Table 1. Characteristics at baseline for CFA subgroups and for randomization groups.
| By CFA Subgroup | By Randomization Group | ||||
|---|---|---|---|---|---|
| All | High CFA (≥88%) | Low CFA (<88%) | Placebo | Encala™ | |
| Characteristic | n = 66 | n = 33 | n = 33 | n = 36 | n = 30 |
| Sex, % males | 61 | 55 | 67 | 67 | 53 |
| F508del homozygous, % | 55 | 42 | 67* | 50 | 60 |
| Age, yr | 10.5 ± 3.0 | 9.6 ± 2.7 | 11.5 ± 2.9* | 10.4 ± 3.1 | 10.7 ± 2.9 |
| FEV1% a | 99 ± 22 | 101 ± 24 | 98 ± 20 | 104 ± 22 | 94 ± 20 |
| Growth and Body Composition | |||||
| Height for age Z score | -0.52 ± 0.93 | -0.54 ± 1.13 | -0.49 ± 0.69 | -0.57 ± 0.90 | -0.46 ± 0.97 |
| Weight for age Z score | -0.45 ± 0.72 | -0.34 ± 0.88 | -0.55 ± 0.52 | -0.35 ± 0.77 | -0.57 ± 0.66 |
| BMI for age Z score | -0.17 ± 0.69 | 0.02 ± 0.73 | -0.37 ± 0.60* | 0.00 ± 0.68 | -0.39 ± 0.66* |
| Whole Body DXA | |||||
| FFM, kg | 26.3 ± 9.7 | 23.9 ± 8.2 | 28.7 ± 10.6* | 26.7 ± 10.9 | 25.8 ± 8.2 |
| FM, kg | 7.1 ± 2.8 | 6.9 ± 2.8 | 7.2 ± 2.8 | 6.9 ± 2.5 | 7.3 ± 3.1 |
| Fat, % | 21.6 ± 5.8 | 22.7 ± 5.2 | 20.6 ± 6.3 | 21.4 ± 6.3 | 21.9 ± 5.3 |
| Coefficient of Fat Absorption | |||||
| Stool fat, g/day | 16.4 ± 13.1 | 8.3 ± 4.8 | 24.4 ± 13.8*** | 15.6 ± 14.6 | 17.3 ± 11.1 |
| Dietary fat intake, g/day | 103.3 ± 35.4 | 108.5 ± 41.4 | 98.0 ± 28.0 | 105.9 ± 38.7 | 100.2 ± 31.4 |
| CFA, % | 83.9 ± 11.2 | 92.4 ± 2.6 | 75.3 ± 9.9*** | 85.1 ± 12.1 | 82.4 ± 10.2 |
| CFA < Median (88%), % | 50 | 0 | 100 | 39 | 63* |
| Dietary energy intake, Kcal/day | 2556 ± 692 | 2611 ± 792 | 2501 ± 582 | 2588 ± 740 | 2518 ± 639 |
| EER % | 124 ± 29 | 132 ± 37 | 116 ± 17* | 124 ± 31 | 123 ± 28 |
| Plasma Fatty Acids, nmol/ L b | |||||
| Linoleic acid | 2292 ± 514 | 2348 ± 428 | 2239 ± 584 | 2308 ± 562 | 2271 ± 453 |
| α-Linolenic acid | 51.4 ± 24.4 | 52.0 ± 21.0 | 50.9 ± 27.6 | 52.6 ± 28.7 | 49.9 ± 18.0 |
| γ-Linolenic acid | 65.4 ± 28.7 | 61.4 ± 28.7 | 69.2 ± 28.6 | 63.7 ± 29.4 | 67.6 ± 28.1 |
| Arachidonic acid | 438 ± 153 | 445 ± 167 | 432 ± 141 | 441 ± 160 | 436 ± 145 |
| Eicosapentaenoic acid | 37.1 ± 23.4 | 37.3 ± 23.1 | 36.8 ± 24.1 | 36.7 ± 21.3 | 37.5 ±26.4 |
| Docosahexaenoic acid | 61.1 ± 23.6 | 62.3 ± 23.6 | 60.0 ± 28.8 | 62.1 ± 24.8 | 59.9 ± 28.4 |
| Docosapentaenoic acid | 36.2 ± 16.0 | 37.5 ±17.8 | 35.0 ± 14.3 | 37.5 ± 17.5 | 34.5 ± 14.1 |
| Plasma Fatty Acid Group, mmol/L b | |||||
| Saturated | 3.2 ± 0.7 | 3.2 ± 0.7 | 3.2 ± 0.7 | 3.2 ± 0.7 | 3.1 ± 0.7 |
| Monounsaturated | 2.4 ± 0.5 | 2.3 ± 0.5 | 2.4 ± 0.7 | 2.4 ± 0.7 | 2.3 ± 0.5 |
| Polyunsaturated | 3.2 ± 0.7 | 3.1 ± 0.6 | 3.2 ± 0.8 | 3.2 ± 0.8 | 3.1 ± 0.6 |
| Total Fatty Acids | 8.8 ± 1.8 | 8.7 ± 1.7 | 8.8 ± 2.0 | 8.8 ± 2.0 | 8.7 ± 1.7 |
CFA: coefficient of fat absorption; DXA: dual x-ray absorptiometry; FFM: fat free mass; FM: fat mass; Fat: fat mass as percent total body mass; FEV1%: Forced expiratory volume at 1 second percent predicted value.
Data are presented as mean ± SD and frequency (percentage) for categorical data.
Bolded values show statistical significance.
*Significantly different between randomization groups by unpaired t test for continuous variables and χ-squared test or Fisher’s exact test for categorical variables, p<0.05,
***p<0.001
a All, n = 63; High CFA, n = 32; Low CFA, n = 31: Placebo, n = 36; Encala™, n = 27
b All, n = 64; High CFA, n = 31; Low CFA, n = 33: Placebo, n = 36; Encala™, n = 28