Lung density and lung function measurements in RCTs and observational studies with augmentation therapy in AATD
| Study | Year | Treatment and comparator | Subjects n | Treatment duration years | Infusion frequency | Decline in lung density at TLC g⋅L−1⋅year−1 | FEV1 decline mL⋅year−1 |
| RCT versus placebo | |||||||
| Dirksen [44] | 1999 | 250 mg⋅kg−1 α1-AT versus 625 mg⋅kg−1 albumin solution | 58 | 3 | Monthly | 2.6 versus 1.5 (p=0.07) | 59 versus 79 (p=0.25) |
| Dirksen [45] | 2009 | 60 mg⋅kg−1 α1-AT (Prolastin) versus 2% albumin solution | 77 | 2 | Weekly | 1.4 versus 2.2 (p=0.06) | |
| Chapman [46] | 2015 | 60 mg⋅kg−1 α1-AT (Zemaira) versus lyophilised preparation | 180 | 2 | Weekly | 1.5 versus 2.2 (p=0.03) | 44.3 versus 44.9 (p=0.21) |
| Observational with control | |||||||
| Seersholm [47] | 1997 | 60 mg⋅kg−1 α1-AT (Prolastin or Trypsone) versus no therapy | 295 | 1 | Weekly | 53 versus 75 (p=0.02) | |
| AATD registry group [48] | 1998 | 60 mg⋅kg−1 α1-AT (Prolastin) versus no regular therapy | 1129 | 1–7 | Weekly | 73 versus 93 (p=0.01) | |
| Wencker [49] | 2001 | 60 mg⋅kg−1 α1-AT versus data prior to infusion | 96 | 1 | Weekly | 34 versus 49 (p=0.02) | |
| Tonelli [50] | 2009 | Any dose regimen versus no therapy | 164 | 3.5 | 37 versus 46 (p=0.05) |
RCT: randomised controlled trial; TLC: total lung capacity.