Evaluation of the efficacy and safety of home treatment with the recombinant human C1-inhibitor in hereditary angioedema resulting from C1-inhibitor deficiency

• Published in: International immunopharmacology 2020-03, Vol.80, p.106216, Article 106216
• Main Authors: Andrási, Noémi, Veszeli, Nóra, Holdonner, Ágnes, Temesszentandrási, György, Kőhalmi, Kinga Viktória, Varga, Lilian, Farkas, Henriette
• Format: Article
• Language: English
• Subjects: Angioedema; Complement C1 Inhibitor Protein - administration & dosage; Complement C1 Inhibitor Protein - adverse effects; Complement C1 Inhibitor Protein - genetics; Disease Progression; Drug Administration Schedule; Edema; Efficacy; Female; Hereditary angioedema; Hereditary Angioedema Types I and II - diagnosis; Hereditary Angioedema Types I and II - drug therapy; Hereditary Angioedema Types I and II - genetics; Hereditary Angioedema Types I and II - prevention & control; Home Care Services; Humans; Inhibitors; Injections, Intravenous; Injections, Subcutaneous; Male; Prophylaxis; Prospective Studies; Recombinant human C1 inhibitor acute treatment; Recombinant Proteins - administration & dosage; Recombinant Proteins - adverse effects; Safety; Self Care; Severity of Illness Index; Short term prophylaxis; Symptom Flare Up; Treatment Outcome; Visual Analog Scale
• ISSN: 1567-5769; 1878-1705
• DOI: 10.1016/j.intimp.2020.106216

•Recombinant human C1-inhibitor (rhC1-INH) is an acute therapy for hereditary angioedema.•Studies found that rhC1-INH is effective as a prophylactic treatment as well.•Early administration of rhC1-INH reduces the time to the improvement and to the complete relief of symptoms.•Treatment with rhC1-INH is effective both as on-demand therapy and as short-term prophylaxis.•The repeated administration of rhC1-INH did not reduce its efficacy. Conestat alpha, a C1-inhibitor produced by recombinant technology (rhC1-INH) is an acute treatment for edematous attacks occurring in hereditary angioedema (HAE) with C1-inhibitor deficiency (C1-INH-HAE). Our study evaluated the efficacy and safety of rhC1-INH administered during HAE attacks, and for short-term prophylaxis (STP). Our prospective study analyzed the course of 544 HAE attacks experienced by the 21 C1-INH-HAE patients treated, as well as the outcome of 97 instances of STP implemented with rhC1-INH. Using a purpose-designed questionnaire, the patients recorded relevant, treatment-related information. Time to the administration of rhC1-INH was 90.0 min (median) after the onset of HAE attacks. The symptoms started to improve as early as 60 min after the injection of rhC1-INH, and the attack resolved 730.0 min after treatment. The interval between the onset of the HAE attack and the administration of rhC1-INH correlated with time until the onset of improvement (R = 0.2053 p