Table 1

Patient demographics and characteristics (N=130)

Characteristics (N=130)Treatments (N=116)*Outcomes (N=130)
Sex
 Female
 Male		66 (50.8%)
64 (49.2%)		Previous treatment
 Cystadrops
 Cystaran
 Cysteamine 0.1%
 Unspecified cysteamine concentration
 Unnamed cysteamine treatment		3 (2.6%)
1 (0.9%)
28 (24.1%)
82 (70.7%)
2 (1.7%)		Visual acuity: log scale
 N (missing)
 Mean (SD)
 Median (range)		230 (30)
0.13 (0.28)
0.00 (−0.52* to −2.00)
Age at ATU prescription request (years)
 NOT
 Average (±SD)
 Median (range)		130
19.5 (±13.8)
17.0 (2.0–78.0)		Previous treatment period (years)
 N (missing data)
 Mean (SD)
 Median (range)		112 (4)
12.8 (9.1)
10.7 (0.0–36.7)		Cystinosis Corneal Crystal Score
 N (missing)
 Mean (SD)
 Median (range)		180 (80)
2.13 (0.72)
2.00 (0.00–3.00)
Age at ATU prescription request (years)
 2–6 
 6–12 
 12–18 
 ≥18 		24 (18.5%)
17 (13.1%)
25 (19.2%)
64 (49.2%)		Frequency of instillations per day per eye
 Missing data
 1
 2
 3
 4
 5
 6
 8
 10
 12		3
2 (1.8%)
7 (6.2%)
10 (8.8%)
42 (37.2%)
11 (9.7%)
30 (26.5%)
7 (6.2%)
2 (1.8%)
2 (1.8%)		Photophobia
 N (missing)
 Mean (SD)
 Median (range)		250 (10)
2.16 (1.34)
2.00 (0–5)
Age at diagnosis (years)
 N (missing)
 Mean (SD)
 Median (range)		125 (5)
3.3 (8.1)
1.0 (0.0–76.0)		Number of instillations per day per eye
 N (missing)
 Mean (SD)
 Median (range)		113 (3)
4.9 (1.93)
4.0 (1–12)
Age stratification at diagnosis (years)
 Missing data
 0–1 
 1–2 
 2–6 
 6–12 
 12–18
 ≥18 		5
38 (30.4%)
40 (32.0%)
28 (22.4%)
10 (8.0%)
5 (4.0%)
4 (3.2%)
Disease duration (years)
 Missing data
 Mean (SD)
 Median (range)		125 (5)
15.6 (12.3)
14.0 (0.0–54.0)

  • *Fourteen patients did not receive ophthalmic treatment with cysteamine before initiating treatment Cystadrops.

  • ATU, Temporary Authorisation for Use.