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4. Est-il important de phénotyper les patients recevant une VNI à domicile ? Analyse des résultats des premiers 2052 patients inclus dans la Cohorte ANTADIR-GAVO2

Author

Rabec, C., primary, Cuvelier, A., additional, Cervantes, P., additional, Morelot, C., additional, Georges, M., additional, Patout, M., additional, Pontier, S., additional, Mercy, M., additional, Guillaumot, A., additional, Prigent, A., additional, Gagnadoux, F., additional, Perrin, C., additional, Lavergne, F., additional, Mallart, A., additional, Grassion, L., additional, Kessler, R., additional, Claude, F., additional, Goubert, L., additional, Jaffre, S., additional, Prigent, H., additional, Perel, A., additional, Duval, Y., additional, Sedkaoui, K., additional, Chabot, F., additional, Llontop, C., additional, Berrier, A., additional, Delrieu, J., additional, Mba, L., additional, Muir, J.F., additional, Melloni, B., additional, and Gonzalez, J., additional

Published
2024
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6. P69 Scoping review on the assessment tools used on SMA adolescent and adult patients

Author

Hogrel, J., primary, Barrière, A., additional, Bonnyaud, C., additional, Boyer, F., additional, Gargiulo, M., additional, Li, D., additional, Montagu, G., additional, Berling, E., additional, Cintas, P., additional, Goff, L Le, additional, Marchadier, B., additional, Sekou, G N'Dah, additional, Orlikowksi, D., additional, Pouplin, S., additional, Prigent, H., additional, Ropars, J., additional, Salort-Campana, E., additional, Stojkovic, T., additional, Attarian, S., additional, and Laforêt, P., additional

Published
2023
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7. P70 What are the priorities of adolescents and adults with SMA and their health care practitioners toward evaluation? A French qualitative study

Author

Hogrel, J., primary, Berling, E., additional, Prigent, H., additional, Montagu, G., additional, Barrière, A., additional, Bonnyaud, C., additional, Boyer, F., additional, Cintas, P., additional, Gargiulo, M., additional, Le Goff, L., additional, Marchadier, B., additional, Sekou, N G'Dah, additional, Orlikowski, D., additional, Pouplin, S., additional, Pruvot, A., additional, Ropars, J., additional, Salort-Campana, E., additional, Stojkovic, T., additional, Attarian, S., additional, and Laforêt, P., additional

Published
2023
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9. Prognosis of right ventricular systolic dysfunction in Duchenne muscular dystrophy patients

Author

Orlikowski, D., primary, Mansencal, N., additional, Nguyen, S.L., additional, Wahbi, K., additional, Becane, H.-M., additional, Yaou, R. Ben, additional, Leturcq, F., additional, Lofaso, F., additional, Prigent, H., additional, Dubourg, O., additional, Stojkovic, T., additional, Fontaine, B., additional, Duboc, D., additional, Annane, D., additional, Laforet, P., additional, and Fayssoil, A., additional

Published
2023
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17. sj-pdf-4-crd-10.1177_14799731211036901 ��� Supplemental Material for Expiratory obstruction in patients with Duchenne muscular dystrophy under non-invasive ventilation: A step-by-step analysis of a new obstructive pattern

Author

L��otard, Antoine, Delorme, Mathieu, Delord, Vincent, Niel-Duriez, Myriam, Orlikowski, David, Annane, Djillali, Prigent, H��l��ne, and Lofaso, Fr��d��ric

Subjects
110203 Respiratory Diseases, FOS: Clinical medicine, 111702 Aged Health Care, FOS: Health sciences
Abstract

Supplemental Material, sj-pdf-4-crd-10.1177_14799731211036901 for Expiratory obstruction in patients with Duchenne muscular dystrophy under non-invasive ventilation: A step-by-step analysis of a new obstructive pattern by Antoine L��otard, Mathieu Delorme, Vincent Delord, Myriam Niel-Duriez, David Orlikowski, Djillali Annane, H��l��ne Prigent and Fr��d��ric Lofaso in Chronic Respiratory Disease

Published
2022
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18. sj-pdf-3-crd-10.1177_14799731211036901 ��� Supplemental Material for Expiratory obstruction in patients with Duchenne muscular dystrophy under non-invasive ventilation: A step-by-step analysis of a new obstructive pattern

Author

L��otard, Antoine, Delorme, Mathieu, Delord, Vincent, Niel-Duriez, Myriam, Orlikowski, David, Annane, Djillali, Prigent, H��l��ne, and Lofaso, Fr��d��ric

Subjects
110203 Respiratory Diseases, FOS: Clinical medicine, 111702 Aged Health Care, FOS: Health sciences
Abstract

Supplemental Material, sj-pdf-3-crd-10.1177_14799731211036901 for Expiratory obstruction in patients with Duchenne muscular dystrophy under non-invasive ventilation: A step-by-step analysis of a new obstructive pattern by Antoine L��otard, Mathieu Delorme, Vincent Delord, Myriam Niel-Duriez, David Orlikowski, Djillali Annane, H��l��ne Prigent and Fr��d��ric Lofaso in Chronic Respiratory Disease

Published
2022
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19. sj-pdf-1-crd-10.1177_14799731211036901 ��� Supplemental Material for Expiratory obstruction in patients with Duchenne muscular dystrophy under non-invasive ventilation: A step-by-step analysis of a new obstructive pattern

Author

L��otard, Antoine, Delorme, Mathieu, Delord, Vincent, Niel-Duriez, Myriam, Orlikowski, David, Annane, Djillali, Prigent, H��l��ne, and Lofaso, Fr��d��ric

Subjects
110203 Respiratory Diseases, FOS: Clinical medicine, 111702 Aged Health Care, FOS: Health sciences
Abstract

Supplemental Material, sj-pdf-1-crd-10.1177_14799731211036901 for Expiratory obstruction in patients with Duchenne muscular dystrophy under non-invasive ventilation: A step-by-step analysis of a new obstructive pattern by Antoine L��otard, Mathieu Delorme, Vincent Delord, Myriam Niel-Duriez, David Orlikowski, Djillali Annane, H��l��ne Prigent and Fr��d��ric Lofaso in Chronic Respiratory Disease

Published
2022
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20. sj-pdf-2-crd-10.1177_14799731211036901 ��� Supplemental Material for Expiratory obstruction in patients with Duchenne muscular dystrophy under non-invasive ventilation: A step-by-step analysis of a new obstructive pattern

Author

L��otard, Antoine, Delorme, Mathieu, Delord, Vincent, Niel-Duriez, Myriam, Orlikowski, David, Annane, Djillali, Prigent, H��l��ne, and Lofaso, Fr��d��ric

Subjects
110203 Respiratory Diseases, FOS: Clinical medicine, 111702 Aged Health Care, FOS: Health sciences
Abstract

Supplemental Material, sj-pdf-2-crd-10.1177_14799731211036901 for Expiratory obstruction in patients with Duchenne muscular dystrophy under non-invasive ventilation: A step-by-step analysis of a new obstructive pattern by Antoine L��otard, Mathieu Delorme, Vincent Delord, Myriam Niel-Duriez, David Orlikowski, Djillali Annane, H��l��ne Prigent and Fr��d��ric Lofaso in Chronic Respiratory Disease

Published
2022
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24. Mouthpiece ventilation in neuromuscular disorders: Narrative review of technical issues important for clinical success

Author

Toussaint M, Chatwin M, Goncalves M, Gonzalez-Bermejo J, Benditt J, McKim D, Sancho J, Hov B, Sansone V, Prigent H, Carlucci A, Wijkstra P, Garabelli B, Escarrabill J, Pinto T, Audag N, Verweij-van den Oudenrijn L, Ogna A, Hughes W, Devaux C, Chaulet J, Andersen T, and ENMC Respiratory Therapy Consortium

Abstract

In neuromuscular disorders (NMDs), nocturnal non-invasive ventilation (NIV) via a nasal mask is offered when hypercapnic respiratory failure occurs. With disease progression, nocturnal NIV needs to be extended into the daytime. Mouthpiece ventilation (MPV) is an option for daytime NIV. MPV represents a difficult task for home ventilators due to rapidly changing load conditions resulting from intermittent connections and disconnections from MPV circuit. The 252nd ENMC International Expert Workshop, held March 6th to 8th 2020 in Amsterdam, reported general guidelines for management of daytime MPV in NMDs. This report could not present all the detail regarding the technical issues important for clinical success of MPV. Based on the expert workshop discussions and the evidence from existing studies, the current narrative review aims to identify the technical issues of MPV and offers guidance via a decisional algorithm and educational figures providing relevant information that is important for successful implementation of MPV.

Published
2021

28. Long-term benefit of enzyme replacement therapy with alglucosidase alfa in adults with Pompe disease: prospective analysis from the French Pompe Registry

Author

Semplicini, C., de Antonio, M., Taouagh, N., Béhin, A., Bouhour, F., Echaniz-Laguna, A., Magot, A., Nadaj-Pakleza, A., Orlikowski, D., Sacconi, S., Salort-Campana, E., Solé, G., Tard, Celine, Zagnoli, F., Jean-Yves, H., Hamroun, D., Laforêt, P., Attarian, S., Aubé-Nathier, A. C., Arrassi, A., Bassez, G., Bedat-Millet, A. L., Bouibede, F., Boyer, F. C., Caillaud, C., Canal, A., Carlier, R. Y., Chanson, J. B., Chapon, F., Cintas, P., Deibener-Kaminsky, J., Demurger, F., Desnuelle, C., Durieu, I., Eymard, B., Feasson, L., Fournier, M., Froissart, R., Furby, A., Garcia, P. Y., Germain, D. P., Ghorab, K., Morales, R. J., Krim, E., Labauge, P., Lacour, A., Lagrange, E., Lefeuvre, C., Leguy-Seguin, V., Leonard-Louis, S., Magy, L., Masseau, A., Michaud, M., Minot-Myhié, M. C., Nicolas, G., Nollet, S., Not, A., Noury, J. B., Ollivier, G., Péréon, Y., Perez, Thierry, Perniconi, B., Piraud, M., Petiot, P., Pouget, J., Praline, J., Prigent, H., Renard, D., Spinazzi, M., Stojkovic, T., Taithe, F., Tiffreau, Vincent, Vincent, D., Lille Neurosciences & Cognition - U 1172 (LilNCog), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Lille-Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille), Unité de Recherche Pluridisciplinaire Sport, Santé, Société (URePSSS) - ULR 7369 - ULR 4488 (URePSSS), and Université d'Artois (UA)-Université du Littoral Côte d'Opale (ULCO)-Université de Lille

Subjects
Adult, Male, Pediatrics, medicine.medical_specialty, Registry, alglucosidase alfa, Adolescent, [SDV]Life Sciences [q-bio], Walk Test, Disease, Sitting, 03 medical and health sciences, FEV1/FVC ratio, Young Adult, Glycogen storage disease type II, Genetics, medicine, Humans, Respiratory function, Prospective Studies, Registries, Child, Alglucosidase alfa, Genetics (clinical), 030304 developmental biology, Aged, 0303 health sciences, late onset Pompe disease, business.industry, 030305 genetics & heredity, alpha-Glucosidases, Enzyme replacement therapy, Middle Aged, medicine.disease, Respiratory Function Tests, Treatment Outcome, Ceiling effect, Female, France, business, medicine.drug, enzyme replacement therapy
Abstract

Despite a wide clinical spectrum, the adult form of Pompe disease is the most common one, and represents more than 90% of diagnosed patients in France. Since the marketing of enzyme replacement therapy (alglucosidase alfa, Myozyme), all reports to date in adults demonstrated an improvement of the walking distance, and a trend toward stabilization of respiratory function, but the majority of these studies were less than 5 years of duration. We report here the findings from 158 treated patients included in the French Pompe Registry, who underwent regular clinical assessments based on commonly used standardized tests (6-minute walking test, MFM scale, sitting vital capacity, MIP and MEP). For longitudinal analyses, the linear mixed effects models were used to assess trends in primary endpoints over time under ERT. A two-phase model better described the changes in distance traveled in the 6-minute walk test and MFM. 6MWT showed an initial significant increase (1.4% ± 0.5/year) followed by a progressive decline (-2.3%/year), with a cut-off point at 2.2 years. A similar pattern was observed in total MFM score (6.6% ± 2.3/year followed by a - 1.1%/year decline after 0.5 years). A single-phase decline with a slope of -0.9 ± 0.1%/year (P < .001) was observed for FVC, and MEP remained stable over the all duration of follow-up. This study provides further evidence that ERT improves walking abilities and likely stabilizes respiratory function in adult patients with Pompe disease, with a ceiling effect for the 6MWT in the first 3 years of treatment.

Published
2020

34. Nutrition et ventilation : le dilemme du patient myopathe adulte de type Duchenne. Analyse d’une cohorte de 117 patients en VAD (ventilation à domicile)

Author

Fayssol, A., primary, Chaffaut, C., additional, Prigent, H., additional, Laforet, P., additional, Clair, B., additional, Orlikowsky, D., additional, Ogna, A., additional, Chevret, S., additional, Meng, P., additional, Annane, D., additional, Lofaso, F., additional, and Crenn, P., additional

Published
2020
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40. 233rd ENMC International Workshop:: Clinical Trial Readiness for Calpainopathies, Naarden, The Netherlands, 15–17 September 2017

Author

Lostal, W, Urtizberea, Ja, Richard, I, Alonso-Jiménez, A, Carlier, Ry, Carson, V, Diaz-Manera, J, Eymard, BRUNO, MICHEL, ANTOINE, JOSEPH, MARIE, Fardeau, M, Gourlay, Ml, Guglieri, M, Hogrel, Jy, Kullmann, B, Levy, J1, Ono, Y, Prigent, H, Saenz, A, Semplicini, C, Vainzof, M, Vissing, J, and Walter, M.

Subjects
Muscular Dystrophies, Limb-Girdle, Neurology, Disease Progression, Humans, Pediatrics, Perinatology and Child Health, Neurology (clinical), Genetics (clinical), Perinatology and Child Health, Pediatrics, Education, Netherlands
Published
2018

41. Diaphragm: Pathophysiology and Ultrasound Imaging in Neuromuscular Disorders

Author

Fayssoil, A., Behin, A., Ogna, A., Mompoint, D., Amthor, H., Clair, B., Laforet, P., Mansart, A., Prigent, H., Orlikowski, D., Stojkovic, T., Vinit, S., Carlier, R., Eymard, B., Lofaso, F., and Annane, D.

Subjects
Diaphragm/diagnostic imaging, Diaphragm/physiopathology, Humans, Neuromuscular Diseases/diagnostic imaging, Neuromuscular Diseases/physiopathology, Ultrasonography/methods, Diaphragm, neuromuscular disorders, ultrasound, musculoskeletal system
Abstract

Respiratory muscles are classically involved in neuromuscular disorders, leading to a restrictive respiratory pattern. The diaphragm is the main respiratory muscle involved during inspiration. Ultrasound imaging is a noninvasive, radiation-free, accurate and safe technique allowing assessment of diaphragm anatomy and function. The authors review the pathophysiology of diaphragm in neuromuscular disorders, the methodology and indications of diaphragm ultrasound imaging as well as possible pitfalls in the interpretation of results.

Published
2018

44. Cohorte ANTADIR-GAV de patients insuffisants respiratoires nouvellement traités par ventilation non invasive à domicile en France. Résultats à 18 mois

Author

Rabec, C., primary, Cuvelier, A., additional, Melloni, B., additional, Cervantes, P., additional, Morelot, C., additional, Georges, M., additional, Pontier, S., additional, Guillaumot, A., additional, Mercy, M., additional, Gagnadoux, F., additional, Perrin, C., additional, Lavergne, F., additional, Prigent, A., additional, Mallart, A., additional, Hilbert, G., additional, Kessler, R., additional, Claude, F., additional, Goubert, L., additional, Prigent, H., additional, Jaffre, S., additional, Perel, A., additional, Llontop, C., additional, Sedkaoui, K., additional, Foret, D., additional, Mounier, L., additional, Muir, F., additional, and Gonzalez, J., additional

Published
2018
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46. Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

Author

Masat, Elisa, Laforêt, Pascal, De Antonio, Marie, Corre, Guillaume, Perniconi, Barbara, Taouagh, Nadjib, Mariampillai, Kuberaka, Amelin, Damien, Mauhin, Wladimir, Hogrel, Jean-Yves, Caillaud, Catherine, Ronzitti, Giuseppe, Puzzo, Francesco, Kuranda, Klaudia, Colella, Pasqualina, Mallone, Roberto, Benveniste, Olivier, Mingozzi, Federico, Bassez, G., Bedat-Millet, A. L., Behin, A., Eymard, B., Leonard-Louis, S., Stojkovic, T., Canal, A., Decostre, V., Bouhour, F., Boyer, F., Castaing, Y., Chapon, F., Cintas, P., Durieu, I., Echaniz-Laguna, A., Feasson, L., Furby, A., Hamroun, D., Ferrer, X., Solé, G., Froissart, R., Piraud, M., Germain, D., Benistan, K., Guffon-Fouilhoux, N., Journel, H., Labauge, P., Lacour, A., Levy, A., Magot, A., Péréon, Y., Minot-Myhié, M. -C., Nadaj-Pakleza, A., Nathier, C., Orlikowski, D., Pellegrini, N., Petiot, P., Praline, J., Lofaso, F., Prigent, H., Dutry, A., Renard, D., Sacconi, S., Desnuelle, C., Salort-Campana, E., Pouget, J., Tiffreau, V., Vincent, D., Zagnoli, F., Centre de recherche en myologie, Université Pierre et Marie Curie - Paris 6 (UPMC)-Association française contre les myopathies (AFM-Téléthon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Institut de Myologie, Université Pierre et Marie Curie - Paris 6 (UPMC)-Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Association française contre les myopathies (AFM-Téléthon)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Centre de Recherche des Cordeliers (CRC), Université Paris Diderot - Paris 7 (UPD7)-École pratique des hautes études (EPHE), Université Paris sciences et lettres (PSL)-Université Paris sciences et lettres (PSL)-Université Pierre et Marie Curie - Paris 6 (UPMC)-Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), Généthon, CHU Pitié-Salpêtrière [AP-HP], Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Service de biochimie métabolique [CHU Necker], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-CHU Necker - Enfants Malades [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Institut Cochin (IC UM3 (UMR 8104 / U1016)), Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Service de diabétologie [CHU Cochin], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Cochin [AP-HP], Université Pierre et Marie Curie - Paris 6 (UPMC)-École Pratique des Hautes Études (EPHE), Université Paris sciences et lettres (PSL)-Université Paris sciences et lettres (PSL)-Université Paris Diderot - Paris 7 (UPD7)-Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Centre National de la Recherche Scientifique ( CNRS ) -Institut National de la Santé et de la Recherche Médicale ( INSERM ) -Association française contre les myopathies ( AFM-Téléthon ) -Université Pierre et Marie Curie - Paris 6 ( UPMC ), Université Pierre et Marie Curie - Paris 6 ( UPMC ) -Commissariat à l'énergie atomique et aux énergies alternatives ( CEA ) -Assistance publique - Hôpitaux de Paris (AP-HP)-Association française contre les myopathies ( AFM-Téléthon ) -Institut National de la Santé et de la Recherche Médicale ( INSERM ) -Centre National de la Recherche Scientifique ( CNRS ), Centre de Recherche des Cordeliers ( CRC ), Université Paris Diderot - Paris 7 ( UPD7 ) -École pratique des hautes études ( EPHE ) -Université Pierre et Marie Curie - Paris 6 ( UPMC ) -Université Paris Descartes - Paris 5 ( UPD5 ) -Institut National de la Santé et de la Recherche Médicale ( INSERM ), GENETHON, Genethon, DHUI2B, CHU Pitié-Salpêtrière [APHP], CHU Necker - Enfants Malades [AP-HP]-Assistance publique - Hôpitaux de Paris (AP-HP), Institut Cochin ( UM3 (UMR 8104 / U1016) ), Université Paris Descartes - Paris 5 ( UPD5 ) -Institut National de la Santé et de la Recherche Médicale ( INSERM ) -Centre National de la Recherche Scientifique ( CNRS ), Assistance publique - Hôpitaux de Paris (AP-HP)-CHU Cochin [AP-HP], Université Pierre et Marie Curie - Paris 6 [UPMC], CHU Necker - Enfants Malades [AP-HP], Hôpital Cochin [AP-HP], Hôpital Henri Mondor, CHU Rouen, Hôpital neurologique et neurochirurgical Pierre Wertheimer [CHU - HCL], Centre Hospitalier Universitaire de Reims [CHU Reims], CHU de Bordeaux Pellegrin [Bordeaux], CHU Caen, Centre d'investigation clinique de Toulouse [CIC 1436], Centre Hospitalier Lyon Sud [CHU - HCL] [CHLS], Centre Hospitalier Universitaire de Saint-Etienne [CHU Saint-Etienne] [CHU ST-E], CHU Montpellier, CHU Bordeaux [Bordeaux], Hôpital de l'Hôtel Dieu [CHU-HCL], Centre Hospitalier Régional Universitaire [Lille] [CHRU Lille], Centre hospitalier universitaire de Nantes [CHU Nantes], CHU Pontchaillou [Rennes], Centre Hospitalier Universitaire d'Angers [CHU Angers], Hôpital Raymond Poincaré [AP-HP], Hôpital de la Croix-Rousse [CHU - HCL], Centre Hospitalier Régional Universitaire de Tours [CHRU Tours], Centre Hospitalier Universitaire de Nice [CHU Nice], Hôpital de la Timone [CHU - APHM] [TIMONE], Unité de Recherche Pluridisciplinaire Sport, Santé, Société (URePSSS) - ULR 7369 - ULR 4488 [URePSSS], Hôpital d'Instruction des Armées Clermont Tonnerre, Université Pierre et Marie Curie - Paris 6 (UPMC)-École pratique des hautes études (EPHE), and HAL UPMC, Gestionnaire

Subjects
0301 basic medicine, Adult, Male, Chemokine, congenital, hereditary, and neonatal diseases and abnormalities, [SDV.IMM] Life Sciences [q-bio]/Immunology, T cell, T-Lymphocytes, Antibodies, Article, 03 medical and health sciences, 0302 clinical medicine, Immune system, Medicine, [ SDV.IMM ] Life Sciences [q-bio]/Immunology, Humans, Enzyme Replacement Therapy, Age of Onset, Aged, [SDV.MHEP.EM] Life Sciences [q-bio]/Human health and pathology/Endocrinology and metabolism, Multidisciplinary, biology, business.industry, Glycogen Storage Disease Type II, Immunogenicity, Case-control study, Antibody titer, nutritional and metabolic diseases, alpha-Glucosidases, Enzyme replacement therapy, Dendritic Cells, [ SDV.MHEP.EM ] Life Sciences [q-bio]/Human health and pathology/Endocrinology and metabolism, Middle Aged, [SDV.MHEP.EM]Life Sciences [q-bio]/Human health and pathology/Endocrinology and metabolism, 3. Good health, 030104 developmental biology, medicine.anatomical_structure, Treatment Outcome, Case-Control Studies, Immunoglobulin G, Immunology, biology.protein, Interleukin-2, [SDV.IMM]Life Sciences [q-bio]/Immunology, Female, Antibody, business, 030217 neurology & neurosurgery
Abstract

Immunogenicity of recombinant human acid-alpha glucosidase (rhGAA) in enzyme replacement therapy (ERT) is a safety and efficacy concern in the management of late-onset Pompe disease (LOPD). However, long-term effects of ERT on humoral and cellular responses to rhGAA are still poorly understood. To better understand the impact of immunogenicity of rhGAA on the efficacy of ERT, clinical data and blood samples from LOPD patients undergoing ERT for >4 years (n = 28) or untreated (n = 10) were collected and analyzed. In treated LOPD patients, anti-rhGAA antibodies peaked within the first 1000 days of ERT, while long-term exposure to rhGAA resulted in clearance of antibodies with residual production of non-neutralizing IgG. Analysis of T cell responses to rhGAA showed detectable T cell reactivity only after in vitro restimulation. Upregulation of several cytokines and chemokines was detectable in both treated and untreated LOPD subjects, while IL2 secretion was detectable only in subjects who received ERT. These results indicate that long-term ERT in LOPD patients results in a decrease in antibody titers and residual production of non-inhibitory IgGs. Immune responses to GAA following long-term ERT do not seem to affect efficacy of ERT and are consistent with an immunomodulatory effect possibly mediated by regulatory T cells.

Published
2016

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