ADA-SCID patients on ERT with PEG-ADA display reduced growth
. | Age at last follow-up, y . | PEG-ADA, y . | Percentile height, Z-score . | RANKL, pg/mL . | OPG, pg/mL . | RANKL/OPG, × 102 . |
---|---|---|---|---|---|---|
Patient no. 6 | 5.6 | 5 | −1.69 | 0 | 78.1 | 0 |
Patient no. 7 | 1.5 | 1 | −2.06 | 0 | 120.6 | 0 |
Patient no. 8 | 1.4 | 1 | −0.29 | 1.61 | 153.2 | 1.05 |
Patient no. 9 | 1.3 | 1 | 1.29 | 0 | 145.2 | 0 |
Patient no. 10 | 6.2 | 6 | −0.38 | 2.85 | 18.4 | 15.48 |
Patient no. 11 | 2.5 | 1 | −2.14 | 2.57 | 173.8 | 1.48 |
Patient no. 12 | 12.5 | 10 | −3.61 | 0 | 93.3 | 0 |
Patient no. 13 | 22.5 | 21 | −0.93 | 0 | 167.5 | 0 |
Patient no. 14 | 19 | 17 | −1.98 | 0 | 135.3 | 0 |
ND 1-15 years of age (n = 14), mean (SEM) | 8.06 (1.6) | 106.9 (5.6) | 8.4 (1.7) | |||
P | .004 | .504 | .033 |
. | Age at last follow-up, y . | PEG-ADA, y . | Percentile height, Z-score . | RANKL, pg/mL . | OPG, pg/mL . | RANKL/OPG, × 102 . |
---|---|---|---|---|---|---|
Patient no. 6 | 5.6 | 5 | −1.69 | 0 | 78.1 | 0 |
Patient no. 7 | 1.5 | 1 | −2.06 | 0 | 120.6 | 0 |
Patient no. 8 | 1.4 | 1 | −0.29 | 1.61 | 153.2 | 1.05 |
Patient no. 9 | 1.3 | 1 | 1.29 | 0 | 145.2 | 0 |
Patient no. 10 | 6.2 | 6 | −0.38 | 2.85 | 18.4 | 15.48 |
Patient no. 11 | 2.5 | 1 | −2.14 | 2.57 | 173.8 | 1.48 |
Patient no. 12 | 12.5 | 10 | −3.61 | 0 | 93.3 | 0 |
Patient no. 13 | 22.5 | 21 | −0.93 | 0 | 167.5 | 0 |
Patient no. 14 | 19 | 17 | −1.98 | 0 | 135.3 | 0 |
ND 1-15 years of age (n = 14), mean (SEM) | 8.06 (1.6) | 106.9 (5.6) | 8.4 (1.7) | |||
P | .004 | .504 | .033 |