Background: Hereditary angioedema (HAE) is an autosomal dominant inherited disease in which patients suffer from local attacks primarily affecting skin and gastrointestinal tract, and sometimes even the upper respiratory tract leading to asphyxiation. Since head-to-head trials between authorized treatments are lacking, this study compares efficacy and safety of lanadelumab and intravenous plasma-derived C1-esterase inhibitor (pdC1-INH i.v.) in HAE patients on long-term prophylaxis by means of an indirect treatment comparison.
Methods: Efficacy and safety of lanadelumab against pdC1-INH i.v. were analyzed in a fully prespecified indirect comparison based on individual patient data (n = 231) from the HELP and CHANGE clinical trials. Primary and secondary efficacy endpoints were compared using a generalized linear model for count data. Confounding variables were identified a priori via systematic literature research and validated by clinical experts. Adjustment of confounders was implemented using a conditional regression model.
Results: Lanadelumab showed a statistically significant improvement in reduction of HAE attack rates compared to pdC1-INH i.v. across multiple endpoints: Monthly attack rate of patients treated with lanadelumab was less than half compared to pdC1-INH i.v. (Rate ratio: 0.486; 95% CI: 0.253, 0.932). Monthly rate of laryngeal attacks was found to be five times lower for lanadelumab (Rate ratio: 0.2; 95% CI: 0.044, 0.915) and monthly rate of acute treated HAE attacks among lanadelumab patients was about one third of the attack rate of pdC1-INH i.v. patients (Rate ratio: 0.366; 95% CI: 0.185, 0.727).
Conclusion: This study contributes to current knowledge in the treatment of HAE by indicating a statistically significant reduction of HAE attacks under lanadelumab compared to pdC1-INH i.v.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1111/all.15861 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Real-world outcomes of patients with hereditary angioedema with normal C1-inhibitor function and patients with idiopathic angioedema of unknown etiology in Canada.
Allergy Asthma Clin Immunol
September 2024
Unity Health Toronto, St Michael's Hospital, University of Toronto, Toronto, ON, Canada.
Article Synopsis
- The study examines Hereditary angioedema with normal C1-inhibitor function (HAE nC1-INH) and idiopathic angioedema of unknown etiology (AE-UNK) in Canada by analyzing electronic health records from 2012 to 2022.
- Out of 60 patients analyzed, the average age of symptom onset was 21.5 for HAE nC1-INH and 23 for AE-UNK, with significant differences in triggers for episodes and treatment received post-diagnosis.
- The results highlight that while most patients received long-term prophylaxis after diagnosis, the types of treatments and the prevalence of angioedema attacks varied between the two conditions, indicating distinct
Background: Hereditary angioedema (HAE) is an autosomal dominant inherited disease in which patients suffer from local attacks primarily affecting skin and gastrointestinal tract, and sometimes even the upper respiratory tract leading to asphyxiation. Since head-to-head trials between authorized treatments are lacking, this study compares efficacy and safety of lanadelumab and intravenous plasma-derived C1-esterase inhibitor (pdC1-INH i.v.
View Article and Find Full Text PDFTreatment of hereditary angioedema-single or multiple pathways to the rescue.
Front Allergy
September 2022
Department of Immunology, Auckland District Health Board, and Department of Medicine, University of Auckland, Auckland, New Zealand.
Article Synopsis
- Hereditary angioedema (HAE) is a rare genetic disorder caused by mutations leading to low or dysfunctional C1 esterase inhibitor (C1-INH), affecting several bodily pathways important for inflammation and clotting.
- Treatment options for HAE focus on replacing C1-INH or targeting bradykinin generation through medications like C1-INH replacement therapy, B2 receptor antagonists (like icatibant), or plasma kallikrein inhibitors (like ecallantide), which help manage and prevent attacks.
- Clinical studies show that both intravenous C1-INH products and pathway-specific therapies reduce the frequency of HAE attacks, but some patients may still experience breakthrough attacks, necessitating careful management and
Real-Life Experience With Subcutaneous Plasma-Derived C1-Inhibitor for Long-Term Prophylaxis in Patients With Hereditary Angioedema: A Case Series.
Front Allergy
April 2022
UOC Medicina Generale, ASST Fatebenefratelli Sacco, Ospedale Luigi Sacco-Università degli Studi di Milano, Milan, Italy.
Hereditary angioedema due to C1-inhibitor deficiency (C1-INH-HAE) is characterized by swelling attacks that may be even life-threatening. To reduce the frequency of attacks, some patients need a long-term prophylaxis (LTP). In addition to the intravenous administration, plasma-derived C1-inhibitor (pdC1-INH) has been proved effective also if administered subcutaneously at the dose of 120 IU/kg/week.
View Article and Find Full Text PDFModeling Cost-Effectiveness of On-Demand Treatment for Hereditary Angioedema Attacks.
J Manag Care Spec Pharm
February 2020
Applied Health Care Research Management (AHRM), Raleigh, North Carolina.
Background: Hereditary angioedema (HAE) is a rare C1-inhibitor (C1-INH) deficiency disease. Low levels of functional C1-INH can lead to recurrent attacks of severe swelling occurring in areas such as the limbs, face, gastrointestinal tract, and throat. These attacks are both painful and disabling and, if not treated promptly and effectively, can result in hospitalization or death.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!