Table 3.
List of ages reaching different stages, period before transition between different stages, ADA titers, genotypes, and uGAG levels by treatment.
| Untreated until stage 4 (Late-treated) |
ERT initiated at stage 2 (Early ERT) |
HSCT at stage 2 (Early HSCT) |
||||
|---|---|---|---|---|---|---|
| n | Median age (IQR) | n | Median age (IQR) | n | Median age (IQR) | |
| Treatment age | 8 | 7.0 (7.0–10.5) | 6 | 2.5 (2.0–3.0) | 3 | 4.0 (2.0–5.0) |
| Age at stage 2 | 8 | 3.0 (1.5–3.0) | 6 | 2.0 (2.0–3.0) | 3 | 2.0 (1.0–3.0) |
| Age at stage 4 | 8 | 5.0 (5.0–6.5) | 6 | 6.0 (5.0–6.0) | 3 | 7.0 (6.0–15.0) |
| Age at stage 5 | 6 | 12.5 (12.0–18.0) | 1 | 11.0 | 0 | – |
| Age at death | 3 | 17.0 (14.0–21.0) | 0 | – | 0 | – |
| n | Mean year (SD) | n | Mean year (SD) | n | Mean year (SD) | |
| Stage 2–4 interval | 8 | 3.1 (1.5) | 6 | 3.5 (0.7) | 3 | 7.3 (3.3) |
| Missense mutation | 2 | 3.0 (1.0) | 3 | 4.0 (0.8) | 2 | 8.5 (3.5) |
| Other type mutation | 6 | 3.2 (1.7) | 3 | 3.0 (0.0) | 1 | 5.0 |
| Stage 4–5 interval | 6 | 9.0 (3.8) | 1 | 6.0 | 0 | – |
| Stage 5 to death | 3 | 4.0 (0.8) | 0 | – | 0 | – |
| Pt | Pt | |||||
| ID | ADA titer | ID | ADA titer | |||
| Missense mutation | 13 | 2 | 17 | 0 | ||
| 10 | 50 | 11 | 2 | |||
| 18 | 328,000 | |||||
| Other type mutations | 27 | 20 | 16 | 163,840 | ||
| 7 | 160 | 15 | 655,360 | |||
| 6 | 200 | 14 | 20,971,520 | |||
| 5 | 40,960 | |||||
| 9 | 163,840 | |||||
| Mean (SD) | Mean (SD) | |||||
| n | (mg/g creatinine) | n | (mg/g creatinine) | n | ||
| uGAG baseline level | 5 | 208 (66.8) | 5 | 334 (81.2) | 0 | – |
| uGAG lowest level | 5 | 27.1 (11.3) | 5 | 67.8 (24.0) | 0 | – |
ADA = anti-drug antibody; ERT = enzyme replacement therapy; HSCT = hematopoietic stem cell transplantation, uGAG = urinary glycosaminoglycan.