Table 1.
Demographics and characteristics in CFLD patients
| Description | All n =561 | Malesa n =353 (63%) | Females n =208 (37%) |
|---|---|---|---|
| Age (years) at enrollment, mean (±SD) | 18.3 (±7.2) | 18.1 (±6.8) | 18.7 (±7.8) |
| Median (range) | 17.4 (2.1-52.5) | 17.2 (2.8-44.8) | 17.9 (2.1-52.5) |
| Race, n (%) | |||
| Caucasian | 529 (94) | 334 (95) | 195 (94) |
| Hispanic | 16 (3) | 8 (2) | 8 (4) |
| African American | 4 (1) | 2 (1) | 2 (1) |
| Other | 12 (2) | 9 (2) | 3 (1) |
| Enrollment by continent, n (%) | |||
| North America | 273 (49) | 166 (47) | 107 (52) |
| Europe | 242 (43) | 158 (45) | 84 (40) |
| Australia | 25 (5) | 17 (5) | 8 (4) |
| Asia | 13 (2) | 8 (2) | 5 (2) |
| South America | 8 (1) | 4 (1) | 4 (2) |
| Pancreatic status, n (%) | |||
| Pancreatic insufficient (PI) | 556 (99) | 351 (99) | 205 (99) |
| Pancreatic sufficient (PS) | 5 (1) | 2 (1) | 3 (1) |
| PI/PS genotype based on CFTR alleles, n (%) | |||
| PI/PI | 514 (92) | 324 (92) | 190 (92) |
| PI/PS | 11 (2) | 6 (2) | 5 (2) |
| PI/Unknown | 28 (5) | 20 (5) | 8 (4) |
| Unknown/unknown | 8 (1) | 3 (1) | 5 (2) |
| Pancreatic insufficiency prevalence, n (%) | n =486 | n =311 | n =175 |
| 0.75 – 1.0 (severe) | 473 (97) | 303 (97) | 170 (97) |
| 0.25 – < 0.75 (moderate) | 12 (2) | 8 (3) | 4 (2) |
| < 0.25 (mild) | 1 (1) | 0 (0) | 1 (1) |
| CFTR genotype available, n (%) | n =525 | n =330 | n =195 |
| Phe508del/Phe508del | 298 (57) | 196 (59) | 102 (52) |
| Phe508del/Other | 183 (35) | 106 (32) | 77 (40) |
| Other/Other | 44 (8) | 28 (9) | 16 (8) |
a
CFLD occurs in males more frequently than females (p = 0.0006).
b Pancreatic insufficiency prevalence (PIP) score developed and validated by Ooi and Durie11.