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Background: Hereditary angioedema (HAE), caused by absolute or functional deficiency of C1-esterase inhibitor (C1-INH), is characterized by recurrent, disfiguring, and potentially life-threatening attacks of subcutaneous and submucosal edema. The mean… Click to show full abstract
Background: Hereditary angioedema (HAE), caused by absolute or functional deficiency of C1-esterase inhibitor (C1-INH), is characterized by recurrent, disfiguring, and potentially life-threatening attacks of subcutaneous and submucosal edema. The mean age of first attack is 10 years and earlier onset may be predictive of a more severe disease course. HAE treatment guidelines recommend on-demand (acute) therapy as standard of care for all HAE patients and prophylaxis for those with frequent and/or more disabling attacks, as well as those with inadequate response to on-demand therapy. This phase 3 trial compared twice-weekly prophylaxis …
Journal Title: Pediatrics
Year Published: 2018
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