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Open Access Long-term prophylaxis in hereditary angioedema management: Current practices in France and unmet needs

Background:

Hereditary angioedema (HAE) is characterized by unpredictable and potentially life-threatening attacks of cutaneous and submucosal swelling. Over the past decade, new agents, based on a better understanding of the underlying biologic mechanisms of HAE, have changed the face of long-term prophylaxis (LTP).

Objective:

The objective was to describe current practices and unmet needs with regard to LTP for HAE in expert centers in France.

Methods:

The study was conducted in France in 2020. Based on their experience with patients with HAE who had visited their center at least once in the past 3 years, physicians from 25 centers who are expert in the management of HAE were requested to fill in a questionnaire that encapsulated their active patient list, criteria for prescribing LTP, and medications used. They were asked about potential unmet needs with currently available therapies. They were asked to express their expectations with regard to the future of HAE management.

Results:

Analysis was restricted to 20 centers that had an active patient file and agreed to participate. There were 714 patients with C1 inhibitor (C1-INH) deficiency, of whom 423 (59.2%) were treated with LTP. Altered quality of life triggered the decision to start LTP, as did the frequency and severity of attacks. Ongoing LTP included androgens (28.4%), progestins (25.8%), lanadelumab (25.3%), tranexamic acid (14.2%), intravenous C1-INHs (5.6%), and recombinant C1-INH (0.7%). Twenty-nine percent of the patents with LTP were considered to still have unmet needs. Physicians' concerns varied among therapies: poor tolerability for androgens and progestins, a lack of efficacy for tranexamic acid and progestins, dosage form, and high costs for C1-INHs and lanadelumab. Physicians' expectations encompassed more-efficacious and better-tolerated medications, easier treatment administration for the sake of improved quality of life of patients, and less-expensive therapies.

Conclusion:

Despite the recent enrichment of the therapeutic armamentarium for LTP, physicians still expressed unmet needs with currently available therapies.

Keywords: Hereditary angioedema; administration route; decision-making; expert opinion; long-term prophylaxis; quality of life; side effects; survey; treatment; unmet needs

Document Type: Research Article

Affiliations: 1: From the National Reference Center for Angioedema (CREAK), Department of Internal Medicine, Grenoble Alpes University, Laboratoire T-RAIG, UMR 5525 TIMC-IMAG (UGA-CNRS), Grenoble, France; 2: Department of Internal Medicine, Sorbonne University, AP-HP, Saint Antoine Hospital, Paris, France; 3: Department of Internal Medicine, Rouen University Hospital, Rouen, France; 4: Department of Internal Medicine, Hospices Civils de Lyon, Edouard Herriot Hospital, France; 5: Department of Internal Medicine, Niort Hospital Center, France; 6: Department of Internal Medicine, Hautepierre Hospital, Strasbourg University Hospital, France; 7: Department of Internal Medicine, La Cavale Blanche University Hospital, Brest, France; 8: Department of Dermatology, University Montpellier, Montpellier, France; 9: Allergy Diseases Unit, Internal Medicine and Post-Emergency, GH Pellegrin, Bordeaux University Hospital, Bordeaux, France; 10: Transversal Unit of Allergology and Clinical Immunology, Medicine Department, Tours Regional University Hospital, Tours University, France; 11: Department of Internal Medicine and Clinical Immunology, Lorraine University, CHRU-Nancy, Nancy, France; 12: Department of Internal Medicine, Nice University Hospital Center, Côte d'Azur University, Nice, France; 13: Dermatology Department, Le Mans Regional Hospital, Le Mans, France; 14: Department of Medicine, Saint Louis University Hospital, Saint Pierre, Réunion; 15: Department of Internal Medicine, Nantes University Hospital, Nantes, France; 16: Limoges University Hospital, Limoges, France; 17: Department of Immuno-Allergology, Caen University Hospital, Caen, France; 18: Department of Internal Medicine, Besancon University Hospital, Besancon, France; 19: Department of Gynecology Obstetrics and Reproductive Medicine, Cochin University Hospital, Paris, France; 20: Internal Medicine, University Hospital Center Toulouse, Toulouse, France; 21: Department of Internal Medicine, Nîmes University Hospital, Nîmes, France; 22: Carely, Lille, France; 23: Soladis Clinical Studies, Roubaix, France; 24: Department of Internal Medicine, CREAK, ACARE Center of Excellence, Grenoble Alpes University Hospital, France, and 25: Department of Internal Medicine and Clinical Immunology, Lille University, Inserm, Lille University Hospital, CREAK, U1286 - INFINITE - Institute for Translational Research in Inflammation, Lille, France

Publication date: September 25, 2022

This article was made available online on July 22, 2022 as a Fast Track article with title: "Long-term prophylaxis in hereditary angioedema management: current practices in France and unmet needs".

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