Therapeutics/pharmacology

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Prophylaxis with Recombinant Human c1 Inhibitor (rhc1inh) Is Efficacious and Well Tolerated for Prevention of Hereditary Angioedema (hae) Attacks

Wednesday, June 14
6:15 PM - 7:45 PM

Background: rhC1INH is indicated for the treatment of acute HAE attacks. However, prophylactic replacement therapy to provide functional C1INH levels may deter HAE attacks.ÂÂ

Methods: A phase 2, randomized, double-blind, 3-period, crossover study was conducted in patients aged ≥13 years with functional C1INH levels < 50% of normal and ≥4 HAE attacks during previous 3 months. Patients received rhC1INH 50 IU/kg (max, 4200 IU) once or twice weekly or placebo in three 4-week periods; each treatment was separated by 1 week and symptoms monitored by a daily diary. Primary endpoint (intent-to-treat [ITT] population) was number of HAE attacks per 4-week period. Percentage reduction in number of attacks with rhC1INH from placebo treatment was examined in 10% increments (per-protocol [PP] population).

Results: Thirty-two patients (mean age, 45.9 years [range, 16.9-73.5]) were included in the ITT population (modified ITT, n=31; PP, n=23). Mean number of HAE attacks was reduced from 7.2 (placebo) to 4.4 (rhC1INH once weekly; P=0.0004) and 2.7 (rhC1INH twice weekly; P < 0.0001). The majority who received rhC1INH twice weekly (modified ITT: 74.2% [95% confidence interval (CI), 56.8âˆ'86.3] and PP: 95.7% [95% CI, 79.0âˆ'99.2]) had ≥50% reduction in number of attacks that occurred during active treatment versus during placebo. A greater percentage of patients had 90%-100% reduction in HAE attacks with twice weekly rhC1INH (26.1%) than with once weekly rhC1INH (8.7%). The most common adverse events with rhC1INH were headache and nasopharyngitis.

Conclusions: rhC1INH as prophylactic replacement therapy was efficacious for the prevention of HAE attacks and results support further research.

William H. Yang

Ottawa Allergy Research Corporation & University of Ottawa Medical School

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    H. Henry Li

    Institute for Allergy and Asthma

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      Dumitru Moldovan

      University of Medicine and Pharmacy, Mures County Hospital

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        Vesna Grivcheva Panovska

        PHU Clinic of Dermatology, Medical University Skopje

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          Joseph R. Harper

          Department of Clinical Research and Medical Affairs, Pharming Technologies BV

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            Bruno M. Giannetti

            Department of Clinical Research and Medical Affairs, Pharming Technologies BV

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              Marc Riedl

              Associate Clinical Professor
              University of California, San Diego

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                Prophylaxis with Recombinant Human c1 Inhibitor (rhc1inh) Is Efficacious and Well Tolerated for Prevention of Hereditary Angioedema (hae) Attacks



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