Patient characteristics, 2014 ivacaftor and comparator cohorts, USA and UK
| Characteristic | US CFFPR | UK CFR | ||||
| Ivacaftor (n=1256), n (%) | Comparator (n=6200), n (%) | χ2 P values | Ivacaftor (n=411), n (%) | Comparator (n=2069), n (%) | χ2 P values | |
| Age in 2014, years | ||||||
| 0 to <6 | 42 (3.3) | 205 (3.3) | 0.8897 | 8 (1.9) | 40 (1.9) | 0.6486 |
| 6 to <12 | 215 (17.1) | 1109 (17.9) | 72 (17.5) | 325 (15.7) | ||
| 12 to <18 | 268 (21.3) | 1277 (20.6) | 75 (18.2) | 426 (20.6) | ||
| ≥ 18 | 731 (58.2) | 3609 (58.2) | 256 (62.3) | 1278 (61.8) | ||
| Sex | ||||||
| Male | 626 (49.8) | 3108 (50.1) | 0.8522 | 216 (52.6) | 1083 (52.3) | 0.9808 |
| Female | 630 (50.2) | 3092 (49.9) | 195 (47.4) | 986 (47.7) | ||
| Genotype, overall | ||||||
| Class I–III | 1129 (89.9)* | 5464 (88.1) | 0.0755 | 384 (93.4)† | 1944 (94.0) | 0.7681 |
| Class IV–VI and unknown | 127 (10.1)‡ | 736 (11.8) | 27 (6.6)§ | 125 (6.0) | ||
| ppFEV 1, 2014¶ | ||||||
| <40 | 106 (8.4) | 720 (11.6) | 0.0002 | 38 (9.2) | 266 (12.9) | <0.001 |
| 40 to <70 | 303 (24.1) | 1640 (26.5) | 89 (21.7) | 646 (31.2) | ||
| ≥70 | 807 (64.3) | 3602 (58.1) | 274 (66.7) | 1048 (50.7) | ||
| Missing | 40 (3.2) | 238 (3.8) | 10 (2.4) | 109 (5.3) | ||
| ppFEV 1, baseline** | ||||||
| <40 | 90 (7.2) | 435 (7.0) | 0.5430 | 46 (12.0) | 204 (10.2) | 0.2356 |
| 40 to <70 | 290 (23.1) | 1330 (21.5) | 100 (26.2) | 603 (30.3) | ||
| ≥70 | 639 (50.9) | 3191 (51.5) | 193 (50.5) | 981 (49.2) | ||
| Missing | 237 (18.9) | 1244 (20.1) | 43 (11.3) | 205 (10.3) | ||
| PEx, baseline** | 444 (38.5) | 2187 (37.4) | 0.5002 | 207 (54.2) | 1061 (53.2) | 0.7755 |
| Hospitalisations, baseline** | 443 (38.4) | 2294 (39.3) | 0.5818 | 173 (45.3) | 862 (43.3) | 0.9151 |
*All but one patient had approved gating mutations on at least one allele.
†All patients had approved gating mutations on at least one allele.
‡Includes 36 patients with approved gating mutations on one allele and a class IV–VI mutation on the second and 15 patients with mutations unknown.
§Includes 16 patients with approved gating mutation on at least one allele and 11 patients with mutations unknown.
¶Average of best available quarterly values in 2014 from the US CFFPR and 2014 annual assessment value from the UK CFR.
**Baseline is defined as 2011 for the US CFFPR and 2012 for the UK CFR.
CFFPR, Cystic Fibrosis Foundation Patient Registry; CFR, Cystic Fibrosis Registry; PEx, pulmonary exacerbation; ppFEV1, per cent predicted FEV1.