The Icatibant Outcome Survey: experience of hereditary angioedema management from six European countries

Abstract

BackgroundHereditary angioedema (HAE) due to C1‐inhibitor deficiency (C1‐INH‐HAE) is a rare, potentially fatal, bradykinin‐mediated disease. Icatibant is a bradykinin B2 receptor antagonist originally approved in 2008 in the European Union and 2011 in the United States as an acute therapy option for HAE attacks in adults.ObjectiveTo compare demographics, disease characteristics and treatment outcomes of icatibant‐treated HAE attacks in patients with C1‐INH‐HAE enrolled in the Icatibant Outcome Survey across six European countries: Austria, France, Germany, Italy, Spain and the UK.MethodsThe Icatibant Outcome Survey [IOS; Shire, Zug, Switzerland (NCT01034969)] is an international observational study monitoring the safety and effectiveness of icatibant. Descriptive, retrospective analyses compared IOS country data derived during July 2009–April 2015.ResultsOverall, 481 patients with C1‐INH‐HAE provided demographic data. A significant difference across countries in age at onset (P = 0.003) and baseline attack frequency (P < 0.001) was found although no significant differences were found with respect to gender (majority female; P = 0.109), age at diagnosis (P = 0.182) or delay in diagnosis (P = 0.059). Icatibant was used to treat 1893 attacks in 325 patients with majority self‐administration in all countries. Overall, significant differences (all P < 0.001) were found across countries in time to treatment [median 1.8 h; median range: 0.0 (Germany–Austria) to 4.4 (France) h], time to resolution [median 6.5 h; median range: 3 (Germany–Austria) to 12 (France) h] and attack duration [median 10.5 h; median range: 3.1 (Germany–Austria) to 18.5 (France) h].ConclusionThese data form the first European cross‐country comparison of disease characteristics and icatibant use in patients with C1‐INH‐HAE who are enrolled in IOS. International variation in icatibant practice and treatment outcomes across the six European countries assessed highlight the need to further investigate the range of country‐specific parameters driving regional variations in icatibant use.

Management of patients with hereditary angioedema in Germany: comparison with other countries in the Icatibant Outcome Survey

“…Data from the IOS have clearly shown that early icatibant treatment results in earlier resolution of attacks and that real‐world outcomes of icatibant are comparable with the aforementioned controlled studies . More recently, a range of IOS publications have further described icatibant use and disease characteristics for HAE‐C1‐INH patients in the real‐world setting across the IOS countries …”

“…9 More recently, a range of IOS publications have further described icatibant use and disease characteristics for HAE-C1-INH patients in the real-world setting across the IOS countries. [10][11][12][13][14][15] A recent country-specific IOS analysis, using 2015 data, clearly identified German patients with HAE-C1-INH who are enrolled in IOS administer icatibant to treat their attacks significantly earlier, following symptom onset, than similarly diagnosed IOS patients from Austria, France, Italy, Spain, and the United Kingdom. 14 Here, we report data from IOS that focus on icatibant treatment outcomes in patients with HAE-C1-INH from seven HAE specialist centres across Germany and compares outcomes with patients from 11 other IOS countries.…”

Unnecessary abdominal interventions in patients with hereditary angioedema

“…HAE betrifft alle ethnischen Gruppen [ 11 ] . In einer Studie der Icatibant Outcome Survey Group in sechs europäischen Ländern war die Verteilung hinsichtlich Geschlecht und HAE-Typ in allen Ländern ähnlich, mit einem Überwiegen der Frauen, wie auch Maurer et al feststellten [ 12,13 ] . Während der letzten siebzig Jahre hat das Verständnis der Krankheit selbst sowie der therapeutischen Optionen zugenommen.…”

Disease Severity, Activity, Impact, and Control and How to Assess Them in Patients with Hereditary Angioedema

“…A conceptual model has been developed to illustrate the health-related QoL impacts of HAE ( 21 ). The model depicts five main areas of disease burden: unnecessary treatments and procedures, symptom triggers, attack impacts, caregiver impacts, and long-term impacts.…”

“…During the last 10 years, several new evidence-based and very effective treatments have become available. For the treatment of attacks, early therapy and self-administration are recommended and are now widely practiced in many countries ( 21 ). Long-term prophylaxis with intravenous plasma derived C1-INH has been introduced ( 22 ), and additional prophylactic treatment options are under development ( 23 ).…”