Abstract
INTRODUCTION: Real-world data are needed to improve our understanding of hereditary angioedema (HAE) management in different geographic regions. Here, we report outcomes of patients from Puerto Rico who received lanadelumab for HAE prophylaxis in the noninterventional EMPOWER Study (NCT03845400). METHODS: Eligible patients with HAE due to C1-inhibitor deficiency who initiated lanadelumab treatment were classified as newly treated or established on lanadelumab based on number of lanadelumab doses received before enrollment (<4 or ≥4, respectively). Lanadelumab exposure data were captured using electronic case report forms. Effectiveness was assessed by change in HAE attack rate before vs. after lanadelumab initiation. Safety was assessed through treatment-emergent adverse events (TEAEs). RESULTS: The Puerto Rican site enrolled 9 patients (1 newly treated who was lanadelumab-naïve, 8 established on lanadelumab). All patients were of Hispanic/Latino ethnicity (median [range] age: 55.0 [36-73] years; female: 77.8%). Eight out of 9 patients received lanadelumab 300 mg every 2 weeks; 1 patient established on lanadelumab received dosing every 4 weeks. Average lanadelumab exposure ranged from approximately 2.3 years to >2.5 years for patients newly treated and established on lanadelumab, respectively. In the newly treated patient, the physician-reported HAE attack rate decreased by 76% (1.99 vs. 0.47 attacks/month pre- vs. post-lanadelumab initiation); most physician-reported attacks were mild (16.7%) or moderate (58.3%). In patients established on lanadelumab, the mean (95% confidence interval) physician-reported HAE attack rate was 0.26 (0.09-0.42) attacks/month; most physician-reported attacks were mild (25.7%) or moderate (56.8%). Real-world data on attack incidence reinforced the variability in presentation among patients; 2 were attack-free while on lanadelumab treatment. A total of 5 TEAEs were reported in 4 patients, none of which were serious or fatal. One patient experienced mild fatigue that was considered lanadelumab related. CONCLUSION: Findings from this analysis support the clinical benefits and safety of lanadelumab treatment for HAE prophylaxis in patients from Puerto Rico.