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Article Dans Une Revue Allergy and Asthma Proceedings Année : 2022

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

1 CHUGA - Centre Hospitalier Universitaire [CHU Grenoble]
2 CREAK - Centre de référence national des angiœdèmes
3 TIMC - Translational Innovation in Medicine and Complexity / Recherche Translationnelle et Innovation en Médecine et Complexité - UMR 5525
4 CHU Saint-Antoine [AP-HP]
5 CHU Rouen
6 HCL - Hospices Civils de Lyon
7 CH Georges Renon Niort - Centre Hospitalier Georges Renon [Niort]
8 CHU Strasbourg - Centre Hospitalier Universitaire [Strasbourg]
9 DMIP - Brest - Département de Médecine Interne et Pneumologie [Brest]
10 PCCEI - Pathogenesis and Control of Chronic and Emerging Infections
11 UM - Université de Montpellier
12 CHU de Bordeaux Pellegrin [Bordeaux]
13 CHU Trousseau [Tours]
14 CHRU Nancy - Centre Hospitalier Régional Universitaire de Nancy
15 CHU Nice - Centre Hospitalier Universitaire de Nice
16 Centre Hospitalier Le Mans (CH Le Mans)
17 CHU Sud Saint Pierre [Ile de la Réunion]
18 CHU Nantes - Centre Hospitalier Universitaire de Nantes
19 CH Esquirol - CH Esquirol [Limoges]
20 CHU Caen
21 CHRU Besançon - Centre Hospitalier Régional Universitaire de Besançon
22 Maternité Port-Royal [CHU Cochin]
23 EPOPé [CRESS - U1153 / UMR_A 1125] - Obstetrical, Perinatal and Pediatric Epidemiology | Épidémiologie Obstétricale, Périnatale et Pédiatrique
24 CHU Toulouse - Centre Hospitalier Universitaire de Toulouse
25 CHU Nîmes - Centre Hospitalier Universitaire de Nîmes
26 Soladis
27 INFINITE (Ex-Liric) - Institute for Translational Research in Inflammation - U 1286
Olivier Fain
Guillaume Armengol
  • Fonction : Auteur
Delphine Gobert
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  • PersonId : 989516
Cyrille Hoarau
  • Fonction : Auteur
  • PersonId : 963538
Agathe Masseau
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  • PersonId : 934438
Yann Ollivier
  • Fonction : Auteur
Benoit Bouquillon
  • Fonction : Auteur
Edouard Verdier
  • Fonction : Auteur
Pierre Clerson
  • Fonction : Auteur

Résumé

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.
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Dates et versions

hal-03977467 , version 1 (07-02-2023)

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Laurence Bouillet, Olivier Fain, Guillaume Armengol, Magali Aubineau, Claire Blanchard-Delaunay, et al.. Long-term prophylaxis in hereditary angioedema management: Current practices in France and unmet needs. Allergy and Asthma Proceedings, 2022, 43 (5), pp.406-412. ⟨10.2500/aap.2022.43.220046⟩. ⟨hal-03977467⟩
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