| NB-2004 (n = 32) | NB-2009 (n = 27) | P value |
---|---|---|---|
Endocrinopathy | |||
 Hypothyroidism, n (%) | 12 (37.5) | 11 (40.7) | 0.799 |
 Growth hormone deficiency, n (%) | 9 (28.1) | 1 (3.7) | 0.016 |
 Glucocorticoid deficiency, n (%) | 1 (3.1) | 0 (0.0) | 0.542 |
 Sex hormone deficiency, n (%) | 1/2a (50.0) | 1/3a (33.3) | 0.700 |
Height, Z-score, median (range) | −1.46 (−5.66–0.93) | −0.65 (−2.78–1.40) | 0.004 |
Weight, Z-score, median (range) | −0.84 (−2.91–0.75) | −0.32 (−2.15–2.08) | 0.014 |
BMI, Z-score, median (range) | −0.38 (−2.58–1.62) | −0.42 (−1.86–2.68) | 0.166 |
Hearing loss, n (%) | 24 (75.0) | 16 (59.3) | 0.197 |
Cataract, n (%) | 9 (28.1) | 0 (0) | 0.002 |
Chronic lung disease, n (%) | 2 (6.3) | 0 (0.0) | 0.673 |
Renal | |||
 Glomerulopathy, n (%) | 18 (56.3) | 4 (14.8) | 0.001 |
 Tubulopathy, n (%) | 20 (62.5) | 17 (63.0) | 0.971 |
Cardiac dysfunction, n (%) | 0 (0.0) | 0 (0.0) | 1.000 |
Presence of grade 3 dysfunction, n (%) | 4 (12.5) | 2 (7.4) | 0.678 |
Number of dysfunctions/patient, median (range) | 3 (0–7) | 2 (0–4) | 0.003 |