Real-world outcomes in hereditary angioedema: first experience from the Icatibant Outcome Survey in the United Kingdom

Abstract

Table 1 Patient demographics and baseline characteristics Characteristic Patients with HAE type I or II UK (N = 73) Non-UK (N = 579) Age at extract, years Mean (SD) 42.9 (14.7)a 45.1 (15.2)b Median (range) 42.2 (18–82)a 43.3 (6–86)b Gender, n (%) Male 29 (39.7) 235 (40.6) Female 44 (60.3) 344 (59.4) Age at first symptoms, years Mean (SD) 11.3 (9.5)c 14.1 (11.2)d Median (IQR) 10.0 (5.0, 16.0)c 13.0 (6.0, 19.0)d Age at diagnosis, years Mean (SD) 21.5 (12.7)e 24.6 (15.9)f Median (IQR) 18.9 (12.6, 30.0)e 21.0 (13.2, 34.2)f Delay between first symptoms and diagnosis, years Mean (SD) 9.5 (13.9)c 10.5 (13.6)g Median (IQR) 6.2 (0.0, 17.5)c 5.9 (0.4, 17.6)g Employment status, n (%)h Employee 46 (63.0) 218 (52.0) Self-employed 7 (9.6) 15 (3.6) Homemaker 3 (4.1) 20 (4.8) Leave of absence/sabbatical 1 (1.4) 2 (0.5) Pre-school 0 4 (1.0) Retired 3 (4.1) 42 (10.0) Student 8 (11.0) 53 (12.6) Unemployed 4 (5.5) 34 (8.1) Other/unknown 4 (5.5) 43 (10.3) HAE hereditary angioedema, IQR interquartile range, SD standard deviation an = 73 bn = 579 cn = 58 dn = 497 en = 70 fn = 535 gn = 486 hPatients could have more than one employment status (UK sample n = 73, non-UK patients n = 419) Socioeconomic data Employment status for UK and non-UK patients at IOS entry is shown in Table 1. For non-UK patients, the median number of untreated attacks per patient was lower (1.0 versus 2.0, respectively) in the IOS observation period than in the period prior to IOS entry. Since C1-INH-treated attacks were not recorded, no conclusion can be drawn as to overall percentages. Table 4 Time to treatment, time to resolution and duration of icatibant-treated HAE attacks Endpoint Patients with HAE type I or II UK (N = 73) Non-UK (N = 579) p valueb n a Mean (SD) Median (IQR) n a Mean (SD) Median (IQR) Time from attack onset to treatment, hc 222 2.9 (4.8) 0.8 (0.4, 3.0) 1120 3.9 (7.3) 1.3 (0.5, 4.0) 0.0632 Time to complete symptom resolution, hd 222 10.5 (14.1) 6.0 (1.3, 14.0) 1120 11.8 (16.1) 5.8 (2.0, 14.1) 0.2774 Duration of attack, he 222 13.4 (15.4) 9.0 (2.5, 18.5) 1120 15.6 (18.9) 8.6 (4.0, 20.0) 0.1022 HAE hereditary angioedema, IQR interquartile range, n number of evaluable attacks, SD standard deviation aAttacks with complete data for time to treatment, time to complete resolution and attack duration, excluding attacks treated > 100 h after attack onset bMixed-model analysis of repeated measures comparing the UK versus non-UK IOS datasets cTime between the start of the attack and the first icatibant injection dTime between first injection of icatibant and complete resolution of symptoms eTime between start of attack and complete resolution of symptoms Severity of attacks In the UK, 65.5% (279/426) of attacks before treatment were classified (where data were available) as severe/very severe; 26.1% (111/426) as moderate; and 8.5% (36/426) as mild or very mild. [...]the ease of use of icatibant has enabled a greater proportion of patients to self-administer treatment for HAE attacks than is possible with C1-INH treatment, which requires intravenous access.