Table 2

Lung density and lung function measurements in RCTs and observational studies with augmentation therapy in AATD

StudyYearTreatment and comparatorSubjects nTreatment duration yearsInfusion frequencyDecline in lung density at TLC g⋅L−1⋅year−1FEV1 decline mL⋅year−1
RCT versus placebo
 Dirksen [44]1999250 mg⋅kg−1 α1-AT versus 625 mg⋅kg−1 albumin solution583Monthly2.6 versus 1.5 (p=0.07)59 versus 79 (p=0.25)
 Dirksen [45]200960 mg⋅kg−1 α1-AT (Prolastin) versus 2% albumin solution772Weekly1.4 versus 2.2 (p=0.06)
 Chapman [46]201560 mg⋅kg−1 α1-AT (Zemaira) versus lyophilised preparation1802Weekly1.5 versus 2.2 (p=0.03)44.3 versus 44.9 (p=0.21)
Observational with control
 Seersholm [47]199760 mg⋅kg−1 α1-AT (Prolastin or Trypsone) versus no therapy2951Weekly53 versus 75 (p=0.02)
 AATD registry 
 group [48]199860 mg⋅kg−1 α1-AT (Prolastin) versus no regular therapy11291–7Weekly73 versus 93 (p=0.01)
 Wencker [49]200160 mg⋅kg−1 α1-AT versus data prior to infusion961Weekly34 versus 49 (p=0.02)
 Tonelli [50]2009Any dose regimen versus no therapy1643.537 versus 46 (p=0.05)

RCT: randomised controlled trial; TLC: total lung capacity.