Idiopathic non-mast cell angioedema: Treatment insights from global experts
Background:
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.
Objective:
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.
Methods:
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.
Results:
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.
Conclusion:
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.
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.
Objective:
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.
Methods:
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.
Results:
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.
Conclusion:
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.
Keywords: Angioedema; HAE-nl-C1INH; INMA; bradykinin; long term prophylaxis; on-demand treatment; treatment
Document Type: Research Article
Affiliations: From the Division of Allergy and Immunology, Department of Medicine, University of California San Diego, La Jolla, California and
Publication date: January 1, 2025
- Allergy and Asthma Proceedings is a peer reviewed publication dedicated to distributing timely scientific research regarding advancements in the knowledge and practice of allergy, asthma and immunology. Its primary readership consists of allergists and pulmonologists.
The goal of the Proceedings is to publish articles with a predominantly clinical focus which directly impact quality of care for patients with allergic disease and asthma and by having the potential to directly impact the quality of patient care. AAP welcomes the submission of original works including peer-reviewed original research and clinical trial results. Additionally, as the official journal of the Eastern Allergy Conference (EAC), AAP will publish content from EAC poster sessions as well as review articles derived from EAC lectures.
Featured topics include asthma, rhinitis, sinusitis, food allergies, allergic skin diseases, diagnostic techniques, allergens, and treatment modalities. Published material includes peer-reviewed original research, clinical trials and review articles.
Articles marked "F" offer free full text for personal noncommercial use only.
The journal is indexed in Thomson Reuters Web of Science and Science Citation Index Expanded, plus the National Library of Medicine's PubMed service. - Editorial Board
- Information for Authors
- Submit a Paper
- Information for Advertisers
- Reprint Requests
- Commercial level: Permission to use content
- www.JFoodAllergy.com
- Ingenta Connect is not responsible for the content or availability of external websites
Receive new issue alert- Access Key
- Free content
- Partial Free content
- New content
- Open access content
- Partial Open access content
- Subscribed content
- Partial Subscribed content
- Free trial content