D2.334 - Real-World Effectiveness of Lanadelumab in Hereditary Angioedema: A 12-Month Retrospective Study at a Single Center in Colombia

Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent episodes of subcutaneous and submucosal edema. Lanadelumab is a first-line option for long-term prophylaxis (LTP) in patients with HAE type I and II. This study aims to evaluate the clinical effectiveness of Lanadelumab in real-world conditions in patients with HAE type I treated at a single center in Colombia over a 12-mo period.

An observational and retrospective study was conducted by reviewing clinical records of patients with confirmed HAE type I diagnosis who were treated with Lanadelumab for at least one yr. Demographic data, clinical characteristics, number of attacks, use of rescue medication, and disease control scores were collected using the Angioedema Control Test (AECT) and the Angioedema Quality of Life (AE-QoL) questionnaire. Data analyses were performed at 6 and 12 mo of treatment. Statistical analysis was performed using Python with support from ChatGPT, employing the Wilcoxon test to compare clinical scores before and after treatment.

Data from 18 patients were analyzed; 72% were female. The mean age was 35.6 yrs (range: 13–60). Before starting Lanadelumab, patients reported a mean of 21.2 attacks/yr (SD: 10.2). After 6 mo of treatment, the mean number of attacks decreased to 1.1 (SD: 1.3), and after 12 mo, it was 1.6 (SD: 1.8). The mean AECT score increased from 4.0 before treatment to 13.6 at 6 mo and 14.3 at 12 mo, indicating sustained disease control. The AE-QoL score improved from a mean of 66.6 to 16.7 at 6 mo. The use of icatibant decreased from a mean of 6 doses in the 3 mo before starting Lanadelumab (SD: 2.7) to 0.3 doses in the 3 mo after 6 mo of treatment. Statistical analysis showed a significant reduction in the number of attacks (p < 0.01) and improvements in AECT and AE-QoL scores (p < 0.05). Eight patients experienced administrative delays in the timely supply of the drug, extending the administration interval to more than 30 days, with some reporting mild to moderate attacks.

The results of this study demonstrate that Lanadelumab treatment in real-world conditions reduces the frequency of attacks, the use of rescue medications, and improves disease control and quality of life in patients with HAE type I. Despite delays in drug supply, medium-term disease control appears to remain unaffected. These findings support the use of Lanadelumab as an effective and safe option for long-term prophylaxis in this population