Table 1.
Patient Demographics and Characteristics
| AATD with Liver Disease | AATD with Both Liver and Lung Disease | ||||
|---|---|---|---|---|---|
| Patients Aged <1 Year (n=41) | Patients Aged 1–5 Years (n=24) | Patients Aged 6–17 Years (n=58) | Patients Aged ≥18 Years (n=771) | Patients Aged ≥18 Years (n=541) | |
| Age, years, median (IQR) | 0 (0–0) | 3 (2–5) | 14 (10–16) | 49 (38–57) | 55 (46–61) |
| Sex, male, n (%) | 25 (61) | 13 (54) | 36 (62) | 451 (58) | 275 (51) |
| Time in health state, months, median (IQR)a | 37 (22.6–55) | 22 (9.0–36) | 36 (15.0–60) | 22 (10–40) | 21 (10–38) |
| Most common comorbidities, n (%)b | N/Ac | ||||
| Asthma | 2 (8) | 8 (14) | 62 (8) | 90 (17) | |
| Diabetes mellitus | 0 | 2 (3) | 107 (14) | 107 (20) | |
| Hyperlipidemia | 0 | 3 (5) | 208 (27) | 166 (31) | |
| Hypertension | 2 (8) | 2 (3) | 221 (29) | 217 (40) | |
| Liver cirrhosis | 0 | 0 | 15 (2) | 60 (11) | |
| Liver/gall bladder/pancreatic disease | 1 (4) | 0 | 80 (10) | 132 (24) | |
| Obesity | 1 (4) | 8 (14) | 124 (16) | 129 (24) | |
| Sleep disorders | 0 | 2 (3) | 97 (13) | 100 (18) | |
| Study liver disease definitiond | 0 | 0 | 0 | 93 (17) | |
| Study lung disease definitiond | 1 (4) | 4 (7) | 0 | 127 (23) | |
| Charlson Comorbidity Index, n (%) | N/Ac | ||||
| 0 | 21 (88) | 48 (83) | 515 (67) | 191 (35) | |
| 1 | 2 (8) | 8 (14) | 138 (18) | 162 (30) | |
| 2 | 0 | 1 (2) | 73 (10) | 74 (14) | |
| 3 | 0 | 0 | 30 (4) | 52 (10) | |
| ≥4 | 1 (4) | 1 (2) | 15 (2) | 62 (11) | |
| Payer type, n (%) | |||||
| Commercial | 25 (61) | 12 (50) | 42 (72) | 511 (66) | 353 (65) |
| Self-insured | 12 (29) | 9 (38) | 13 (22) | 224 (29) | 151 (28) |
| Medicare risk | 0 | 0 | 0 | 21 (3) | 25 (5) |
| Medicaid | 4 (10) | 3 (13) | 3 (5) | 12 (2) | 8 (1) |
| Other/unknown | 0 | 0 | 0 | 3 (<1) | 4 (<1) |
| Geographic region of the USA, n (%) | |||||
| South | 13 (32) | 10 (42) | 22 (38) | 341 (44) | 272 (50) |
| Midwest | 13 (32) | 10 (42) | 16 (28) | 187 (24) | 129 (24) |
| Northeast | 8 (20) | 2 (8) | 12 (21) | 147 (19) | 76 (14) |
| West | 7 (17) | 2 (8) | 7 (12) | 86 (11) | 57 (11) |
| Unknown/missing | 0 | 0 | 1 (2) | 10 (1) | 7 (1) |
Notes: aDifference in time from first lung disease diagnosis (after liver disease diagnosis), or first liver disease diagnosis (after lung disease diagnosis), or first liver or first lung disease diagnosis (if within 90 days of each other) to last day of continuous enrollment period. bPresent in ≥10% of patients in at least one of the subgroups. cPatients <1 year were indexed at age 0 years and therefore did not have a 6-month baseline period for the collection of clinical characteristics. dClinician determined specific liver/lung disease diagnosis codes as AATD-specific; separately reported comorbidities may include broader definitions (eg alcoholic liver cirrhosis).
Abbreviations: AATD, alpha-1 antitrypsin deficiency; IQR, interquartile range; N/A, not available.