Cargando…

Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions

BACKGROUND: Enzyme replacement therapy (ERT) with alglucosidase alfa is the treatment for patients with Pompe disease, a hereditary metabolic myopathy. Home-based ERT is unavailable in many countries because of the boxed warning alglucosidase alfa received due to the risk of infusion-associated reac...

Descripción completa

Detalles Bibliográficos
Autores principales:	Ditters, Imke A. M., van Kooten, Harmke A., van der Beek, Nadine A. M. E., Hardon, Jacqueline F., Ismailova, Gamida, Brusse, Esther, Kruijshaar, Michelle E., van der Ploeg, Ans T., van den Hout, Johanna M. P., Huidekoper, Hidde H.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Springer International Publishing 2023
Materias:	Original Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10432339/ https://www.ncbi.nlm.nih.gov/pubmed/37326923 http://dx.doi.org/10.1007/s40259-023-00609-2

Ejemplares similares

Are Anti-rhGAA Antibodies a Determinant of Treatment Outcome in Adults with Late-Onset Pompe Disease? A Systematic Review
por: Ditters, Imke A. M., et al.
Publicado: (2023)

Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study
por: Ditters, Imke A. M., et al.
Publicado: (2023)

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease
por: Kanters, Tim A., et al.
Publicado: (2017)

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease
por: Kanters, Tim A, et al.
Publicado: (2014)

Effects of higher and more frequent dosing of alglucosidase alfa and immunomodulation on long‐term clinical outcome of classic infantile Pompe patients
por: Poelman, Esther, et al.
Publicado: (2020)

Long-term benefits of physical activity in adult patients with late onset Pompe disease: a retrospective cohort study with 10 years of follow-up
por: Ismailova, Gamida, et al.
Publicado: (2023)

Antibodies against recombinant human alpha-glucosidase do not seem to affect clinical outcome in childhood onset Pompe disease
por: van Kooten, Harmke A., et al.
Publicado: (2022)

MRI changes in diaphragmatic motion and curvature in Pompe disease over time
por: Harlaar, Laurike, et al.
Publicado: (2022)

Enzyme replacement therapy and fatigue in adults with Pompe disease
por: Güngör, Deniz, et al.
Publicado: (2013)

Chest MRI to diagnose early diaphragmatic weakness in Pompe disease
por: Harlaar, Laurike, et al.
Publicado: (2021)

Phenotypical variation within 22 families with Pompe disease
por: Wens, Stephan C A, et al.
Publicado: (2013)

Mild disease course of SARS-CoV-2 infections and mild side effects of vaccination in Pompe disease: a cohort description
por: Ismailova, G., et al.
Publicado: (2022)

Impact of enzyme replacement therapy on survival in adults with Pompe disease
por: Güngör, Deniz, et al.
Publicado: (2013)

Association of Muscle Strength and Walking Performance in Adult Patients With Pompe Disease
por: Favejee, Marein M, et al.
Publicado: (2018)

Minutes of the European POmpe Consortium (EPOC) Meeting March 27 to 28, 2015, Munich, Germany
por: Schoser, Benedikt, et al.
Publicado: (2015)

Enzyme replacement therapy reduces the risk for wheelchair dependency in adult Pompe patients
por: van der Meijden, Jan C., et al.
Publicado: (2018)

Large variation in effects during 10 years of enzyme therapy in adults with Pompe disease
por: Harlaar, Laurike, et al.
Publicado: (2019)

Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study
por: van Gelder, C. M., et al.
Publicado: (2016)

Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy
por: van der Meijden, Jan C., et al.
Publicado: (2018)

A conceptual disease model for adult Pompe disease
por: Kanters, Tim A., et al.
Publicado: (2015)

Increased aortic stiffness and blood pressure in non-classic Pompe disease
por: Wens, Stephan C. A., et al.
Publicado: (2014)

Alglucosidase alfa: Long term use in the treatment of patients with Pompe disease
por: Beck, Michael
Publicado: (2009)

Alglucosidase alfa: 5 years of experience in late-onset Pompe disease
por: Schoser, Benedikt
Publicado: (2013)

Long-Term Experience with Anaphylaxis and Desensitization to Alglucosidase Alfa in Pompe Disease
por: Ertoy Karagol, Hacer Ilbilge, et al.
Publicado: (2023)

Is the brain involved in patients with late‐onset Pompe disease?
por: van den Dorpel, Jan J. A., et al.
Publicado: (2022)

Lung MRI and impairment of diaphragmatic function in Pompe disease
por: Wens, Stephan CA, et al.
Publicado: (2015)

Positive association between physical outcomes and patient-reported outcomes in late-onset Pompe disease: a cross sectional study
por: Yuan, Meng, et al.
Publicado: (2020)

Diffusion tensor imaging of the brain in Pompe disease
por: van den Dorpel, Jan J. A., et al.
Publicado: (2022)

Lack of robust satellite cell activation and muscle regeneration during the progression of Pompe disease
por: Schaaf, Gerben J., et al.
Publicado: (2015)

Hearing in adults with Pompe disease
por: van der Beek, Nadine A. M. E., et al.
Publicado: (2011)

Effects of exercise training in 23 adults with Pompe disease receiving enzyme therapy
por: van den Berg, Linda, et al.
Publicado: (2013)

Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study
por: Güngör, Deniz, et al.
Publicado: (2013)

Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel
por: Gragnaniello, Vincenza, et al.
Publicado: (2022)

Quality of life and participation in daily life of adults with Pompe disease receiving enzyme replacement therapy: 10 years of international follow-up
por: Güngör, Deniz, et al.
Publicado: (2015)

Neurofilament Light and Its Association With CNS Involvement in Patients With Classic Infantile Pompe Disease
por: Mackenbach, Maarten J., et al.
Publicado: (2023)

Newborn screening for pompe disease? a qualitative study exploring professional views
por: van El, Carla G, et al.
Publicado: (2014)

Broad variation in phenotypes for common GAA genotypes in Pompe disease
por: Niño, Monica Y., et al.
Publicado: (2021)

Burden of illness of Pompe disease in patients only receiving supportive care
por: Kanters, Tim A., et al.
Publicado: (2011)

Multicenter, non-interventional, double cohort study to assess the safety of alglucosidase alfa and laronidase in real-world home infusion setting
por: Toscano, Antonio, et al.
Publicado: (2022)

The ACE I/D polymorphism does not explain heterogeneity of natural course and response to enzyme replacement therapy in Pompe disease
por: Kuperus, Esther, et al.
Publicado: (2018)

Cannot write session to /tmp/vufind_sessions/sess_rul7ssbhqd5fsdvfkmbb1hk80n