Long-term prophylaxis reduces attacks, improves QoL in Chinese patients with hereditary angioedema

In a prospective study by the University of Hong Kong, long-term prophylaxis (LTP) with garadacimab or lanadelumab reduces attacks and improves quality of life (QoL) in Chinese patients with hereditary angioedema (HAE) who experienced frequent attacks. No adverse events (AEs) were reported.

“This is the first study investigating the use of LTP medications in the Asian population,” wrote the investigators. “Our study demonstrated the efficacy and safety of LTP [with garadacimab or lanadelumab] in Chinese HAE patients, showing significant improvements in both clinical and psychological measures.” [JACI:Global 2023;doi:10.1016/j.jacig.2023.100166]

Guidelines of the World Allergy Organization and European Academy of Allergy and Clinical Immunology recommend LTP with regular use of medication, such as lanadelumab, for select patients to reduce disease burden by preventing HAE attacks. [Allergy 2022;77:1961-1990]

“However, there has been a lack of clinical studies assessing LTP for HAE in Asian populations with frequent HAE attacks,” the investigators pointed out. “To evaluate the efficacy and safety of HAE-specific LTP medications, garadacimab and lanadelumab, we conducted a study in Chinese adults with ≥3 episodes of HAE attacks within 3 months between July 2021 and July 2022.” [JACI:Global 2023;doi:10.1016/j.jacig.2023.100166]

During the study period, 11 of 36 HAE patients (30.5 percent) experienced frequent attacks (2.5 attacks/month). In these patients (mean age, 42.5 years; female, 54.4 percent; type I HAE, 81.8 percent), eight (72.7 percent) participated in a phase III trial and received subcutaneous (SC) garadacimab (400 mg loading dose, followed by 200 mg Q4W), while three (27.3 percent) accessed SC lanadelumab (300 mg Q2W) via a compassionate use programme. Clinical data and questionnaire surveys were obtained before and after 6 months of LTP treatment.

“Our results showed that LTP [with garadacimab or lanadelumab] significantly reduced the time-normalized number of HAE attacks to 0.1 attacks/month at 6 months vs 2.5 attacks/month at baseline [difference, -2.4 attacks/month; 95 percent confidence interval, -3.3 to -1.5; p<0.01]. During the 6-month treatment, 72.7 percent of patients [n=8] were free from HAE attacks,” the researchers reported. “Among those who still experienced HAE attacks [while on garadacimab or lanadelumab], the duration of each attack was significantly reduced by 38.4 hours vs baseline [22.6 hours vs 61.0 hours; p<0.01]. None of the patients required hospitalization during the 6-month treatment.”

With no AEs reported for LTP with garadacimab or lanadelumab, patients in the study experienced better treatment satisfaction and QoL vs baseline. After 6 months, significant improvements were found in patient-reported overall treatment satisfaction (score after vs before LTP, 92.4 vs 64.4), treatment effectiveness (score, 97.0 vs 65.2) and treatment convenience (score, 85.9 vs 62.1) (p<0.01 for each). The patients also reported significant improvements across total and domain scores in Angioedema QoL assessment (p<0.01 for each), with significantly reduced activity impairment (p=0.01) as assessed by Work Productivity and Activity Impairment Questionnaire: General Health. Scores on the Hospital Anxiety and Depression Scale remained within normal ranges before and after LTP use.

“In line with international recommendations, our initial findings support the urgent need for LTP medications among HAE patients with frequent attacks in Asian countries and beyond,” the investigators concluded. “Further multicentre studies are warranted to confirm the generalizability [ie, the effectiveness and safety of LTP with garadacimab and lanadelumab] in other Asian populations.”