Table 1.
Demographic Characteristics of the Study Population at the Baseline Visit
| Characteristic | GOAL (n = 5) | SickKids (n = 4) | GOAL + SickKids (n = 9) |
|---|---|---|---|
| Age, yr | 4.2 (4.0 to 5.1) | 4.8 (3.0 to 5.8) | 4.2 (3.9 to 5.2) |
| Female, n (%) | 3 (60%) | 2 (50%) | 5 (56%) |
| Genetics, first allele, n (%) | |||
| G551D | 5 (100%) | 4 (100%) | 9 (100%) |
| Genetics, second allele, n (%) | |||
| F508del | 3 (75%) | 4 (80%) | 7 (78%) |
| 1717-1G→A | 1 (11%) | 1 (20%) | 0 (0%) |
| R1066C | 1 (11%) | 0 (0%) | 1 (25%) |
| Height z-score | 0.52 (0.47 to 1.06) | −0.49 (−1.08 to 0.14) | 0.34 (−0.92 to 0.52) |
| Weight z-score | 0.55 (−0.13 to 0.88) | −0.19 (−0.80 to −0.02) | −0.09 (−0.30 to 0.55) |
| BMI z-score | −0.12 (−0.73 to 0.62) | −0.28 (−0.55 to 0.39) | −0.12 (−0.55 to 0.62) |
| LCI | 10.6 (8.9 to 11.2) | 8.9 (7.6 to 11.6) | 9.8 (8.2 to 11.2) |
| Sweat chloride | 105 (103 to 107) | 98 (94 to 103) | 103 (96 to 105) |
| Pancreatic insufficiency, n (%) | 5 (100%) | 4 (100%) | 9 (100%) |
| Infections, n (%) | |||
| Staphylococcus aureus | 2 (50%)* | 1 (25%) | 3 (38%)* |
| Haemophilus influenzae | 2 (50%)* | 0 (0%) | 2 (25%)* |
| Pseudomonas aeruginosa | 0 (0%)* | 0 (0%) | 0 (0%)* |
| Concomitant medications, n (%) | |||
| Dornase alfa | 2 (40%) | 1 (25%) | 3 (33%) |
| Hypertonic saline | 2 (40%) | 0 (0%) | 2 (22%) |
| Oral antibiotics | 0 (0%) | 1 (25%) | 1 (11%) |
Definition of abbreviations: BMI = body mass index; GOAL = G551D Observational Study; LCI = lung clearance index; SickKids = Hospital for Sick Children in Toronto.
All continuous variables are presented as medians with interquartile ranges.
*
One GOAL subject did not have microbiology assessments performed at baseline.