Augmentation Therapy for Alpha-1 Antitrypsin Deficiency: Improved Survival and Quality of Life Compared to Matched Augmentation Naive Controls Followed for Up to 15 Years

Assessing the efficacy of Alpha1-Proteinase inhibitor (human) augmentation therapy for Alpha1-Antitrypsin deficiency – Related emphysema: Challenges and opportunities

“…An 11.5 % absolute reduction in mortality with A 1 -PI was observed in a retrospective cross-country analysis by Sandhaus et al [ 52 ], as well as a substantial reduction in lung transplantation and a slight reduction in the rate of deterioration in SGRQ. However, differences in care between the U.S. and U.K. may have affected the reported outcomes of this retrospective cross-country comparison.…”

“…A retrospective analysis of two prospectively followed PI*ZZ (“or worse” genotype) AATD patient cohorts with emphysema in two countries differing in access to A 1- PI augmentation therapy was undertaken [ 52 ]. The U.S. cohort comprised patients receiving A 1 -PI and followed in AlphaNet's Disease Management and Prevention program with monthly telephone interviews.…”

“…Annual worsening of SGRQ total was on average 1.3 points worse/year in control group patients compared to those receiving A 1 -PI (95 % CI 0.41 to 2.19, p = 0.004). Features of the AlphaNet Program other than A 1 -PI augmentation therapy and other differences in care between the U.S. and U.K. may have affected the reported outcomes of this retrospective cross-country comparison [ 52 ].…”

Disease burden associated with alpha-1 antitrypsin deficiency: systematic and structured literature reviews

“…Where reported, the mean age ranged from 40.7 years [14] to 60.3 years [75], and there were more males than females in 19 out of 30 studies, with the proportion of males ranging from 50.3% to 83% (supplementary table S2) [12,[76][77][78]. Most studies assessed QoL using the SGRQ (22 studies; table 3 [12,14,[73][74][75][76][78][79][80][81][82][83][84][85][86][87][88][89][90][91][92][93]). Other disease-specific patient-reported outcomes included the COPD Assessment Test (CAT), the Chronic Respiratory Disease Questionnaire (CRQ) and the Living with COPD (LCOPD) scale.…”

Alpha 1 Antitrypsin Therapy in Patients with Alpha 1 Antitrypsin Deficiency: Perspectives from a Registry Study and Practical Considerations for Self-Administration During the COVID-19 Pandemic

“…26 Benefits on QoL deterioration were also seen. 27 There is likely to be a heavy reliance on patient registries to support the clinical efficacy of AAT therapy on health-related outcomes in the future.…”