One-year real-life outcomes of lanadelumab therapy in Romanian patients with hereditary angioedema due to C1-inhibitor deficiency.

Introduction: In the majority of patients with hereditary angioedema (HAE) due to C1-inhibitor deficiency (HAE-C1INH), effective long-term prophylactic (LTP) treatment can achieve complete disease control. Lanadelumab is one of the first-line option recommended for this purpose. Our study aimed to evaluate changes in disease control, quality of life, and attack frequency among Romanian HAE-C1INH patients, during the first year of treatment with lanadelumab.

Methods: This non-interventional prospective study included the Romanian HAE-C1INH patients enrolled in the first year of the national lanadelumab treatment program. Angioedema Control Test, (AECT), Angioedema Quality of Life Questionnaire (AE-QoL) and attacks frequency were recorded at baseline and at 3, 6, 9 and 12 months.

Results: Twenty-four patients (14 women [58.3%], 10 men [41.7%]) initiated lanadelumab therapy, with a mean age of 40.7 years. Most had HAE-C1INH type I (22 patients, 91.7%), and one patient was under 18 years of age. Ten patients switched from LTP with intravenous plasma-derived C1-INH, while 14 were previously managed with on-demand therapy only. Baseline scores were: AECT 4.5 [interquartile range (IQR) 2.0], AE-QoL 66.1 [standard deviation (SD) 18.3], and a mean attack frequency of 10.0 (IQR 4.0) (over the preceding three months). Improvements were observed at each follow-up point, with respective scores at 3, 6, 9 and 12 months as follows: three months: AECT 12.0 (IQR 5.8) / AE-QoL 35.3 (SD 23.2)/ attacks 3.4 (IQR 5.0); six months: AECT 12.3 (IQR 5.3) / AE-QoL 35.4 (SD 25.4)/ attacks 2.8 (IQR 4.8); nine months: AECT 12.6 (IQR 5.8) / AE-QoL 34.1 (SD 23.2)/ attacks 2.2 (IQR 3.8) and 12 months: AECT 12.9 (IQR 5.5) / AE-QoL 32.1 (SD 21.6) / attacks 1.4 (IQR 2.0). Seven patients became symptom-free after the first dose, and four more achieved this status within the first three months.

Discussion: LTP with lanadelumab provided effective disease control and significantly improved quality of life in patients with HAE-C1INH over the course of one year. Regular evaluations at relatively short intervals by the availability and ease of administration of validated questionnaires serve as a useful tool for clinicians in the comprehensive assessment of HAE patients and offer a valuable means of monitoring treatment effectiveness.