D3.421 - Real-world Effectiveness and Safety of Lanadelumab Treatment in Patients With Hereditary Angioedema: The 12-Month Interim Analysis of a Polish, Prospective, Multicenter, Observational Study (CHOPIN)

To evaluate real-world outcomes of lanadelumab in hereditary angioedema (HAE) due to C1 inhibitor (C1INH) deficiency, the CHOPIN study assessed its effectiveness and safety in Polish patients (pts) with HAE enrolled in the National Drug Program.

CHOPIN (NCT05147181) is a prospective, observational study conducted across 13 Polish sites. Eligible pts (≥12 years) had HAE-C1INH-Type1 or -Type2 and ≥12 attacks in the 6 months before lanadelumab prophylactic treatment initiation. This post hoc interim analysis (Mar 2022–Dec 2023) in enrolled pts (>18 years) assessed changes in HAE attack frequency and severity from baseline (3 months pre-treatment) to Month 12 (M12). Work Productivity and Activity Impairment Questionnaire: General Health (WPAI:GH), Angioedema Quality of Life (AE-QoL), and Angioedema Control Test (AECT) scores and adverse events (AEs) were also evaluated. 

Of 48 adults enrolled (median age 36 years, 77.1% female), 45 (93.8%) completed the12-month interim period (treatment discontinuation due to pregnancy: n=2 and loss of contact: n=1). At baseline, 23 (47.9%) pts had lower, and 25 (52.1%) equal to or higher than the median number of HAE attacks (12.0; cohorts L and H, respectively). Overall, lanadelumab reduced HAE attack frequency by 97.9%, lowering the incidence rate (IR) from 3.41 attacks/pt/4 weeks at baseline (74% severe) to 0.07 at M12 (60% mild or moderate). In cohort L, HAE attack frequency decreased by 96.4% from an IR of 1.99 to 0.07 attacks/pt/4 weeks and in cohort H, by 98.5% from an IR of 4.71 to 0.07 attacks/pt/4 weeks, from baseline to M12, respectively. Mean WPAI:GH scores reduced from baseline to M12: absenteeism, 9.4 to 0.0 (p=0.058); presenteeism, 57.9 to 1.3 (p<0.001); overall work productivity loss, 62.0 to 1.3 (p<0.001); and activity impairment, 60.2 to 2.0 (p<0.001). The baseline mean AE-QoL and AECT total scores improved from 66.7 to 10.9 and from 3.8 to 15.2, respectively at M12 (both p<0.001). These improvements were consistent across both cohorts. Seven lanadelumab-related AEs were reported, including 3 pts with injection site reactions.  

Lanadelumab treatment reduced the incidence and severity of HAE attacks, improved patient-reported outcomes, and was well tolerated in Polish pts with HAE. These benefits were consistent across cohorts, regardless of baseline disease severity.