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Managing Hereditary Angioedema in a Three-Generation Family: Danazol's Promise in Resource-Limited Settings.
Cureus
November 2024
Department of Pediatrics, Mahatma Gandhi Mission (MGM) Medical College and Hospital, Aurangabad, IND.
Background Hereditary angioedema (HAE) is a rare disorder in India, and while prevalence data is limited, it is believed that a significant number of individuals may be affected. Due to restricted access to first-line treatments, older therapies like danazol are commonly used despite associated risks in resource-constrained settings. This study aimed to assess the efficacy of danazol as an affordable long-term prophylaxis (LTP) for HAE in a three-generation family.
View Article and Find Full Text PDFPreimplantation genetic testing for hereditary angioedema as a potential dominant cost-effective intervention.
J Allergy Clin Immunol Pract
December 2024
Department of Microbiology, Immunology and Transplantation, Allergy and Clinical Immunology Research Group, KU Leuven, Leuven, Belgium. Electronic address:
Interplay between on-demand treatment trials for hereditary angioedema and treatment guidelines.
J Allergy Clin Immunol
December 2024
University of Cincinnati, College of Medicine, Cincinnati, Ohio, USA. Electronic address:
Over the past two decades, guidelines for the on-demand treatment of hereditary angioedema (HAE) attacks have undergone significant evolution. Early treatment guidelines, such as the Canadian 2003 International Consensus Algorithm, often gated on-demand treatment by attack location and/or severity. Pivotal trials for on-demand injectable treatments (plasma-derived C1 esterase inhibitor [C1INH], icatibant, ecallantide [US only], recombinant C1INH), which were approved in the US and EU between 2008-2014, were designed accordingly.
View Article and Find Full Text PDFPhenotypic and molecular characterization of the largest worldwide cluster of hereditary angioedema type 1.
PLoS One
December 2024
School of Medicine and Health Sciences, Center for Research in Genetics and Genomics (CIGGUR), Institute of Translational Medicine (IMT), Universidad del Rosario, Bogotá D.C., Colombia.
Hereditary angioedema type 1 (HAE1) is a rare, genetically heterogeneous, and autosomal dominant disease. It is a highly variable, insidious, and potentially life-threatening condition, characterized by sudden local, often asymmetric, and episodic subcutaneous and submucosal swelling, caused by pathogenic molecular variants in the SERPING1 gene, which codes for C1-Inhibitor protein. This study performed the phenotypic and molecular characterization of a HAE1 cluster that includes the largest number of affected worldwide.
View Article and Find Full Text PDFReal-world Effectiveness of Lanadelumab in Hereditary Angioedema: Multi-country INTEGRATED Observational Study.
J Allergy Clin Immunol Pract
December 2024
EUCAN Medical Affairs, Takeda Pharmaceuticals International AG, Glattpark-Opfikon (Zürich), Switzerland.
Background: Hereditary angioedema (HAE) is a rare genetic disease characterized by recurrent episodes of cutaneous or subcutaneous edema. There is clinical need for treatments that reduce the rate of HAE attacks in patients.
Objectives: Primary objectives were to evaluate the effectiveness of lanadelumab on attack free rate (AFR; proportion of patients who had zero HAE attacks), and on every two weeks (Q2W) and every four weeks (Q4W) adjustments on AFR.
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