Titre :

Hypersensitivity infusion-associated reactions induced by enzyme replacement therapy in a cohort of patients with late-onset Pompe disease: An experience from the French Pompe Registry

Auteur(s) :

Lessard, Lola E.R. [Auteur]
Tard, Celine [Auteur]
170930|||Centre de référence des maladies rares neuromusculaires (VALID)
Troubles cognitifs dégénératifs et vasculaires - U1171
Lille Neurosciences & Cognition (LilNCog) - U 1172
Salort-Campana, Emmanuelle [Auteur]
Sacconi, Sabrina [Auteur]
Béhin, Anthony [Auteur]
Bassez, Guillaume [Auteur]
Orlikowski, David [Auteur]
Merle, Philippe [Auteur]
CHU Amiens-Picardie
Nollet, Sylvain [Auteur]
Gallay, Laure [Auteur]
Bérard, Frédéric [Auteur]
Robinson, Philip [Auteur]
Bouhour, Françoise [Auteur]
Laforêt, Pascal [Auteur]

Titre de la revue :

Molecular Genetics and Metabolism

Nom court de la revue :

Mol Genet Metab

Numéro :

139

Pagination :

107611

Éditeur :

Elsevier

Date de publication :

2023-07

ISSN :

1096-7206

Mot(s)-clé(s) en anglais :

Late-onset Pompe disease
Infusion-associated reaction
Enzyme-replacement therapy
Drug hypersensitivity reaction
Drug allergy
Desensitization

Discipline(s) HAL :

Sciences du Vivant [q-bio]

Résumé en anglais : [en]

Background and objectives Pompe disease is a rare hereditary glycogen storage disorder due to lysosomal acid alpha-glucosidase deficiency. Enzyme replacement therapy (ERT) is the only available treatment. Infusion-associated ...
Lire la suite >Background and objectives Pompe disease is a rare hereditary glycogen storage disorder due to lysosomal acid alpha-glucosidase deficiency. Enzyme replacement therapy (ERT) is the only available treatment. Infusion-associated reactions (IAR) are challenging since there are no guidelines for ERT rechallenge after a drug hypersensitivity reaction (DHR) in Pompe disease. The objective of the present study was to describe IAR and their management in late-onset Pompe disease (LOPD) patients in France, and to discuss the various possibilities of ERT rechallenge. Methods An exhaustive screening of LOPD patients receiving ERT between 2006 and 2020 from the 31-participating hospital-based or reference centers was performed. The patients who had presented at least one hypersensitivity IAR (=DHR) episode were included. Demographic characteristics of the patients, IAR onset and timing, were retrospectively collected from the French Pompe Registry. Results Fifteen patients among 115 treated LOPD patients in France presented at least 1 IAR; 80.0% were women. Twenty-nine IAR were reported; 18 (62.1%) IAR were Grade I reactions, 10 (34.5%) IAR were Grade II, and 1 (3.4%) IAR was Grade III. IgE-mediated hypersensitivity was found in 2/15 patients (13.3%). The median [IQR] time from ERT introduction to the first IAR was 15.0 months [11.0–24.0]. ERT was safely and effectively re-introduced either with premedication alone, or in combination with either modified regimen or desensitization protocol, in all 9 rechallenged patients; including in patients with IgE-mediated hypersensitivity, in the patient with the Grade III reaction, as well as in patients with very high anti-GAA titer. Discussion Based on the results herein and previous reports, we discuss premedication and modified regimen for Grade I reactions, and desensitization in Grade II and III reactions. In conclusion, ERT-induced IAR can be safely and effectively managed with a modified regimen or desensitization protocol in LOPD patients.Lire moins >

Langue :

Anglais

Comité de lecture :

Oui

Audience :

Internationale

Vulgarisation :

Non

Établissement(s) :

Université de Lille
Inserm
CHU Lille

Collections :

• Lille Neurosciences & Cognition (LilNCog) - U 1172

Équipe(s) de recherche :

Troubles cognitifs dégénératifs et vasculaires

Date de dépôt :

2024-01-15T22:52:11Z
2024-11-15T14:43:05Z