Table 1.
Description of the population included in the main studies with augmentation therapy.
| Study | N | Age | FEV1 | Mutations | |||
|---|---|---|---|---|---|---|---|
| Eligibility Criteria | Cohort Results * | Eligibility Criteria | Cohort Result * | Eligibility Criteria | Cohort Result | ||
| Observational studies with control | |||||||
| Seersholm N, et al.; WALT. Eur Respir J, 1997 [18] | 295 | NR | 46 (8) | FEV1 < 65% or decline > 120 mL/year | 37 (14)% | Severe AATD (NR) | NR |
| NHLBI Registry. AJRCCM, 1998 [21] | 927 | ≥18 years | 46 (11) | NR | <35%: 43.6% 35–49%: 21.1% 50–79%: 16.2% ≥80%: 19.1% |
AAT ≤ 11 µM or ZZ | NR |
| Wencker M, et al.; WALT. Eur Respir J, 1998 [19] | 443 | >18 years | 45 (7) | FEV1 < 65% or decline > 120 mL/year | Ex-smokers: 35.5 (14.8)% Non-smokers: 42.2 (18.2)% |
AAT < 50 mg/dL (nephelometry) AAT < 80 mg/dL (immunodiffusion) |
ZZ: 88.9% SZ: 7.1% Other/unknown: 4.0% |
| Wencker M, et al.; WALT. Chest, 2001 [20] | 96 | NR | 44 (8) | FEV1 < 65% or decline > 120 mL/year | 41 (17.3)% | AAT < 35% | ZZ: 85% SZ: 8% Other: 3% |
| Stoller JK, et al.; NHLBI. Chest, 2003 [22] | 747 | NR | 48 (9) | NR | 37 (18)% | AAT ≤ 11 µM or ZZ or null | NR |
| Tonelli AR, et al. Int J COPD, 2009 [23] | 164 | NR | 61.3 (0.7) | NR | 43 (2)% | ZZ | ZZ |
| Barros-Tizón JC, et al. Ther Adv Respir Dis, 2012 [24] | 127 | >18 years | 51.7 (9.1) | NR | 1.25 (0.50) L | AAT ≤ 11 µM (50 mg/dL) and ZZ. rare or null | ZZ: 93.6% SZ: 0.8% Other: 5.6% |
| Clinical trials | |||||||
| Dirksen A, et al.; Danish–Dutch study. AJRCCM, 1999 [3] | 56 | NR | Danish: 50.4 (1.62) Dutch 45.1 (1.17) |
FEV1 30–80% |
Danish: 1.5 (0.9) L Dutch: 1.6 (0.1) L |
ZZ | ZZ |
| Dirksen A, et al.; EXACTLE. Eur Respir J, 2009 [2] | 77 | ≥18 years | 54.7 (8.4) | NR | 46.3 (19.6)% | AAT ≤ 11 µM | ZZ or Zn |
| Chapman KR, et al.; RAPID. Lancet, 2015 [4] | 180 | 18–65 | 53.8 (6.9) | FEV1 35–70% |
47.4 (12.1)% | AAT ≤ 11 µM | ZZ: 93% Other: 7% |
* Numerical data expressed as mean (standard deviation). FEV1: forced expiratory volume in 1 s (expressed as absolute values in liters or as percentage of the theoretical value the patient should have according to its age, gender, height, weight, and race). NR, not reported; AAT, alpha1 antitrypsin ; AATD, Alpha1 antitrypsin deficiency; NHLBI, National Health Lung Blood Institute; COPD, chronic obstructive pulmonary disease; WALT, from the German abbreviation: Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen; AJRCCM, American Journal of Respiratory and Critical Care Medicine