Indirect Comparison of Lanadelumab and Intravenous C1-INH Using Data from the HELP and CHANGE Studies: Bayesian and Frequentist Analyses

• DOI: 10.1007/s40268-021-00337-4
• Publication Date: 2021-03-01T14:04:13Z
• Authors: Joan Mendivil; Mia Malmenäs; Katrin Haeussler; Matthias Hunger; Gagan Jain; Giovanna Devercelli
• Keywords: Hereditary Angioedema

Hereditary angioedema (HAE) with C1-esterase inhibitor (C1-INH) deficiency is a rare disease associated painful, potentially fatal swelling episodes affecting subcutaneous or submucosal tissues. HAE attacks recur unpredictable severity and frequency throughout patients’ lives; long-term prophylaxis essential for some patients. In the absence of head-to-head studies, indirect treatment comparison (ITC) prophylactic agents valid approach to evaluate comparative efficacy. We conducted an ITC using data from placebo-controlled HELP study (assessing patients receiving lanadelumab 300 mg every 2 4 weeks) 12-week, parallel arm, crossover CHANGE intravenous C1-INH). Outcomes interest were attack rate ratio (ARR) time after day 0 (TTA0) 70 (TTA70). Two methodologies used: Bayesian results update non-informative prior distributions posterior on relative effects, frequentist patient-level generate Poisson regressions (for ARR) Cox models TTA0 TT70). Both analyses suggested that reduced by 46–73% versus C1-INH. Relative C1-INH, risk first was comparable between C1-INH both doses; 81–83% weeks, compared Findings these two support favorable efficacy in reducing extending attack-free intervals HAE.