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In hereditary angioedema (HAE), deficient levels or function of the C1 inhibitor protein cause swelling of the extremities, gut mucosa, and upper airways (NEJM JW Gen Med Mar 15 2017 and N Engl J Med 2017; 376:717). In addition to on-demand treatment for acute events, patients with frequent or life-threatening HAE episodes should be offered preventive therapy. Twice-weekly C1 inhibitor infusions are effective but currently must be given intravenously, and some patients have breakthrough swelling.
In an industry-funded, phase III crossover trial, 79 patients with frequent HAE attacks (i.e., ≥4 during 2 months) received one of two doses of a subcutaneous human plasma–derived C1 inhibitor (not yet FDA approved) or placebo twice weekly for 16 we…