Table 1.
Baseline Characteristics and Treatment Parameters
| Early Robust Responder per Protocol Population | Non–early Robust Responder Intent to Treat Population | |||||
|---|---|---|---|---|---|---|
| Duration assignment | 10 d | 14 d | Total | 14 d | 21 d | Total |
| N | 107 | 107 | 214 | 353 | 352 | 705 |
| Sex, male (%) | 61 (57) | 54 (50.5) | 115 (53.7) | 161 (45.6) | 181 (51.4) | 342 (48.5) |
| Mean age, yr (SD) | 26.6 (6.3) | 27.1 (8.9) | 26.9 (7.7) | 31.8 (10.5) | 31.5 (9.6) | 31.7 (10.1) |
| Genotype | ||||||
| F508del homozygous, n (%) | 46 (43.0) | 55 (51.4) | 101 (47.2) | 171 (48.4) | 174 (49.4) | 345 (48.9) |
| F508del heterozygous, n (%) | 45 (42.1) | 40 (37.4) | 85 (39.7) | 127 (36.0) | 135 (38.4) | 262 (37.2) |
| Other/UK, n (%) | 16 (14.9) | 12 (11.2) | 28 (13.1) | 55 (15.6) | 43 (12.2) | 98 (13.9) |
| Body mass index, kg/m2, mean (SD) | 21.7 (4.4) | 21.6 (4.0) | 21.6 (4.2) | 22.3 (4.1) | 22.4 (3.9) | 22.3 (4.0) |
| ppFEV1 | ||||||
| Visit 1, mean (SD) | 49.5 (18.6) | 49.8 (19.6) | 49.7 (19.1) | 49.5 (21.2) | 50.0 (20.3) | 49.7 (20.7) |
| Visit 1 < 50%, n (%) | 60 (56.1) | 61 (57.0) | 121 (56.5) | 192 (54.4) | 194 (55.1) | 386 (54.8) |
| Average 6 mo prior in CFFPR | ||||||
| n | 98 | 91 | 189 | 334 | 328 | 662 |
| Mean (SD) | 60.2 (17.9) | 60.3 (19.3) | 60.3 (18.6) | 51.8 (20.0) | 53.2 (20.1) | 52.5 (20.0) |
| Change from average 6 mo prior to visit 1, mean (SD) | −9.3 (9.3) | −9.2(8.1) | −9.2 (8.7) | −2.2 (10.3) | −2.9 (6.4) | −2.5 (8.6) |
| History of PEx in last year | ||||||
| 0–1, n (%) | 43 (40.2) | 45 (42.1) | 88 (41.1) | 145 (41.1) | 144 (40.9) | 289 (41.0) |
| ⩾2, n (%) | 64 (59.8) | 62 (57.9) | 126 (58.9) | 208 (58.9) | 208 (59.1) | 416 (59.0) |
| Chronic oral antibiotics (%) | 59 (55.1) | 55 (51.4) | 114 (53.3) | 197 (55.8) | 206 (58.5) | 403 (57.2) |
| CFTR modulator* | ||||||
| None, n (%) | 73 (68.2) | 72 (67.3) | 145 (67.8) | 214 (60.6) | 206 (58.5) | 420 (59.6) |
| Highly effective, n (%) | 7 (6.5) | 3 (2.8) | 10 (4.7) | 25 (7.1) | 28 (8.0) | 53 (7.5) |
| CF-related diabetes | ||||||
| Insulin dependent, n (%) | 26 (24.3) | 24 (22.4) | 50 (23.4) | 105 (29.7) | 116 (33.0) | 221 (31.3) |
| Non-insulin dependent, n (%) | 11 (10.3) | 12 (11.2) | 23 (10.7) | 28 (7.9) | 34 (9.7) | 62 (8.8) |
| Allergic bronchopulmonary aspergillosis, n (%) | 16 (15.0) | 9 (8.4) | 25 (11.7) | 21 (5.9) | 36 (10.2) | 57 (8.1) |
| NTM treated in last 2 yr, n (%) | 6 (5.6) | 7 (6.5) | 13 (6.1) | 21 (5.9) | 15 (4.3) | 36 (5.1) |
| Pneumothorax in last 2 yr, n (%) | 3 (2.8) | 2 (1.9) | 5 (2.3) | 4 (1.1) | 8 (2.3) | 12 (1.7) |
| Treatment location prior to randomization | ||||||
| Any hospital, n (%) | 92 (86) | 90 (84.1) | 182 (85) | 265 (75.1) | 266 (75.6) | 531 (75.3) |
| All at home, n (%) | 15 (14) | 17 (15.9) | 32 (15) | 88 (24.9) | 86 (24.4) | 174 (24.7) |
| Antibiotics initiated† | ||||||
| Intravenous, n (%) | 107 (100) | 107 (100) | 214 (100) | 353 (100) | 352 (100) | 705 (100) |
| Oral, n (%) | 29 (27.1) | 24 (22.4) | 53 (24.8) | 99 (28.0) | 82 (23.3) | 181 (25.7) |
| Inhaled, n (%) | 2 (1.9) | 5 (4.7) | 7 (3.3) | 9 (2.5) | 11 (3.1) | 20 (2.8) |
| Steroids initiated† | 13 (12.1) | 10 (9.3) | 23 (10.7) | 33 (9.3) | 41 (11.6) | 74 (10.5) |
Definition of abbreviations: CF = cystic fibrosis; CFFPR = Cystic Fibrosis Foundation patient registry; CFTR = cystic fibrosis transmembrane conductance regulator; NTM = nontuberculosis Mycobacterium; PEx = pulmonary exacerbations; ppFEV1 = percent of predicted FEV1.
*
Highly effective include ivacaftor, elexacaftor/tezacaftor/ivacaftor; other CFTR modulators include tezacaftor/ivacaftor and lumacaftor/ivacaftor.
†
Initiated on or after day of intravenous antibiotics start and before randomization.