The Association Between Patient-Reported Outcomes and Pulmonary Exacerbation Frequency in Patients with Alpha-1 Antitrypsin Deficiency

Abstract

Introduction: the negative effect that pulmonary exacerbations have on alpha-1 antitrypsin deficiency patients’ lung function has been explored and proven. Nonetheless, studies addressing the association between patient-reported outcomes (PROs) and exacerbation frequency in patients with AATD are lacking. Study Aim: To analyse the association between pulmonary exacerbations, and patientreported outcomes in alpha-1 antitrypsin deficiency patients. Method: Patients’ data was extracted from The AATD cohort/registry. Information regarding patients’ demographics, genotype, smoking status, smoking rate, COPD diagnosis, and FEV1% were included. The PROs include The St. George's Respiratory Questionnaire (SGRQ), The COPD Assessment Test (CAT), Modified Medical Research Council Dyspnoea Scale (mMRC). The exacerbation frequency in the last 12 months was reported by patients. Statistical association and correlation were reported. Patients with 2 or more exacerbations were labelled as frequent exacerbators (FE), and patients with less than 2 exacerbations were labelled infrequent exacerbators (IE) in the last 12 months. Also, patients were grouped as non-exacerbators with 0 exacerbations or exacerbators with 1 or more exacerbations in the last 12 months. Results: A total of 234 patients were included in this study. The FE group (n=69) reported significantly poorer PROs when compared to the IE group (n=165), even though FEV1% was not significantly different between the two groups (p = 0.055). when comparing exacerbators to non-exacerbators, all PROs and FEV1% were significantly different with poorer results seen in the exacerbators group (p < 0.001 for SGRC and CAT and p = 0.007 for mMRC). Spearman rank correlation supported that lower FEV1% and more exacerbations are significantly correlated with poorer PROs (p ≤ 0.001 in all correlations). The regression model proved that more exacerbations and declined FEV1% are significant predictors of PROs worsening. Conclusion: Frequent exacerbations and declined FEV1% are associated with worse patientreported outcomes in patients with alpha-1 antitrypsin deficiency.