Characteristics of individuals in Canada, France, Australia and New Zealand at the most recent measurement within the study window (2015–2019; n=14 842)*
| Australia | Canada | France | New Zealand | |
| Total number of individuals | 3537 | 4434 | 6411 | 460 |
| Sex | ||||
| Women | 1676 (47.4) | 2025 (45.7) | 3048 (47.5) | 209 (45.4) |
| Men | 1861 (52.6) | 2409 (54.3) | 3363 (52.5) | 251 (54.6) |
| Age at diagnosis | ||||
| <2 years old | 3010 (85.1) | 2809 (63.4) | 4477 (69.8) | 349 (75.9) |
| ≥2 years old | 527 (14.9) | 1625 (36.6) | 1934 (30.2) | 111 (24.1) |
| %Imputed | 533 (15.1%) | 79 (1.8%) | 205 (3.2%) | 14 (3.0%) |
| Newborn screening | 1805 (51.0) | 611 (13.8) | 2786 (43.5) | 218 (47.4) |
| Genotype | ||||
| Phe508del homozygote | 1632 (46.1) | 2089 (47.1) | 2615 (40.8) | 228 (49.6) |
| Phe508del heterozygote | 1418 (40.1) | 1775 (40.0) | 2755 (43) | 166 (36.1) |
| Other | 258 (7.3) | 503 (11.3) | 958 (14.9) | 48 (10.4) |
| Missing | 229 (6.5) | 67 (1.5) | 83 (1.3) | 18 (3.9) |
| Mutation class | ||||
| Class I–III | 2980 (84.3%) | 3754 (84.7%) | 5390 (84.1%) | 392 (85.2%) |
| Class IV–V | 291 (8.2%) | 534 (12%) | 741 (10%) | 35 (7.6%) |
| Unclassifiable/missing | 266 (7.5%) | 146 (3.3%) | 321 (5%) | 33 (7.2%) |
| Pancreatic status (ever/never) | ||||
| Insufficient | 2770 (78.3) | 3690 (83.2) | 5749 (89.7%) | 368 (80.0) |
| Sufficient | 602 (17.0) | 744 (16.8) | 662 (10.3%) | 87 (18.9) |
| Missing | 165 (4.7) | 0 | 0 | 5 (1.1) |
| Microbiology (ever/never) | ||||
| P. aeruginosa | N/A | 1793 (40.4) | 2652 (41.4) | 166 (36.1) |
| B. cepacia complex | N/A | 352 (7.9) | 271 (4.2) | 45 (9.8) |
| Intravenous antibiotic courses/year† | ||||
| 0 | 1760 (53.3%) | 2659 (65.4%) | 3246 (56.6%) | 237 (52.4%) |
| 1–2 | 967 (29.3%) | 952 (23.4%) | 1204 (21%) | 123 (27.2%) |
| ≥ 3 | 572 (17.3%) | 450 (11.1%) | 1287 (22.4%) | 92 (20.4%) |
| ppFEV1‡ median, IQR | 82.9 (57.7–96.5) | 75.9 (51.7–93.2) | 77.5 (53.5–94.2) | 83.4 (59.7–98) |
| <40 | 325 (9.9%) | 546 (13.4%) | 782 (13.6%) | 49 (10.8%) |
| 40–69 | 698 (21.2%) | 1021 (25.1%) | 1413 (24.6%) | 94 (20.8%) |
| ≥70 | 1965 (59.6%) | 2054 (50.6%) | 3215 (56%) | 275 (60.8%) |
| Missing | 311 (9.4%) | 440 (10.8%) | 327 (5.7%) | 34 (7.5%) |
| BMI categories§ | ||||
| Underweight | 274 (8.3%) | 387 (9.5%) | 1062 (18.5%) | 39 (8.6%) |
| Normal | 2074 (62.9%) | 2502 (61.6%) | 3875 (67.5%) | 303 (67%) |
| Overweight | 656 (19.9%) | 814 (20%) | 577 (10.1%) | 81 (17.9%) |
| Missing | 295 (8.9%) | 358 (8.8%) | 223 (3.9%) | 29 (6.4%) |
*Data are presented as n (%), unless indicated otherwise. Clinical variables are summarised based on the latest pre-transplant observation within the study period. There were 1293 patients who had no pre-transplant FEV1 % predicted, BMI or courses of intravenous antibiotic clinical measurements recorded because they were transplanted before 2015 and individuals were excluded from the table summary (238 (6.7%) in AUS, 373 (8.4%) in CAN, 674 (10.5%) in FRA and 8 (1.7%) in NZ.
†Intravenous courses includes antibiotic therapy given in hospital and at home.
‡Percent predicted forced expiratory volume in 1 s (ppFEV1) was calculated using the Global Lung Function Initiative reference equation. The best ppFEV1 was chosen for each the year.
§Body mass index (BMI for adults) or BMI percentile (for children) closest to the chosen FEV1 was selected. Individuals were categorised as underweight (BMI <19 kg/m2 or BMI %ile ≤12%), adequate weight (BMI between ≥19 kg/m2 and ≤24.9 kg/m2 or BMI %ile between >12% and <85%) or overweight (BMI >24.9 kg/m2 or BMI %ile ≥85%).
B. cepacia complex, Burkholderia cepacia complex; AUS, Australia; BMI, body mass index; CAN, Canada; FEV1, forced expiratory volume in 1 s; FRA, France; N/A, not available; NZ, New Zealand; P. aeruginosa, Pseudomonas aeruginosa.