.22) | 41.83 (5.91) | 52.33 (8.98) |
| – Anxiety T-score | 49.08 (8.17) | 48.00 (8.86) | 51.25 (7.23) | 43.00 (4.38) | 55.17 (6.24) |
| – Depression T-score | 45.54 (4.33) | 47.25 (4.59) | 42.80 (2.05) | 43.71 (2.06) | 47.67 (5.47) |
| – Missing values for scales below | 4 (26.67%) | 3 (30.00%) | 1 (20.00%) | 4 (44.44%) | 0 (0.00%) |
| – Locus of control T-score ≥ 8b | 46.91 (9.43) | 48.86 (9.65) | 43.50 (9.26) | 42.40 (5.50) | 50.67 (10.78) |
| – Sense of inadequacy T-score ≥ 8 | 50.09 (6.67) | 49.29 (5.94) | 51.50 (8.58) | 46.60 (5.64) | 53.00 (6.42) |
| – Attention problems T-score ≥ 8 | 49.36 (10.29) | 52.29 (11.48) | 44.25 (5.85) | 48.20 (9.26) | 50.33 (11.86) |
| – Hyperactivity T-score ≥ 8 | 50.45 (7.53) | 50.14 (8.32) | 51.00 (7.07) | 46.40 (6.84) | 53.83 (6.77) |
| Adaptive scales (higher scores are better) | |||||
| – Missing values for scales below | 3 (20.00%) | 2 (20.00%) | 1 (20.00%) | 3 (33.33%) | 0 (0.00%) |
| – Interpersonal relations T-score | 51.00 (11.31) | 48.62 (12.68) | 55.75 (6.95) | 55.50 (7.20) | 46.50 (13.44) |
| – Self-esteem T-score ≥ 8 | 53.83 (5.57) | 51.29 (4.75) | 57.40 (4.93) | 54.50 (4.93) | 53.17 (6.55) |
| – Missing values for scale below | 4 (26.67%) | 3 (30.00%) | 1 (20.00%) | 4 (44.44%) | 0 (0.00%) |
| – Relations with parents T-score ≥ 8 | 51.82 (10.00) | 48.00 (8.89) | 58.50 (9.04) | 47.40 (9.32) | 55.50 (9.73) |
| – Missing values for scale below | 5 (33.33%) | 4 (40.00%) | 1 (20.00%) | 4 (44.44%) | 1 (16.67%) |
| – Self-reliance T-score ≥ 8 | 50.60 (14.60) | 45.17 (13.88) | 58.75 (13.07) | 50.20 (12.87) | 51.00 (17.71) |
a
T score (mean 50, standard deviation 10); standard score (mean 100, standard deviation 15).
b
For items marked > 8, item is not give under age 8.