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Variability of disease activity in patients with hereditary angioedema type 1/2: longitudinal data from the Icatibant
M Maurer1,2, T Caballero3, W Aberer4
1Dermatological Allergology, Allergie-Centrum-Charité, Department of Dermatology and Allergy, Charité - Universitätsmedizin Berlin, Berlin, Germany.
Insights
Hereditary angioedema attack frequency is mostly consistent yearly for most patients. However, a significant minority experience substantial yearly changes, indicating high individual variability in disease activity over time.
Area of Science:
- Immunology
- Genetics
- Clinical Medicine
Background:
- Hereditary angioedema due to C1 inhibitor deficiency (HAE-1/2) is a chronic, debilitating condition.
- The unpredictable nature of HAE-1/2 significantly burdens patients.
Purpose of the Study:
- To analyze longitudinal registry data to characterize temporal changes in disease activity in HAE-1/2 patients.
- Investigate disease activity patterns over a 7-year period using the Icatibant Outcome Survey (IOS).
Main Methods:
- Analysis of data from the international observational registry, Icatibant Outcome Survey (IOS).
- Retrospective analysis of angioedema attack frequency over 7 years, comparing patients with and without long-term prophylaxis (LTP).
Main Results:
- At the population level, attack frequency remained generally consistent over 7 years.
- While most patients maintained similar attack rates, 31-51% experienced significant annual changes (≥5 attacks).
- A notable proportion (17-50%) of patients with large attack changes (≥10 attacks) between Years 1 and 2 continued to experience such variability in subsequent years.
Conclusions:
- HAE-1/2 attack frequency shows population-level consistency but significant intra-patient variability.
- A substantial percentage of patients experience fluctuating disease activity, highlighting the need for personalized monitoring and management strategies.
Background:
Hereditary angioedema due to C1 inhibitor deficiency (HAE-1/2) is a chronic and debilitating disease. The unpredictable clinical course represents a significant patient burden.
Objective:
To analyse longitudinal registry data from the Icatibant Outcome Survey (IOS) in order to characterize temporal changes in disease activity in patients with HAE-1/2.
Methods:
Icatibant Outcome Survey (NCT01034969) is an international observational registry monitoring the clinical outcomes of patients eligible for icatibant treatment. The current analyses are based on data collected between July 2009 and July 2019. Retrospective data for attacks recorded in the 12 months prior to IOS enrolment and for each 12-month period up to 7 years were analysed.
Results:
Included patients reported angioedema attacks without long-term prophylaxis (LTP; n = 315) and with LTP (n = 292) use at the time of attack onset. Androgens were the most frequently used LTP option (80.8%). At the population level, regardless of LTP use, most patients (52-80%) reporting <5 attacks in Year 1 continued experiencing this rate; similarly, many patients (25-76%) who reported high attack frequency continued reporting ≥10 attacks/year. However, year on year, 31-51% of patients experienced notable changes (increase/decrease of ≥5 attacks) in annual attack frequency. Of patients who reported an absolute change of ≥10 attacks from Year 1 to 2, 17-50% continued to experience a change of this magnitude in subsequent years.
Conclusion:
At the population level, attack frequency was generally consistent over 7 years. At the small group level, 28.8-34.5% of patients reported a change in attack frequency of ≥5 attacks from Year 1 to Year 2; up to half of these patients continued to experience this magnitude of variation in disease activity in later years, reflecting high intra-patient variability.
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