Table 1.
Characteristics and Outcomes of the G551D Observational Cohort by 1 Month FEV1 and BMI Responder Status
| FEV1 1-Month Response to Ivacaftor |
BMI 1-Month Response to Ivacaftor |
|||
|---|---|---|---|---|
| Demographics and Baseline Characteristics | Non-responders (≤0 ↑ FEV1 PP) (n = 32) | Responders (>0 ↑ FEV1 PP) (n = 112) | Nonresponders (≤0 ↑ kg/m2) (n = 40) | Responders (>0 ↑ kg/m2) (n = 104) |
| Female, n (%) | 18 (56%) | 49 (44%) | 22 (55%) | 45 (43%) |
| Age, mean (SD) | 16.0 (9.9) | 22.6 (11.7) | 23.7 (13.4) | 20.1 (10.8) |
| Other CFTR allele, n (%) | ||||
| Not active | 26 (81%) | 100 (89%) | 35 (88%) | 91 (88%) |
| Partially active | 3 (9%) | 4 (4%) | 3 (8%) | 4 (4%) |
| Unknown | 3 (9%) | 8 (7%) | 2 (5%) | 9 (9%) |
| Baseline FEV1 PP, mean (SD) | 91.7 (25.5) | 78.1 (24.3) | 83.0 (27.1) | 80.4 (24.4) |
| Baseline sweat chloride mEq/L, mean (SD) | 101.5 (12.5) | 103.1 (14.5) | 100.7 (21.6) | 103.4 (10.0) |
| Baseline BMI, kg/m2, mean (SD) | 19.2 (3.9) | 21.6 (4.3) | 22.1 (5.2) | 20.7 (3.8) |
| CF-related diabetes, n (%) | 7 (22%) | 35 (31%) | 14 (35%) | 28 (27%) |
| Pa on respiratory culture, n (%) | 19 (59%) | 79 (71%) | 31 (78%) | 67 (64%) |
Definition of abbreviations: BMI = body mass index; CF = cystic fibrosis; CFTR = cystic fibrosis transmembrane conductace regulator; Pa = Pseudomonas aeruginosa; PP = percentage predicted via Global Lung Function Initiative equations.