Home Therapy with Plasma-Derived C1 Inhibitor: A Strategy to Improve Clinical Outcomes and Costs in Hereditary Angioedema

Abstract

Background: Attacks of hereditary angioedema with C1 inhibitor deficiency (C1-INH-HAE) are commonly treated in the emergency department. Self-administration is emerging as an effective treatment option. In this study, we assessed the impact of home therapy with plasma-derived C1 esterase inhibitor (pdC1-INH) concentrate on treatment outcomes and costs. Methods: This is an observational study in C1-INH-HAE patients who switched to home therapy with pdC1-INH (Berinert®) after learning intravenous self-infusion in a training course at a center in Southern Italy. Before starting home therapy and after the first year of home therapy, patients were interviewed about their treatment and outcomes during the prior 12 months. Annual costs were analyzed by cost minimization from the Italian health care payer perspective and the societal perspective. Outcomes and costs before and after the switch to home therapy were compared. Results: The training course was attended by 36 participants, 17 (47.2%) of whom decided to switch to home therapy. This therapy was associated with a significant decrease in the mean annual number of hospitalizations (16.8 vs. 2.1, p = 0.003) and missed work/school days (20.3 vs. 7.1, p = 0.037) compared to conventional treatment. The times from symptom onset to treatment administration and from treatment administration to symptom improvement/resolution were not significantly different between the two strategies. The mean annual per-patient costs decreased with home therapy from EUR 30,010.57 to EUR 26,621.16 (11.3% saving) and from EUR 29,309.34 to EUR 26,522.04 (9.5% saving) from the societal and payer perspective, respectively. Conclusions: Home therapy with pdC1-INH is a feasible strategy for the management of C1-INH-HAE and may result in cost savings.

International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency

“…PdC1‐INH Ci is approved by the FDA and EMA for prophylaxis for adolescents and adults and is licensed for home/self‐therapy. In Brazil, pdC1‐INH Be is approved for home/self‐therapy and for pediatric and adult use 95, 99, 103, 104, 105, 106, 107.…”

“…Because no drugs for attacks had been approved by the FDA at that time, and only FFP was available for attacks, this study could reflect the situation of patients with established C1‐INH‐HAE diagnosis in countries where attack therapy is not available as in most of Latin American, Asian, or African countries. In addition, we should consider the high cost of being treated in the ED in comparison with self‐treatment at home 106.…”

Recurrent Angioedema: Occurrence, Features, and Concomitant Diseases in an Italian Single-Center Study

“…Recent evidence suggests novel laboratory methods for the differential diagnosis of AAE and HAE when the determination of C1-INH is not yet available [56] . There is widespread agreement on therapeutic goals (i.e., to reduce mortality and morbidity, reduce the frequency of attacks, improve quality of life, and train patients in selfadministration) in C1-INH-related AE whereas no guidelines are available concerning the treatment of ACEI-RA and I-AAE [22,23,[57][58][59] .…”

Personalized Medicine in Allergy

“…Regular profilactic treatment with C1-INH may be necessary for patients having 2 or more attacks per week. Recent evidence suggests that self-administration of C1-INH is emerging as an effective treatment to improve clinical outcomes and reduce costs in HAE 73. Ecallantide (Kalbitor → , Dyax Corp) is a 60-amino acid recombinant protein approved by the FDA, but not by the EMA, as s.c. injection in the treatment of acute attacks of angioedema 74.…”