Idiopathic non-mast cell angioedema (INMA) is a rare disease typified by recurrent attacks of cutaneous and subcutaneous swelling. Every attack carries the potential for severe morbidity and, in the case of laryngeal involvement, mortality. Whereas therapies approved for hereditary angioedema (HAE) have been used in the care of patients with INMA, little is known with regard to their efficacy for the treatment of this disease. The objective was to gather evidence from global experts, ranking their assessment of on-demand therapy (ODT) and long-term prophylactic (LTP) treatment efficacy for INMA. A survey was developed and distributed to international experts invited to attend a 2023 symposium. INMA was diagnosed by standardized criteria. Linkert scales were used to rate the efficacy for ODT and LTP therapy. Enrollment was closed after 1 month and the data were analyzed. Surveys were distributed to 31 experts from 16 countries with a 77% response rate (n = 24) reporting on 300 patients with INMA. Efficacy rankings of ODT were the following: icatibant (14 experts with 93 treated patients), 46.2% high and 38.7% moderate; and plasma-derived C1 inhibitor (C1INH) (13 experts with 31 treated patients), 32.3% moderate and 45.2% mild. Efficacy rankings of LTP were the following: antifibrinolytics (11 experts with 52 treated patients), 23.1% high and 38.5% moderate; lanadelumab (5 experts with 19 treated patients), 21% high and 79% moderate; and subcutaneous C1INH (3 experts with 19 treated patients), 21.1% moderate and 79.0% mild. LTP efficacy was also recorded for berotralstat and progestin. Icatibant (ODT) and either antifibrinolytics or lanadelumab (LTP) were ranked as the most efficacious treatments for the patients with INMA (among medications with at least five treated patients) by the expert physicians. Progestins, berotralstat, and plasma derived C1INH each demonstrated a favorable prophylactic effect; however, broader experience will be required to formulate overall recommendations.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.2500/aap.2025.46.240091 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Using Virtual Reality to Promote Self-Identity Reconstruction as the Main Focus of Therapy.
J Clin Psychol
January 2025
Department of Clinical Psychology and Psychobiology, The Institute of Neurosciences, Universitat de Barcelona, Barcelona, Spain.
Based on the repertory grid technique, we developed Explore Your Meanings (EYME), a digital platform that helps patients explore identity values and internal conflicts using virtual reality (VR). EYME was part of a research project treating depression in young adults, including 10 weekly, 1-h sessions aimed at changing personal constructs-cognitive schemas that shape how individuals interpret reality. We present the case of Mary, a 21-year-old woman diagnosed with persistent major depressive disorder and social phobia.
View Article and Find Full Text PDFLow-Level Red Light for the Progression Myopia in Children: A Meta-Analysis.
Semin Ophthalmol
January 2025
Department of Ophthalmology, The Affiliated Hospital of Qingdao University, Qingdao, China.
Purposes: This meta-analysis aims to systematically analyze the efficacy of low-level red light (LRL) therapy for myopia control and prevention in children.
Methods: All the data were searched from the PubMed, EMBASE, and the Cochrane Library. The Cochrane Handbook was used to evaluate the quality of the included studies.
Adverse Events of Factor Xa Inhibitors in Pediatric Patients: A Meta-analysis and Pharmacovigilance Study.
Paediatr Drugs
January 2025
Institute of Clinical Pharmacology, Peking University First Hospital, Beijing, China.
Background: This study aimed to provide a comprehensive review of adverse events (AEs) associated with factor Xa (FXa) inhibitors in pediatric patients.
Methods: We searched PubMed, Embase, Cochrane Library, ClinicalTrials.gov, and the European Union Clinical Trials Register for English-language records from the establishment of the database up to October 17, 2023.
The Effect of Colchicine on Platelet Function Profiles in Patients with Stable Coronary Artery Disease: The ECLIPSE Pilot Study.
Cardiol Ther
January 2025
Adult Medicine, Department of Clinical Medical Sciences, Faculty of Medical Sciences, The University of the West Indies, St. Augustine, Trinidad and Tobago.
Introduction: This prospective, single-arm pharmacodynamic study assessed the effect of colchicine (COLC) [Strides Pharma UK Ltd, Watford, Hertfordshire, England] 0.5 mg administered orally once daily for 14 days on platelet reactivity with respect to aspirin reaction units (ARUs) and P2Y reaction units (PRUs).
Methods: Twenty-two patients with stable coronary artery disease (CAD) on dual antiplatelet therapy (DAPT) with daily maintenance aspirin and clopidogrel were recruited.
The effectiveness of a conservative approach in the management of groove pancreatitis.
Indian J Gastroenterol
January 2025
Department of Gastroenterology, Christian Medical College, Vellore, 632 517, India.
Background: Groove pancreatitis (GP) is a form of pancreatitis that affects the pancreaticoduodenal groove area, which lies between the head of the pancreas, the second part of the duodenum and the distal bile duct, presenting as abdominal pain and gastric outlet obstruction. In this study, we present the clinical and radiological characteristics of individuals diagnosed with groove pancreatitis at our center and discuss the use of a conservative treatment approach in managing GP.
Methods: The data of patients with groove pancreatitis treated at our center between January 2012 and December 2021 was analyzed.
Want 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!