Motor and respiratory decline in patients with late onset Pompe disease after cessation of enzyme replacement therapy during COVID‐19 pandemic. (8th January 2022)

• Record Type: Journal Article
• Authors: Tard, Céline; Salort‐Campana, Emmanuelle; Michaud, Maud; Spinazzi, Marco; Nadaj Pakleza, Aleksandra; Durr, Hélène; Bouhour, Françoise; Lefeuvre, Claire; Thomas, Romain; Arrassi, Azzeddine; Taouagh, Nadjib; Solé, Guilhem; Laforêt, Pascal
• Other Names: P Laforêt investigator.; D Orlikowski investigator.; G Bassez investigator.; D Germain investigator.; JB Noury investigator.; F Zagnoli investigator.; C Tard investigator.; JB Davion investigator.; E Salort‐Campana investigator.; A Verschueren investigator.; S Sacconi investigator.; A Furby investigator.; A Lacour investigator.; J Praline investigator.; G Sole investigator.; D Renard investigator.; F Bouibede investigator.; M Fournier investigator.; F Chapon investigator.; MC Minot investigator.; M Spinazzi investigator.; J Hubert investigator.; F Demurger investigator.; A Magot investigator.; P Cintas investigator.; A Nadaj‐Pakleza investigator.; F Bouhour investigator.; RJ Morales investigator.; F Esselin investigator.; P Petiot investigator.; R Jaussaud investigator.; S Pichard investigator.; A Brassier investigator.; J Do‐Cao investigator.; F Feillet investigator.; G Touati investigator.; YA Echaniz‐Laguna investigator.; L Magy investigator.; M Michaud investigator.; E Lagrange investigator.; E Krim investigator.; F Taithe investigator.; AL Bedat‐Millet investigator.; S Nollet investigator.; U Walter‐Louvier investigator.; B Chabrol investigator.; M Tardieu investigator.;
• Is Part Of: European journal of neurology. Volume 29:Number 4(2022)
• Page Start: 1181
• Page End: 1186
• Publication Date: 2022-01-08
• Subjects: alglucosidase alfa -- enzyme therapy -- stopping -- suspension -- treatment management; Neurology -- Periodicals; Nervous system -- Diseases -- Periodicals; 616.8
• DOI: 10.1111/ene.15222
• Languages: English
• ISSNs: 1351-5101

Abstract

Abstract: Background and purpose: Data on interruption of enzyme replacement therapy (ERT) are scarce in late onset Pompe disease. Due to the COVID‐19 crisis, eight neuromuscular reference centers in France were obligated to stop the treatment for 31 patients. Methods: We collected the motor and respiratory data from our French registry, before COVID‐19 and at treatment restart. Results: In 2.2 months (mean), patients showed a significant deterioration of 37 m (mean) in the 6‐min walk test and a loss of 210 ml (mean) of forced vital capacity, without ad integrum restoration after 3 months of ERT restart. Conclusions: This national study based on data from the French Pompe Registry shows that the interruption of ERT, even as short as a few months, worsens Pompe patients' motor and respiratory function. Abstract : Even brief suspension of approximately 2 months of enzyme therapy in patients with late onset Pompe disease results in significant motor and respiratory degradation. In this case, ad integrum recovery is not guaranteed 3 months after the treatment restart.