| Title: |
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 |
| Authors: |
Lessard, Lola E.R.; Tard, Celine; Salort-Campana, Emmanuelle; Sacconi, Sabrina; Béhin, Anthony; Bassez, Guillaume; Orlikowski, David; Merle, Philippe; Nollet, Sylvain; Gallay, Laure; Bérard, Frédéric; Robinson, Philip; Bouhour, Françoise; Laforêt, Pascal |
| Contributors: |
Université de Lille; Inserm; CHU Lille; Lille Neurosciences & Cognition (LilNCog) - U 1172; Troubles cognitifs dégénératifs et vasculaires - U1171; 170930|||Centre de référence des maladies rares neuromusculaires (VALID); CHU Amiens-Picardie |
| Publisher Information: |
Elsevier |
| Publication Year: |
2024 |
| Collection: |
LillOA (Lille Open Archive - Université de Lille) |
| Subject Terms: |
Late-onset Pompe disease; Infusion-associated reaction; Enzyme-replacement therapy; Drug hypersensitivity reaction; Drug allergy; Desensitization |
| Description: |
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. ; 139;3 |
| Document Type: |
article in journal/newspaper |
| File Description: |
application/rdf+xml; charset=utf-8; application/pdf |
| Language: |
English |
| Relation: |
Molecular Genetics and Metabolism; Mol Genet Metab; http://hdl.handle.net/20.500.12210/101790 |
| Availability: |
https://hdl.handle.net/20.500.12210/101790 |
| Rights: |
Attribution-NonCommercial-NoDerivs 3.0 United States ; info:eu-repo/semantics/openAccess |
| Accession Number: |
edsbas.C865E0CE |
| Database: |
BASE |