ADA-SCID patients treated with hematopoietic stem cell gene therapy display a significant increase in RANKL
| Patient no. . | Age at therapy, y . | Age at last follow-up, y . | Percentile height, Z-score . | RANKL, pg/mL . | OPG, pg/mL . | RANKL/OPG, × 102 . | Percentage of bone age/ actual age, y (post GT) . | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Pre . | Post (at last follow-up) . | Pre . | Post . | Pre . | Post . | Pre . | Post . | ||||
| 1 | 1 | 7.6 | −1.66 | −1.12 | 1.4 | 3.65 | 181.9 | 113.1 | 0.78 | 3.23 (2) | 96% (6) |
| 2 | 1.9 | 8 | −3.91 | −1.48 | 1.0 | 16.1 | 129.6 | 62.3 | 0.79 | 25.95 | 109% (6) |
| 4 | 1.6 | 3.3 | −1.9 | −2.11 | 0 | 0.82 | 195.7 | 172.8 | 0 | 0.47 (1) | 96% (1) |
| 5 | 0.5 | 1.5 | −2.21 | −2.07 | 3.0 | 18.3 | 257.3 | 191.5 | 1.19 | 9.59 (1) | ND |
| 6 | 5.6 | 10.7 | −1.69 | −1.22 | 0 | 2.3 | 78.1 | 157.5 | 0 | 1.46 (1) | 81% (4) |
| 7 | 1.5 | 4.6 | −2.06 | −0.69 | 0 | 3.54 | 120.6 | 412.9 | 0 | 0.85 (2) | 65% (3) |
| 8 | 1.4 | 3.6 | −0.29 | 1.46 | 1.6 | 9.1 | 153.2 | 198.6 | 1.05 | 4.58 (1) | 74% (2) |
| 9 | 1.3 | 2.9 | 1.29 | 0.20 | 0 | 6.84 | 145.2 | 148.1 | 0 | 4.61 (1) | 64% (1) |
| 15 | 1.6 | 6.3 | −3.11 | −0.99 | 2.2 | 8.94 | 121.2 | 64.2 | 1.87 | 13.93 | ND |
| P | .005 | .698, NS | .031 | ||||||||
| Patient no. . | Age at therapy, y . | Age at last follow-up, y . | Percentile height, Z-score . | RANKL, pg/mL . | OPG, pg/mL . | RANKL/OPG, × 102 . | Percentage of bone age/ actual age, y (post GT) . | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Pre . | Post (at last follow-up) . | Pre . | Post . | Pre . | Post . | Pre . | Post . | ||||
| 1 | 1 | 7.6 | −1.66 | −1.12 | 1.4 | 3.65 | 181.9 | 113.1 | 0.78 | 3.23 (2) | 96% (6) |
| 2 | 1.9 | 8 | −3.91 | −1.48 | 1.0 | 16.1 | 129.6 | 62.3 | 0.79 | 25.95 | 109% (6) |
| 4 | 1.6 | 3.3 | −1.9 | −2.11 | 0 | 0.82 | 195.7 | 172.8 | 0 | 0.47 (1) | 96% (1) |
| 5 | 0.5 | 1.5 | −2.21 | −2.07 | 3.0 | 18.3 | 257.3 | 191.5 | 1.19 | 9.59 (1) | ND |
| 6 | 5.6 | 10.7 | −1.69 | −1.22 | 0 | 2.3 | 78.1 | 157.5 | 0 | 1.46 (1) | 81% (4) |
| 7 | 1.5 | 4.6 | −2.06 | −0.69 | 0 | 3.54 | 120.6 | 412.9 | 0 | 0.85 (2) | 65% (3) |
| 8 | 1.4 | 3.6 | −0.29 | 1.46 | 1.6 | 9.1 | 153.2 | 198.6 | 1.05 | 4.58 (1) | 74% (2) |
| 9 | 1.3 | 2.9 | 1.29 | 0.20 | 0 | 6.84 | 145.2 | 148.1 | 0 | 4.61 (1) | 64% (1) |
| 15 | 1.6 | 6.3 | −3.11 | −0.99 | 2.2 | 8.94 | 121.2 | 64.2 | 1.87 | 13.93 | ND |
| P | .005 | .698, NS | .031 | ||||||||
NS indicates not significant; and ND, not done.
Numbers in parentheses indicate year after GT.