Three-year real-world outcomes of lanadelumab prophylaxis in hereditary angioedema: Complete disease suppression and psychosocial benefits in two East Asian patients

Hereditary angioedema (HAE) is a rare, potentially life-threatening disorder characterized by recurrent, disabling episodes of subcutaneous or submucosal swelling. Lanadelumab, a monoclonal antibody targeting plasma kallikrein, is approved for long-term prophylaxis and has shown high efficacy in clinical trials. However, real-world data on its prolonged use, particularly from East Asia, remain scarce. This report evaluates 3-year clinical and patient-reported outcomes of lanadelumab prophylaxis in two Japanese patients with HAE type I. Both male patients (in their 30s and 70s) received subcutaneous lanadelumab, 300 mg, every 2 weeks, later extended to every 4 weeks following disease stabilization. Clinical efficacy was assessed by attack frequency. Patient-reported outcomes (PROs) included the Angioedema Control Test (AECT), Angioedema Quality of Life Questionnaire (AE-QoL), Hospital Anxiety and Depression Scale (HADS), and Treatment Satisfaction Questionnaire for Medication (TSQM-9). Safety and tolerability were also monitored. Both patients achieved complete or near-complete elimination of HAE attacks during the 156- week follow-up. AECT scores reached the maximum of 16 by week 12 and remained stable. AE-QoL scores improved by approximately 30 points, reflecting sustained quality-of-life benefits. HADS-Anxiety scores declined into the normal range, indicating reduced anticipatory anxiety. TSQM-9 global satisfaction remained above 90 out of 100, and no serious adverse events occurred; one patient experienced mild transient injection-site swelling. This case series presents the longest real-world follow-up of lanadelumab in East Asia. Findings confirm its sustained efficacy, safety, and psychosocial benefits, including enhanced quality of life and emotional recovery. These findings suggest that lanadelumab may play an important role in the long-term management of HAE in Asian clinical settings.

Authors

Institutions

Publication Details

Journal
Intractable & Rare Diseases Research
Published
2025-08-08
DOI
https://doi.org/10.5582/irdr.2025.01045
Primary Topic
Coagulation, Bradykinin, Polyphosphates, and Angioedema
Type
article
Field-Weighted Citation Impact
0.00

Funders

Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

Three-year real-world outcomes of lanadelumab prophylaxis in hereditary angioedema: Complete disease suppression and psychosocial benefits in two East Asian patients

S. Takamura, Tomoo Fukuda
Intractable & Rare Diseases Research
Coagulation, Bradykinin, Polyphosphates, and Angioedema
article

Three-year real-world outcomes of lanadelumab prophylaxis in hereditary angioedema: Complete disease suppression and psychosocial benefits in two East Asian patients

S. Takamura, Tomoo Fukuda
article en

Abstract

Hereditary angioedema (HAE) is a rare, potentially life-threatening disorder characterized by recurrent, disabling episodes of subcutaneous or submucosal swelling. Lanadelumab, a monoclonal antibody targeting plasma kallikrein, is approved for long-term prophylaxis and has shown high efficacy in clinical trials. However, real-world data on its prolonged use, particularly from East Asia, remain scarce. This report evaluates 3-year clinical and patient-reported outcomes of lanadelumab prophylaxis in two Japanese patients with HAE type I. Both male patients (in their 30s and 70s) received subcutaneous lanadelumab, 300 mg, every 2 weeks, later extended to every 4 weeks following disease stabilization. Clinical efficacy was assessed by attack frequency. Patient-reported outcomes (PROs) included the Angioedema Control Test (AECT), Angioedema Quality of Life Questionnaire (AE-QoL), Hospital Anxiety and Depression Scale (HADS), and Treatment Satisfaction Questionnaire for Medication (TSQM-9). Safety and tolerability were also monitored. Both patients achieved complete or near-complete elimination of HAE attacks during the 156- week follow-up. AECT scores reached the maximum of 16 by week 12 and remained stable. AE-QoL scores improved by approximately 30 points, reflecting sustained quality-of-life benefits. HADS-Anxiety scores declined into the normal range, indicating reduced anticipatory anxiety. TSQM-9 global satisfaction remained above 90 out of 100, and no serious adverse events occurred; one patient experienced mild transient injection-site swelling. This case series presents the longest real-world follow-up of lanadelumab in East Asia. Findings confirm its sustained efficacy, safety, and psychosocial benefits, including enhanced quality of life and emotional recovery. These findings suggest that lanadelumab may play an important role in the long-term management of HAE in Asian clinical settings.

Intractable & Rare Diseases ResearchVol. 14(3)
Social Insurance Saitama Chuo Hospital (JP), Saitama Medical University (JP)
Eli Lilly and Company, Sanofi, CSL Behring, Taiho Pharmaceutical, Torii Pharmaceutical, Kyowa Hakko Kirin, LEO Pharma, Novartis Pharma
Openalex Percentile: Top 72%
Coagulation, Bradykinin, Polyphosphates, and Angioedema
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.