Patient Driven Automated Data Collection System Demonstrates Effectiveness of C1INH Rescue Therapy

RATIONALE: Attacks of hereditary angioedema (HAE) cause significant morbidity and can be disruptive to patients' daily life functions for several days. Early attack recognition by patients and clinicians is critical to successful disease management. C1-esterase inhibitor (C1-INH) replacement therapy effectively treats symptoms and shortens attack duration.METHODS: A computer/voice-response patient survey is being conducted in parallel with an open-label extension of a phase 3 trial of plasma-derived C1-INH (Berinert, CSL Behring, King of Prussia, PA) in acute HAE attacks to assess attack frequency and treatment, and patients' views of therapy. Responses were collected via home computer or telephone voice response system.RESULTS: Twenty-seven patients (mean 35.1 years) from 7 sites have participated in the survey. Patients reported a yearly average 3.7 ER visits and 4.2 hospital days before study participation. Over a 16 month period, 273 attacks have been reported: median 3.0/patient (range 1 to 70). Approximately 68% of attacks were treated with C1-INH, 98.7% of which were reported by patients as responding satisfactorily. Average time from dosing to symptom relief onset was 1.3 hours and average time to resolution 1 day, 9 hrs. Without the availability of C1-INH, 33% of patients would have sought emergency treatment, 47% would have deferred medical therapy and 7% would have self-treated with analgesics.CONCLUSIONS: A patient-driven reporting system demonstrates that patients with HAE have frequent attacks and that without access to C1INH many attacks would go untreated or require emergency care. Early recognition and treatment resolves most attacks and decreases attack length. RATIONALE: Attacks of hereditary angioedema (HAE) cause significant morbidity and can be disruptive to patients' daily life functions for several days. Early attack recognition by patients and clinicians is critical to successful disease management. C1-esterase inhibitor (C1-INH) replacement therapy effectively treats symptoms and shortens attack duration. METHODS: A computer/voice-response patient survey is being conducted in parallel with an open-label extension of a phase 3 trial of plasma-derived C1-INH (Berinert, CSL Behring, King of Prussia, PA) in acute HAE attacks to assess attack frequency and treatment, and patients' views of therapy. Responses were collected via home computer or telephone voice response system. RESULTS: Twenty-seven patients (mean 35.1 years) from 7 sites have participated in the survey. Patients reported a yearly average 3.7 ER visits and 4.2 hospital days before study participation. Over a 16 month period, 273 attacks have been reported: median 3.0/patient (range 1 to 70). Approximately 68% of attacks were treated with C1-INH, 98.7% of which were reported by patients as responding satisfactorily. Average time from dosing to symptom relief onset was 1.3 hours and average time to resolution 1 day, 9 hrs. Without the availability of C1-INH, 33% of patients would have sought emergency treatment, 47% would have deferred medical therapy and 7% would have self-treated with analgesics. CONCLUSIONS: A patient-driven reporting system demonstrates that patients with HAE have frequent attacks and that without access to C1INH many attacks would go untreated or require emergency care. Early recognition and treatment resolves most attacks and decreases attack length.