Lanadelumab is an effective prophylactic treatment for hereditary angioedema

Reference

Banerji A, Riedl MA, Bernstein JA, et al, for the HELP Investigators. Effect of lanadelumab compared with placebo on prevention of hereditary angioedema attacks. A randomized clinical trial. JAMA 2018;320(20):2108-2121.

Synopsis

Lanadelumab is a human monoclonal antibody that inhibits the generation of bradykinin, possibly reducing or preventing recurrent attacks of hereditary angioedema. These investigators identified patients, 12 years or older, who met standard diagnostic criteria for hereditary angioedema type I or type II. After a 4-week run-in period, eligible patients (N = 125) who'd had 1 or more confirmed attack during the run-in period randomly received (concealed allocation assignment) subcutaneous lanadelumab at 1 of 3 dose regimens--150 mg every 4 weeks, 300 mg every 4 weeks, or 300 mg every 2 weeks--or matched placebo. Individuals who assessed outcomes remained masked to treatment group assignment. Complete follow-up occurred for 90.4% of participants at 26 weeks. Using intention-to-treat analysis, significantly fewer attacks of hereditary angioedema occurred in all 3 treatment groups than in the placebo group (0.48, 0.53, and 0.26 vs 1.97, respectively). Similarly, a 50% reduction in attack rate from baseline occurred significantly more often in the 3 treatment groups than in the placebo group (89.3%, 100%, and 100% vs 31.7%, respectively; numbers needed to treat = 1.46 - 1.74). Adverse effects were minimal in all treatment groups.