The aim of this study is to conduct an in-depth analysis of the Burden of Disease (BoD) perceived by patients with Hereditary Angioedema (HAE), through daily prospective observations based on Ecological Momentary Assessment (EMA) via digital surveys and standardised questionnaires.
Participants will answer online survey questions about their perceived burden of disease for 8 consecutive weeks. The main hypothesis is that daily prospective observation (EMA) will reveal a higher and more fluctuating burden of disease compared to traditional retrospective scales, providing a more accurate representation of the impact of HAE on patients' daily lives.
Confirmed diagnosis of Type 1 or Type 2 hereditary angioedema;
Age ≥ 18 years;
Ability to understand instructions and provide informed consent;
Ownership and proficiency in using a personal smartphone compatible with the m-Path application (Android or iOS);
Diagnosis of acquired angioedema or other forms of angioedema unrelated to C1-inhibitor deficiency;
Severe cognitive or psychiatric disorders that compromise the ability to complete the questionnaires independently;
Age < 18 years;
The study involves the participation of adult patients with a confirmed diagnosis of hereditary angioedema, belonging to the ITACA (Italian Network for Hereditary and Acquired Angioedema) national cohort and receiving follow-up care at the IRCCS Maugeri Milano (Via Camaldoli) center. Participation in the study is voluntary and subject to the signing of informed consent. Patients will complete the questionnaires independently via their own smartphones, with no requirement for in-person visits.
Cohort
Prospective
ecological momentary assessment
Other interventionA real-time data collection method involving brief, repeated digital surveys completed by participants in their natural environments. Participants will receive two notifications every other day for 8 weeks via a mobile application. Surveys are designed to assess physical symptoms, socio-occupational status, and emotional states related to Hereditary Angioedema (HAE) to capture daily fluctuations in disease burden.
Emotional Burden via Ecological Momentary Assessment (EMA)
Evaluation of emotional states (anxiety, mood, stress, and fear of attacks) through a Visual Analogue Scale (VAS). Each item is scored on a range from 0 to 100, where 0 indicates "no emotional impact" and 100 indicates "maximum emotional distress.
Socio-occupational Impairment via Ecological Momentary Assessment (EMA)
Assessment of the impact of Hereditary Angioedema (HAE) on daily, social, and professional activities. Measured via a scale ranging from 1 to 10, where 1 indicates "no impairment" and 10 indicates "maximum perceived impairment"
Disease Activity
Assessment of the physical burden using the Angioedema Activity Score (AAS): A daily symptom diary, total score from 0 to 15.
Angioedema Quality of Life
Assessment of quality of life impairment using the AE-QoL questionnaire. The total score ranges from 17 to 85, where higher scores indicate greater impairment.
Anxiety and Depressive Symptoms
Measured via the Patient Health Questionnaire-4 (PHQ-4), a 4-item screening scale for anxiety and depression. Each item is scored from 0 to 3. The total score ranges from 0 to 12, where higher scores indicate more severe symptoms of anxiety and depression.
Work Productivity and Activity Impairment (WPAI:GH)
The WPAI:GH measures the impact of health problems on work productivity and daily activities. It yields four scores: absenteeism, presenteeism, overall work impairment, and activity impairment. Results are expressed as percentages (0-100%), where higher percentages indicate greater impairment.
Within-person Variability of Emotional States (EMA)
Calculated as the standard deviation or Mean Successive Difference (MSSD) of the daily emotional VAS scores (0-100) collected via EMA over the 8-week period. This measures the "fluctuations" of the patient's emotional state.
Difference in Burden of Disease between LTP and ODT groups
Comparison of the mean Burden of Disease (BoD) scores across all dimensions (physical, emotional, socio-occupational) between patients receiving Long-Term Prophylaxis (LTP) and those receiving On-Demand Treatment (ODT).
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