Table 1.
Baseline Demographic and Clinical Characteristics by Mutation.
| Characteristic |
R117H (n=156) |
F508del (n=6251) |
P-Value* |
|---|---|---|---|
| Age, years, mean (SD) | 29.2 (17.2) | 17.0 (9.1) | <0.001 |
| Female, % | 51.9 | 47.3 | 0.26 |
| Year of CF diagnosis | |||
| median | 1998 | 1992 | <0.001 |
| min, max | 1958, 2006 | 1949, 2006 | |
| Sweat chloride | [n=126] | [n=5,729] | |
| mmol/L, mean (SD) | 77.8 (23.9) | 102.6 (16.3) | <0.001 |
| Height z-score, mean (SD) | 0.11 (1.03) | −0.51 (1.01) | <0.001 |
| Weight z-score, mean (SD) | 0.46 (1.14) | −0.41 (1.02) | <0.001 |
| BMI z-score, | [n=6,250] | ||
| mean (SD) | 0.41 (1.07) | −0.20 (0.96) | <0.001 |
| ppFEV1 baseline first visit, mean (SD) | 79.0 (22.2) | 77.3 (23.0) | 0.35 |
| ppFEV1 best baseline year value, mean (SD) | 83.9 (22.4) | 84.8 (22.3) | 0.61 |
| CF-related diabetes, % | 7.1 | 15.5 | 0.004 |
| Liver disease, % | 0.6 | 4.1 | 0.029 |
| Microbiology | [n=152] | [n=6,130] | |
| Staphylococcus aureus, % | 36.2 | 54.1 | <0.001 |
| Pseudomonas aeruginosa, % | 29.6 | 49.2 | <0.001 |
| Candida species, % | 7.2 | 3.3 | 0.007 |
| Stenotrophomonas maltophilia, % | 5.3 | 7.7 | 0.27 |
| Aspergillus species, % | 3.9 | 8.9 | 0.034 |
| Haemophilus influenzae, % | 3.3 | 6.1 | 0.15 |
| ≥1 IV treatment for PEx, % | 28.2 | 45.9 | <0.001 |
| ≥1 hospitalization, % | 21.2 | 40.8 | <0.001 |
| ≥1 home IV antibiotic treatment, % | 18.6 | 26.3 | 0.031 |
| IV treatment for PEx events/year, mean (SD) | 0.44 (0.84) | 0.87 (1.31) | <0.001 |
| Hospitalization events/year, mean (SD) | 0.32 (0.74) | 0.75 (1.22) | <0.001 |
| Home IV antibiotic events/year, mean (SD) | 0.25 (0.60) | 0.41 (0.84) | 0.020 |
| IV treatment in patients with ≥1 PEx, mean (SD) | 1.57 (0.87) | 1.90 (1.33) | 0.10 |
*
P-Value obtained from t-test or chi-square test.
CF, cystic fibrosis; BMI, body mass index; max, maximum; min, minimum; ppFEV1, percentage of predicted forced expiratory volume in 1 second; IV, intravenous; PEx, pulmonary exacerbation; SD, standard deviation.