Exercise Capacity May Be More Strongly Associated with Health Status in Alpha-1 Antitrypsin COPD Patients than in Alpha-1 Antitrypsin Replete COPD Patients

Physical Activity, Exercise Capacity and Sedentary Behavior in People with Alpha-1 Antitrypsin Deficiency: A Scoping Review

“…A number of different study designs were identified: six cross-sectional studies, 24–29 six prospective studies, 30–35 three retrospective, 36–38 three randomized controlled trials (RCT), 16 , 39 , 40 and one non-randomized and non-concurrent trial. 41 Ten included reports were conference abstracts of which there were three retrospective studies, 42–44 two RCTs, 45 , 46 two case studies, 47 , 48 two cross-sectional 49 , 50 and one prospective study. 51 A further eight protocols 52–59 were identified: one cross-sectional study, 52 two prospective studies, 53 , 54 four RCTs 55–58 and one non-randomized parallel group study.…”

“… Menon et al 2017 48 Conference abstract – Case study Distance walked in: can now walk from half a block to a block Treatment: alpha 1 proteinase inhibitor Control/comparator: N/A Not reported Murray et al 2014 49 Conference abstract – cross sectional 6MWT Treatment/intervention: N/A Control/comparator: N/A To explore the impact of ZZ AATD on the diffusing capacity of the lung for carbon monoxide, and its relationship with functional exercise impairment among the Irish population of ZZ AATD individuals. Durkan et al 2019 50 Conference abstract – cross sectional 6MWT Treatment: N/A Control/comparator: Normal COPD To explore whether exercise capacity is a better surrogate for health status in AATD COPD (ZZ) patients compared to alpha 1 antitrypsin replete MM COPD patients Heinzelmann et al 2015 51 Conference abstract –prospective 6MWT Peak work rate test Treatment: 3 week exercise training Control/comparator: Normal COPD To explore if skeletal muscle adaptation is impaired in A1AD compared with COPD Strange et al 2015 52 Protocol- Cross-sectional study 6MWT Intervention/treatment: N/A Control/comparator: comparing those on augmentation to those not receiving augmentation To compare the lower respiratory tract microbiome and virome population diversity and content in age and stage matched individuals with PiZZ (based on Global Initiative for Chronic Obstructive Lung Disease [GOLD] guidelines) not receiving augmentation therapy, individuals with PiZZ receiving augmentation therapy, PiMZ individuals not receiving augmentation therapy, and (normal nondeficient genotype) individuals with PiMM who have COPD 2. To determine correlations between BAL and peripheral blood gene expression patterns and patterns in lung microbial and viral populations across all cohorts 3.…”

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

“…No difference between these patient groups was reported in two studies [77,94]. Better QoL was reported in patients with AATD with COPD versus general COPD in patients with the PiZZ AATD genotype in two studies [87,95].…”

“…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.…”