Results of ophthalmic evaluations over the duration of treatment for patients with at least one follow-up visit (N=eyes)
| Duration of treatment | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| Start | 0–3 months | 3–9 months | 9–15 months | 15–21 months | 21–27 months | 27–33 months | 33–39 months | 39–45 months | |
| N=164 | N=30 | N=102 | N=64 | N=78 | N=58 | N=50 | N=34 | N=8 | |
| Visual acuity: Log scale | |||||||||
| N (missing) | 148 (16) | 26 (4) | 94 (8) | 64 (0) | 78 (0) | 58 (0) | 50 (0) | 34 (0) | 8 (0) |
| Mean (± SD) | 0.14 (±0.31) | 0.01 (±0.19) | 0.09 (±0.28) | 0.11 (±0.34) | 0.04 (±0.16) | 0.05 (±0.19) | 0.07 (±0.16) | 0.06 (±0.14) | 0.00 (±0.00) |
| Median (range) | 0.00 (−0.15–2.00) | 0.00 (−0.25–0.50) | 0.00 (−0.25–1.00) | 0.00 (−0.25–1.30) | 0.00 (−0.20–0.70) | 0.00 (−0.10–1.30) | 0.00 (0.00–1.00) | 0.00 (0.00–0.70) | 0.00 (0.00–0.00) |
| Cystinosis Corneal Crystal Score (CCCS) | |||||||||
| N (missing) | 120 (44) | 29 (1) | 88 (14) | 55 (9) | 70 (8) | 51 (7) | 48 (2) | 34 (0) | 8 (0) |
| Mean (SD) | 2.19 (±0.64) | 2.19 (±0.75) | 1.99 (±0.77) | 1.91 (±0.77) | 1.84 (±0.89) | 2.01 (±0.82) | 1.87 (±0.76) | 1.84 (±0.79) | 1.22 (±0.67) |
| Median | 2.00 | 2.25 | 2.00 | 2.00 | 2.00 | 2.00 | 2.00 | 2.00 | 1.13 |
| Range | 0.5–3.0 | 0.5–3.0 | 0.00–3.0 | 0.25–3.0 | 0.00–3.0 | 0.50–3.0 | 0.25–3.0 | 0.25–3.0 | 0.50–2.25 |
| Photophobia | |||||||||
| N (missing) | 160 (4) | 28 (2) | 96 (6) | 61 (3) | 74 (4) | 51 (7) | 48 (2) | 34 (0) | 8 (0) |
| Mean (SD) | 2.19 (±1.34) | 1.46 (±0.92) | 1.77 (±1.22) | 1.64 (±1.24) | 1.70 (±1.33) | 1.51 (±1.04) | 2.17 (±1.33) | 1.53 (±1.05) | 1.75 (±0.89) |
| Median | 2.00 | 1.00 | 2.00 | 2.00 | 2.00 | 1.00 | 2.00 | 1.00 | 1.50 |
| Range | 0–5 | 0–4 | −0–5 | 0–4 | 0–5 | 0–5 | 0–5 | 0–3 | 1–3 |
In the ATU cohort, the average visual acuity score decreased over time and tended to 0.0, which corresponds to normal sharpness of vision. Mean CCCS scores tended to decrease over time, stabilising after around 27 months of treatment, between 1.87 and 1.22 with a median of 2.0. Photophobia decreased after 0–3 months and stabilised around 1.7 and 1.5 over time.
ATU, Temporary Authorisation for Use.