Decreasing Attacks and Improving Quality of Life through a Systematic Management Program for Patients with Hereditary Angioedema

Abstract

Introduction: Prevention of attacks is a major goal in management of patients with hereditary angioedema (HAE). We aimed to investigate the effects of a systematic intervention for HAE patients. Methods: Thirty-three patients with HAE with C1-inhibitor deficiency, belonging to a single family, participated in a management program coordinated by an allergist/immunologist. Angioedema attacks before intervention were ascertained by interviews and emergency room charts and recorded prospectively by patients or caregivers after enrollment. Mean number of attacks/month was compared at 12 months preintervention and 8 and 14 months within intervention. Patient-reported outcome instruments were used to assess quality of life, including HAE Quality of Life (HAE-QoL) questionnaire, psychological conditions, and work impairment, at baseline and 8 and 14 months within intervention. Data were stored in REDCap platform and analyzed by adjusted Bayesian models of double Poisson regression. Results: Mean number of attacks/month significantly decreased (95% credible interval [CrI] excluding 0) from 1.15 preintervention to 0.25 and 0.23, 8 and 14 months within intervention, with mean decreases of −0.89 (95% CrI: −1.21 to −0.58) and −0.92 (95% CrI: −1.22 to −0.60), respectively. HAE-QoL scores showed mean total increases of 15.2 (95% CrI: 1.23–29.77) and 26 (95% CrI: 14.56–39.02) at 8 and 14 months within the study, as compared to baseline, revealing marked improvement in quality of life. Significant increase in role-emotional and reduction of depression, stress, and anxiety were observed at 14 months. Conclusion: A systematic approach integrating HAE-specific care with effective handling of psychological issues decreased the number of attacks and improved quality of life, targets for best practice in HAE.

A narrative review of recent literature of the quality of life in hereditary angioedema patients

“… Author, Year Country Type of study Treatment Follow up QoL instrument Results summary Nunes et al. (2021) 24 Brazil NR Systematic intervention 14 months HAE-QoL Mean total HAE-QoL scores differences between baseline and further visits: Increase of 15.2 (95% CrI: 1.23–29.77) at 8 months. Increase of 26 (95% CrI: 14.56–39.02) at 14 months.…”

“…No impairment (0 score) was reported by 88.1%, 73.8%, 73.8%, and 57.2% of patients for absenteeism, presenteeism, work productivity loss and activity impairment domains, respectively. 24 Different study designs may explain divergences between results from Brazil and Japan and Sweden; however, further analysis to determine the reasons for such regional differences are still needed. 14 , 23 , 24 In addition, the Brazilian study was conducted aiming to analyze the effect of an intervention and reinforces the importance of access to treatment.…”

“… 24 Different study designs may explain divergences between results from Brazil and Japan and Sweden; however, further analysis to determine the reasons for such regional differences are still needed. 14 , 23 , 24 In addition, the Brazilian study was conducted aiming to analyze the effect of an intervention and reinforces the importance of access to treatment.…”

Treatment of hereditary angioedema: When the goal is having a normal life

“…Nunes et al 10 conducted a systematic intervention for 33 patients from a single large Brazilian family with HAE-C1-INH; the trial resulted in a significant reduction in mean attacks per month, improvement in quality of life, and a decrease in anxiety and depression within 14 months of intervention. 10 In the vast majority of these patients, LTP was performed, with second-line alternatives including attenuated androgen, tranexamic acid, and progestin. At present, the cost of first-line medications for LTP in Brazil substantially restricts their availability to patients with HAE; however, the results of this real-life study suggest that improving access to HAE-targeted medications for on-demand treatment of attacks contributes a great deal to better health-related quality of life.…”

“…At present, the cost of first-line medications for LTP in Brazil substantially restricts their availability to patients with HAE; however, the results of this real-life study suggest that improving access to HAE-targeted medications for on-demand treatment of attacks contributes a great deal to better health-related quality of life. 10 We are experiencing exceptional advances in the treatment of patients with HAE, with effective options for disease control allowing patients to have total control of symptoms and lead normal lives. In our view, berotralstat fulfills a significant unmet need for HAE treatment options by providing a choice of an oral medication for LTP with good efficacy and acceptable safety profile.…”