Cargando…

Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease

BACKGROUND: Pompe disease is a rare, progressive, metabolic disease, and the first treatable inheritable muscle disorder. Enzyme replacement therapy (ERT) with alglucosidase alfa is disease specific and the only medicinal product authorized for the treatment of Pompe disease. Costs of ERT are very h...

Descripción completa

Detalles Bibliográficos
Autores principales:	Kanters, Tim A., van der Ploeg, Ans T., Kruijshaar, Michelle E., Rizopoulos, Dimitris, Redekop, W. Ken, Rutten-van Mӧlken, Maureen P. M. H., Hakkaart-van Roijen, Leona
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2017
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5729274/ https://www.ncbi.nlm.nih.gov/pubmed/29237491 http://dx.doi.org/10.1186/s13023-017-0731-0

Ejemplares similares

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

A conceptual disease model for adult Pompe disease
por: Kanters, Tim A., 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)

Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions
por: Ditters, Imke A. M., et al.
Publicado: (2023)

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)

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)

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)

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)

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

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

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

STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa
por: Gutschmidt, Kristina, et al.
Publicado: (2021)

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

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)

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

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)

The influence of a polymorphism in the gene encoding angiotensin converting enzyme (ACE) on treatment outcomes in late-onset Pompe patients receiving alglucosidase alfa
por: Baek, Rena C., et al.
Publicado: (2016)

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)

A Multi-Centre Prospective Study of the Efficacy and Safety of Alglucosidase Alfa in Chinese Patients With Infantile-Onset Pompe Disease
por: Zhu, Diqi, et al.
Publicado: (2022)

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)

Earlier and higher dosing of alglucosidase alfa improve outcomes in patients with infantile-onset Pompe disease: Evidence from real-world experiences
por: Chien, Yin-Hsiu, et al.
Publicado: (2020)

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

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

Improved Enzyme Replacement Therapy with Cipaglucosidase Alfa/Miglustat in Infantile Pompe Disease
por: Fiege, Lina, et al.
Publicado: (2023)

Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy
por: Güngör, Deniz, et al.
Publicado: (2011)

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

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

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

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

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

In Absence of Absenteeism: Some Thoughts on Productivity Costs in Economic Evaluations in a Post-corona Era
por: Brouwer, Werner, et al.
Publicado: (2021)

Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease
por: Dimachkie, Mazen M., et al.
Publicado: (2022)

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

Systematic review of available evidence on 11 high-priced inpatient orphan drugs
por: Kanters, Tim A, et al.
Publicado: (2013)

Enzyme therapy and immune response in relation to CRIM status: the Dutch experience in classic infantile Pompe disease
por: van Gelder, Carin M., et al.
Publicado: (2014)

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

Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV
por: Yi, Haiqing, et al.
Publicado: (2016)

Update of the Dutch manual for costing studies in health care
por: Kanters, Tim A., et al.
Publicado: (2017)

Cannot write session to /tmp/vufind_sessions/sess_7u3iivcmo4bthaij133tp90vm7